Archive for the ‘Gene Therapy Research’ Category

Sickle cell disease. Image: Flickr

Sickle cell disease is a slow, vicious killer. Most people diagnosed with the red blood cell disorder in the US live to be between 40 and 60. But those years are a lifetime of pain, as abnormal, crescent-shaped hemoglobin stops up blood flow and deprives tissues of oxygen, causing frequent bouts of agony, along with more severe consequences like organ damage. Now, after decades of searching for a cure, researchers are announcing that, in at least one patient, they seem to have found a very promising treatment.

Two years ago, a French teen with sickle cell disease underwent a gene therapy treatment intended to help his red blood cells from sickling. In a paper published Thursday in the New England Journal of Medicine, the researchers revealed that today, half of his red blood cells have normal-shaped hemoglobin. He has not needed a blood transfusion, which many sickle cell patients receive to reduce complications from the disease, since three months after his treatment. He is also off all medicines.

To reiterate, the paper is a case study of just one patient. Bluebird Bio, the Massachusetts biotech company that sponsored the clinical trial, has treated at least six other trials underway in the US and France, but those results have not yet been fully reported. The gene therapy has not worked quite as well in some of those other patients; researchers say they are adjusting the therapy accordingly. It is also possible that the boy may eventually experience some blood flow blockages again in the future.

The results, though early, are encouraging. They represent the promise of new genetics technologies to address a disease that has long been neglected and tinged with racism. Sickle cell disease affects about 100,000 people in the US, most of whom are black. It is an inherited genetic disease caused by a mutation of a single letter in a persons genetic code.

This single-letter mutation makes it a promising candidate for cutting edge technologies, like the gene-editing technique CRISPR-Cas9, and other gene therapies. Recently, a rush of new research has sought to address it. Two other gene therapy studies for sickle cell are underway in the US one at UCLA and another at Cincinnati Childrens Hospital. Yet another is about to start in a collaboration between Harvard and Boston Childrens Hospital. Last fall, researchers all demonstrated the ability to correct the mutation in human cells using CRISPR, though that strategy will yet have to surpass significant scientific and political hurdles before reaching clinical trials.

In the new study, researchers took bone marrow stem cells from the boy and fed them corrected versions of a gene that codes for beta-globin, a protein that helps produce normal hemoglobin. The hope was that those altered stem cells would interfere with the boys faulty proteins and allow his red blood cells to function normally. They continued the transfusions until the transplanted cells began to produce normal-shaped hemoglobin. In the following months, the numbers of those cells continued to increase until in December 2016, they accounted for more than half the red blood cells in his body. In other words, so far so good.

Currently, the only long-term treatment for sickle cell disease is a bone marrow transplant, a high-risk, difficult procedure which many patients are not even eligible for. Pain and other side-effects are treated with blood transfusions for temporary relief. New technologies offer the hope of a solution that could provide long-term relief and allow patients to live some semblance of a normal life.

For decades, gene therapies have been touted as a cure for everything. But so far, successes have been infrequent, and often for very rare diseases. But early success in treating sickle cell disease means that soon, if were lucky, the benefits of this technology may reach hundreds of thousands of people.

[New England Journal of Medicine]

Read the original post:
Will Sickle Cell Be the Next Disease Genetic Engineering Cures? - Gizmodo

"The numbers are fantastic," said Dr Fred Locke, a blood cancer expert at Moffitt Cancer Center in Tampa who co-led the study.

"These are heavily treated patients who have no other options."

The treatment, which has been dubbed 'a living drug' by doctors, works by filtering a patient's blood to remove key immune system cells called T-cells, which are then genetically engineered in the lab to recognise cancer cells.

Cancer cells are very good a evading the immune system, but the new therapy essentially cuts the brakes, allowing immune cells to do their job properly.

Martin Ledwick, Cancer Research UKs head cancer information nurse, said: These results are promising and suggest that one day CAR-T cells could become a treatment option for some patients with certain types of lymphoma.

"But, we need to know more about the side effects of the treatment and long term benefits.

Read this article:
Terminal cancer patients in complete remission after one gene therapy treatment - Telegraph.co.uk

March 1, 2017 by Marilynn Marchione This 2009 colorized microscope image made available by the Sickle Cell Foundation of Georgia via the Centers for Disease Control and Prevention shows a sickle cell, left, and normal red blood cells of a patient with sickle cell anemia. Researchers say a French teen who was given gene therapy for sickle cell disease more than two years ago now has enough properly working red blood cells to dodge the effects of the disorder. The case is detailed in the March 2, 2017 issue of the New England Journal of Medicine. (Janice Haney Carr/CDC/Sickle Cell Foundation of Georgia via AP)

A French teen who was given gene therapy for sickle cell disease more than two years ago now has enough properly working red blood cells to dodge the effects of the disorder, researchers report.

The first-in-the-world case is detailed in Thursday's New England Journal of Medicine.

About 90,000 people in the U.S., mostly blacks, have sickle cell, the first disease for which a molecular cause was found. Worldwide, about 275,000 babies are born with it each year.

"Vexing questions of race and stigma have shadowed the history of its medical treatment," including a time when blacks who carry the bad gene were urged not to have children, spurring accusations of genocide, Keith Wailoo of Princeton University wrote in a separate article in the journal.

The disease is caused by a single typo in the DNA alphabet of the gene for hemoglobin, the stuff in red blood cells that carries oxygen. When it's defective, the cells sickle into a crescent shape, clogging tiny blood vessels and causing bouts of extreme pain and sometimes more serious problems such as strokes and organ damage. It keeps many people from playing sports and enjoying other activities of normal life.

A stem cell transplant from a blood-matched sibling is a potential cure, but in the U.S., fewer than one in five people have a donor like that. Pain crises are treated with blood transfusions and drugs, but they're a temporary fix. Gene therapy offers hope of a lasting one.

The boy, now 15, was treated at Necker Children's Hospital in Paris in October 2014. Researchers gave him a gene, taken up by his blood stem cells, to help prevent the sickling. Now, about half of his red blood cells have normal hemoglobin; he has not needed a transfusion since three months after his treatment and is off all medicines.

"It's not a cure but it doesn't matter," because the disease is effectively dodged, said Philippe Leboulch, who helped invent the therapy and helped found Bluebird Bio in Cambridge, Massachusetts, the company that treated the boy. The work was supported by a grant from the French government's research agency.

Bluebird has treated at least six others in the U.S. and France. Full results have not been reported, but the gene therapy has not taken hold as well in some of them as it did in the French teen. Researchers think they know why and are adjusting methods to try to do better.

Two other gene therapy studies for sickle cell are underway in the U.S.at the University of California, Los Angeles and Cincinnati Children's Hospitaland another is about to start at Harvard and Boston Children's Hospital using a little different approach.

"This work gives considerable promise" for a solution to a very common problem, said Dr. Stuart Orkin, a Boston Children's Hospital doctor who is an inventor on a patent related to gene editing.

"The results are quite good in this patient," he said of the French teen. "It shows gene therapy is on the right track."

Explore further: BCL11A-based gene therapy for sickle cell disease passes key preclinical test

More information: Gene therapy: ghr.nlm.nih.gov/primer/therapy/availability

2017 The Associated Press. All rights reserved.

A precision-engineered gene therapy virus, inserted into blood stem cells that are then transplanted, markedly reduced sickle-induced red-cell damage in mice with sickle cell disease, researchers from Dana-Farber/Boston Children's ...

Sickle cell disease and the blood disorder beta thalassemia affect more than 180,000 Americans and millions more worldwide. Both diseases can be made milder or even cured by increasing fetal hemoglobin (HbF) levels, but current ...

Scientists at the Center for Regenerative Medicine (CReM) at Boston Medical Center (BMC) and Boston University School of Medicine (BUSM) are creating an induced pluripotent stem cell (iPSC)-based research library that opens ...

UCLA stem cell researchers have shown that a novel stem cell gene therapy method could lead to a one-time, lasting treatment for sickle cell diseasethe nation's most common inherited blood disorder.

A team of researchers at the Stanford University School of Medicine has used a gene-editing tool known as CRISPR to repair the gene that causes sickle cell disease in human stem cells, which they say is a key step toward ...

Scientists have developed a new approach to repair a defective gene in blood-forming stem cells from patients with a rare genetic immunodeficiency disorder called X-linked chronic granulomatous disease (X-CGD). After transplant ...

(Medical Xpress)A team of researchers from China and Japan has found that BHPF, a replacement chemical for BPA in plastics, can also cause estrogen-related problems in mice. In their paper published in the journal Nature ...

Ionizing radiation incidentsnuclear war, nuclear accidents or terrorist dirty bombs, for examplecan cause mass fatalities. Since resources for medical countermeasures are limited, it's critically important to swiftly ...

Using antibodies to treat disease has been one of the great success stories of early 21st-century medicine. Already five of the ten top-selling pharmaceuticals in the United States are antibody products. But antibodies are ...

As people get older so do the hematopoietic stem cells (HSCs) that form their blood, creating an increased risk for compromised immunity and certain blood cancers. Now researchers are reporting in the scientific journal EMBO ...

A French teen who was given gene therapy for sickle cell disease more than two years ago now has enough properly working red blood cells to dodge the effects of the disorder, researchers report.

A research team, led by the University of Minnesota, has discovered a groundbreaking process to successfully rewarm large-scale animal heart valves and blood vessels preserved at very low temperatures. The discovery is a ...

Adjust slider to filter visible comments by rank

Display comments: newest first

Let's see. AIDS kills young white Americans, Sickle cell kills young black Americans. Let's just give all the research funding to AIDS!

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

See more here:
Gene therapy lets a French teen dodge sickle cell disease - Medical Xpress

Listen Download Podcast

News bulletin 03/02 04h00 GMT

Features and analysis 03/02 04h10 GMT

News bulletin 03/02 05h00 GMT

Features and analysis 03/02 05h10 GMT

News bulletin 03/02 06h00 GMT

Features and analysis 03/02 06h10 GMT

News bulletin 03/02 06h30 GMT

Features and analysis 03/02 06h33 GMT

News bulletin 03/02 07h00 GMT

News bulletin 03/02 07h30 GMT

News bulletin 02/26 14h00 GMT

News bulletin 03/02 14h00 GMT

Features and analysis 02/26 14h03 GMT

Features and analysis 03/02 14h06 GMT

News bulletin 03/02 14h30 GMT

Features and analysis 03/02 14h33 GMT

News bulletin 02/26 16h00 GMT

News bulletin 03/02 16h00 GMT

Features and analysis 02/26 16h03 GMT

News bulletin 03/02 16h30 GMT

Features and analysis 02/26 16h33 GMT

For an optimal navigation, the RFI website requires JavaScript to be enabled in your browser. To take full advantage of multimedia content, you must have the Flash plugin installed in your browser. To connect, you need to enable cookies in your browser settings. For an optimal navigation, the RFI site is compatible with the following browsers: Internet Explorer 8 and above, Firefox 10 and +, Safari 3+, Chrome 17 and + etc.

News

Practical

Services

RFI store

RFI in other languages

France Mdias Monde

Sorry but the period of time connection to the operation is exceeded.

Read this article:
In world-first breakthrough, French doctors use gene therapy to treat rare blood disease - RFI

Posted Feb. 28, 2017 at 3:39 p.m.

Published: 2017-02-28 15:39:55 Updated: 2017-02-28 15:39:55

By JIM SHAMP, NCBiotech Writer

Raleigh, N.C. The North Carolina Biotechnology Center has announced that Pfizer has committed to providing funding in the amount of $4 million which will enable the Center to establish and administer a multi-year academic fellowship program to help advance North Carolinas fast-growing expertise in gene therapy.

The new program, to be managed by NCBiotech, will support distinguished postdoctoral fellowships in North Carolina university research laboratories providing advanced scientific training in gene therapy-related research.

Absent or faulty proteins linked to genetic mutations cause numerous devastating diseases, making gene therapy an increasingly important treatment strategy.

Pfizers portfolio in North Carolina has grown in recent years. The company already operates a pharmaceutical manufacturing facility in the Lee County community of Sanford, and in August 2016, it acquired leading-edge gene therapy company Bamboo Therapeutics, Inc. in Chapel Hill.

With that acquisition, Pfizer gained the expertise of Bamboos world-renowned co-founder, R. Jude Samulski, Ph.D., director of the Gene Therapy Centerat the University of North Carolina at Chapel Hill. The deal also included an 11,000-square-foot facility for the highly specialized manufacturing of recombinant adeno-associated viral vectors.

Pfizer is one of several biopharmaceutical companies that have added high-profile gene therapy acquisitions, and several partnerships with biotechnology companies and leading academic institutions, to its R&D portfolio. Numerous other North Carolina scientists and companies are also making significant inroads into gene therapy, gene editing and related applications, many with NCBiotech support. For example, Samulski was recruited to UNC in 1993 as part of a $430,000 NCBiotech grant. Additionally, Bamboos former parent company received more than $700,000 in Biotech Center grants and loans.

Gene therapy advances require specific skills in addition to deep scientific knowledge. The fellowship program being established with Pfizers funding aims to boost that talent pipeline, with talent that has already proven to be exceptional in North Carolina. Such funding will enable NCBiotech to provide two-year fellowship support to postdoctoral scientists. The funding will afford the Center the ability to cover salaries, benefits, materials, professional development and travel for such postdoctoral scientists. The Center will encourage competitive applications from scientists interested in establishing research careers in gene therapy and related research activities.

The Biotech Center will also create and manage a related gene therapy Exchange Group. It will join some 25 other exchange groups designed to unite North Carolina-based academic and industry scientists with shared professional interests. The Gene Therapy EG will include these new postdoctoral fellows, their mentors, and others interested in the burgeoning gene therapy sector.

The field of gene therapy research has made tremendous strides in recent years, and we are pleased to be able to further enhance our leadership position in this area through this unique fellowship program, said Mikael Dolsten, M.D., Ph.D., president of worldwide research and development at Pfizer. We believe that gene therapy may hold the promise of bringing true disease modification for patients suffering from devastating diseases, and North Carolina is uniquely positioned to help us take advantage of collaborative opportunities that can develop the specialized talent well need.

Doug Edgeton, president and CEO of the Biotech Center, said he was deeply honored that Pfizer targeted North Carolina, and the Center, for the groundbreaking fellowship program.

Pfizer embraced the opportunity to work with us given weve proven for more than 30 years that we have the expertise and success metrics to maximize impact, said Edgeton. We not only have outstanding research institutions across our state, but we also have a well-respected culture of partnering and collaboration that allows us to be nimble and responsive. This is a wonderful example.

(C) N.C. Biotechnology Center

WRAL TechWire any time: Twitter, Facebook

Read more here:
Pfizer commits $4M to NC Biotechnology Center gene therapy ... - WRAL Tech Wire

Photo: Michael Cummo / Hearst Connecticut Media

Alliance for Cancer Gene Therapy (ACGT) CEO and President John Walter and Executive Director Margaret Cianci pose for a photo inside the ACGT offices in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy (ACGT) CEO and President John Walter and Executive Director Margaret Cianci pose for a photo inside the ACGT offices in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy (ACGT) CEO and President John Walter and Executive Director Margaret Cianci pose for a photo outside the ACGT offices in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy (ACGT) CEO and President John Walter and Executive Director Margaret Cianci pose for a photo outside the ACGT offices in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy CEO and President John Walter inside his office in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy CEO and President John Walter inside his office in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy Executive Director Margaret Cianci inside her office in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy Executive Director Margaret Cianci inside her office in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy (ACGT) CEO and President John Walter and Executive Director Margaret Cianci pose for a photo inside the ACGT offices in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy (ACGT) CEO and President John Walter and Executive Director Margaret Cianci pose for a photo inside the ACGT offices in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy (ACGT) CEO and President John Walter and Executive Director Margaret Cianci pose for a photo outside the ACGT offices in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy (ACGT) CEO and President John Walter and Executive Director Margaret Cianci pose for a photo outside the ACGT offices in Stamford, Conn. on Monday, Feb. 27, 2017.

Stamford-based ACGT sees success with support of cancer research

STAMFORD The Alliance for Cancer Gene Therapy recently announced two grants totaling $500,000. But its leaders are already filling the funding pipeline to support future recipients.

Describing itself as the only nonprofit in the country solely dedicated to funding and supporting research into cell and gene therapies for cancer, ACGT has built itself into a fundraising force since its founding in 2001. It has awarded 52 researcher grants totaling almost $27 million. Major events such as Swim Across Americas annual swim between Greenwich and Stamford and a gala held other every other year the next gala is set for April 19 at the Harvard Club in Manhattan are founts for the organizations giving.

We want to keep creating new interest in the organization, new donors and new dollars, ACGT President and CEO John Walter said in an interview Monday. For people who are interested in investing in cancer research, were a good place to turn to because of the success weve had.

Research backed by ACGT focuses on gene and cell mutations, a targeted approach that aims to reduce or eliminate debilitating side effects of traditional treatments.

The recipients of ACGTs latest Young Investigator Awards, the University of Calgarys Marco Gallo and the University of Pittsburghs Greg Delgoffe, each earned a $250,000 grant.

We play a very key role in helping young investigators, said Margaret Cianci, ACGTs executive director. They have wonderful ideas, and we want to help them fund that research and move into the next phase.

ACGT-supported research has produced four companies that are developing major new treatments, according to ACGT officials. They said they hope the first gene-therapy drug will launch in the U.S. later this year. One of the drugs that could soon enter the market is based on Car T cell research by an ACGT fellow, Dr. Carl June, who is an immunotherapy professor at the University of Pennsylvania.

Buoyed by recent advances, ACGT officials said that they want to keep supporting research of emerging treatments.

While you have these excellent immunotherapies, how can we combine them with some of the gene therapy going on? Cianci said. The goal is to be sure that the cancers dont come back.

All of ACGTs donations fund research. The nonprofits board covers administrative expenses for the nonprofits staff of four.

Headquartered on Cummings Point Road in the citys Waterside section, ACGT has made several leadership changes recently. Walter and Barbara Gallagher, national director of philanthropy, both arrived last year. Walter succeeded Barbara Netter, who co-founded ACGT with her late husband, Edward Netter.

Barbara Netter, a Greenwich resident, maintains an important role in the organization as honorary chairman. She said that the organizations work is fulfilling Edward Netters vision.

With the promise of new drugs coming on the market, it should really make a statement about gene therapy, she said. Im very optimistic.

pschott@scni.com; 203-964-2236; twitter: @paulschott

Visit link:
Stamford-based ACGT sees success with support of cancer research - The Advocate

A French teen who was given gene therapy for sickle cell disease more than two years ago now has enough properly working red blood cells to dodge the effects of the disorder, researchers report.

The first-in-the-world case is detailed in Thursdays New England Journal of Medicine.

About 90,000 people in the U.S., mostly blacks, have sickle cell, the first disease for which a molecular cause was found. Worldwide, about 275,000 babies are born with it each year.

Vexing questions of race and stigma have shadowed the history of its medical treatment, including a time when blacks who carry the bad gene were urged not to have children, spurring accusations of genocide, Keith Wailoo of Princeton University wrote in a separate article in the journal.

The disease is caused by a single typo in the DNA alphabet of the gene for hemoglobin, the stuff in red blood cells that carries oxygen. When its defective, the cells sickle into a crescent shape, clogging tiny blood vessels and causing bouts of extreme pain and sometimes more serious problems such as strokes and organ damage. It keeps many people from playing sports and enjoying other activities of normal life.

A stem cell transplant from a blood-matched sibling is a potential cure, but in the U.S., fewer than one in five people have a donor like that. Pain crises are treated with blood transfusions and drugs, but theyre a temporary fix. Gene therapy offers hope of a lasting one.

The boy, now 15, was treated at Necker Childrens Hospital in Paris in October 2014. Researchers gave him a gene, taken up by his blood stem cells, to help prevent the sickling. Now, about half of his red blood cells have normal hemoglobin; he has not needed a transfusion since three months after his treatment and is off all medicines.

Its not a cure but it doesnt matter, because the disease is effectively dodged, said Philippe Leboulch, who helped invent the therapy and helped found Bluebird Bio in Cambridge, Massachusetts, the company that treated the boy. The work was supported by a grant from the French governments research agency.

Bluebird has treated at least six others in the U.S. and France. Full results have not been reported, but the gene therapy has not taken hold as well in some of them as it did in the French teen. Researchers think they know why and are adjusting methods to try to do better.

Two other gene therapy studies for sickle cell are underway in the U.S. at the University of California, Los Angeles and Cincinnati Childrens Hospital and another is about to start at Harvard and Boston Childrens Hospital using a little different approach.

This work gives considerable promise for a solution to a very common problem, said Dr. Stuart Orkin, a Boston Childrens Hospital doctor who is an inventor on a patent related to gene editing.

The results are quite good in this patient, he said of the French teen. It shows gene therapy is on the right track.

___

Online:

Gene therapy: http://ghr.nlm.nih.gov/primer/therapy/availability

___

Marilynn Marchione can be followed at http://twitter.com/MMarchioneAP

See more here:
Gene therapy lets a French teen dodge sickle cell disease - The Seattle Times

An experimental gene therapy that turns a patient's own blood cells into cancer killers worked in a major study, with more than one-third of very sick lymphoma patients showing no sign of disease six months after a single treatment, its maker said Tuesday.

In all, 82 percent of patients had their cancer shrink at least by half at some point in the study.

Its sponsor, California-based Kite Pharma, is racing Novartis AG to become the first to win approval of the treatment, called CAR-T cell therapy, in the U.S. It could become the nation's first approved gene therapy.

A hopeful sign: the number in complete remission at six months 36 percent is barely changed from partial results released after three months, suggesting this one-time treatment might give lasting benefits for those who do respond well.

"This seems extraordinary ... extremely encouraging," said one independent expert, Dr. Roy Herbst, cancer medicines chief at the Yale Cancer Center.

The worry has been how long Kite's treatment would last and its side effects, which he said seem manageable in the study. Follow-up beyond six months is still needed to see if the benefit wanes, Herbst said, but added, "this certainly is something I would want to have available."

The therapy is not without risk. Three of the 101 patients in the study died of causes unrelated to worsening of their cancer, and two of those deaths were deemed due to the treatment.

It was developed at the government's National Cancer Institute and then licensed to Kite. The Leukemia and Lymphoma Society helped sponsor the study.

Results were released by the company and have not been published or reviewed by other experts. Full results will be presented at the American Association for Cancer Research conference in April.

The company plans to seek approval from the U.S. Food and Drug Administration by the end of March and in Europe later this year.

The treatment involves filtering a patient's blood to remove key immune system soldiers called T-cells, altering them in the lab to contain a gene that targets cancer, and giving them back intravenously. Doctors call it a "living drug" permanently altered cells that multiply in the body into an army to fight the disease.

Patients in the study had one of three types of non-Hodgkin lymphoma, a blood cancer, and had failed all other treatments. Median survival for such patients has been about six months.

Kite study patients seem to be living longer, but median survival isn't yet known. With nearly nine months of follow-up, more than half are still alive.

Six months after treatment, 41 percent still had a partial response (cancer shrunk at least in half) and 36 percent were in complete remission (no sign of disease).

"The numbers are fantastic," said Dr. Fred Locke, a blood cancer expert at Moffitt Cancer Center in Tampa who co-led the study and has been a paid adviser to Kite. "These are heavily treated patients who have no other options."

One of his patients, 43-year-old Dimas Padilla of Orlando, was driving when he got a call saying his cancer was worsening, chemotherapy was no longer working, and there was no match to enable a second try at a stem cell transplant.

"I actually needed to park ... I was thinking how am I going to tell this to my mother, my wife, my children," he said. But after CAR-T therapy last August, he saw his tumors "shrink like ice cubes" and is now in complete remission.

"They were able to save my life," Padilla said.

Of the study participants, 13 percent developed a dangerous condition where the immune system overreacts in fighting the cancer, but that rate is lower than in some other tests of CAR-T therapy. The rate fell during the study as doctors got better at detecting and treating it sooner.

Roughly a third of patients developed anemia or other blood-count-related problems, which Locke said were easily treated. And 28 percent had neurological problems such as sleepiness, confusion, tremor or difficulty speaking, but these typically lasted just a few days, Locke said.

"It's a safe treatment, certainly a lot safer than having progressive lymphoma," and comparable to combination chemotherapy in terms of side effects, said the cancer institute's Dr. Steven Rosenberg, who had no role in Kite's study. The first lymphoma patient Rosenberg treated this way, a Florida man, is still in remission seven years later.

There were no cases of swelling and fluid in the brain in this or any other study testing Kite's treatment, company officials said. That contrasts with Juno Therapeutics, which has had a CAR-T study put on hold twice after five patient deaths due to this problem.

Company officials would not say what the treatment might cost, but other types of immune system therapies have been very expensive. It's also being tested for some other types of blood cancer.

More here:
Gene therapy to fight a blood cancer succeeds in major study - Fox News

Theres a rare disorder that occurs when a gene mutation halts the production of myotubularina protein that facilitates normal muscle function. The disease, called myotubular myopathy (MTM), only affects males, and its ultimately fatal because it causes breathing difficulties.

Dogs get MTM, tooand that spelled opportunity for scientists at the University of Washington Medicine Institute for Stem Cell and Regenerative Medicine. In collaboration with five other academic institutions, they found a way to replace the faulty MTM gene with a functioning gene in dogs with the disease, they reported in the journal Molecular Therapy.

It worked: After a single infusion of genes, muscle strength was restored in the dogs, according to a press release. One year later, the dogs were indistinguishable from healthy animals, they said. "This regenerative technology allowed dogs that otherwise would have perished to complete restoration of normal health," said Dr. Martin K. "Casey" Childers, UW Medicine researcher and physician.

The researchers used a viral vector called adeno-associated virus serotype 8 (rAAV8) to deliver a healthy canine version of the MTM gene in dogs that were 10 weeks old and already showing symptoms. They believe a similar trial could be designed in people.

Gene therapy is under investigation for a wide range of disorders, though much of the progress to date has occurred outside the realm of muscular disorders. BioMarin Pharmaceutical, for example, is in mid-stage trials of a gene therapy treatment for hemophilia A. UniQure is working on several gene therapy products to treat diseases including Huntingtons and congestive heart failure. Its most advanced project, a gene therapy product to treat hemophilia B, received breakthrough designation status from the FDA in January.

One company that has achieved some success with gene therapy in inherited muscle disorders is AveXis, which is gearing up for a pivotal trial of its treatment for spinal muscular atrophy. AveXis won breakthrough therapy designation for its gene product last year, and high hopes for the product have prompted its stock to more than triple since the company went public early last year.

UW Medicine-led team that worked on the canine MTM trial observed that as they increased the dosage of genes, survival rates improved, they reported. They believe the study proves the potential utility of gene therapy in a wide range of diseases that are linked to mutated genes.

See more here:
Gene therapy tried in dogs with muscle disease could prove useful ... - FierceBiotech

In the past two decades, gene-based clinical trials have increased by almost 500%. The global market for regenerative medicines including cell therapies, gene/gene-modified cell therapy and tissue engineering is poised to reach $67 billion in 2020.

While the majority of cell and gene therapies are still experimental aimed at rare single-gene disorders researchers hope to build on their successes to develop treatments for multi-gene disorders, including heart disease, hypertension, diabetes, arthritis and Alzheimers disease.

That makes the success of clinical trials now underway vitally important. To make those treatments possible, another industry has to evolve: logistics.

Therapies that contain live materials are produced and packaged under strictly controlled conditions. They must be transported around the world and delivered on time, at optimum temperature, so they arrive in perfect condition.

Researchers dealing with cell and gene therapies already face many obstacles given the novelty and unpredictability of the science, the small size of patient pools and a typical single-dosing model, said Sam Herbert, Chief Operating Officer at World Courier.

In delivering cell and gene therapies to the patient whether during a trial or as part of a treatment plan it is critical that the therapies are delivered on time and in pristine condition.

For clinical trial results to be accurate and replicable, biological samples must arrive at investigator sites in the same condition that they left the lab.Compliance in the lab, the production facility, and the hospital or treatment site is fairly easy to assure. Compliance in the outside world, where the unpredictable happens every day, is not as easy.

Even small cell and gene therapy trials are immensely complex, with many moving parts and no room for error.In this research environment, not only the science, but the entire process from start to finish must be flawless.Researchers, sponsors, clinical teams and supply chain providers supporting the project can settle for nothing less than perfection.

Cell and gene therapies are produced one-by-one under strictly controlled conditions, using live bio-materials. Time and temperature variation could destroy them, so they must arrive at the clinical site on time and in pristine condition.

In theory, thats a tough job.

In the real world, its much harder.

Weather, air traffic, road conditions, climate zones, customs regulations and processing times cant be allowed to delay delivery, or affect the temperature-controlled packaging of the samples and therapeutic materials.

To ensure success, World Courier starts working with study sponsors years in advance.

Our dedicated project team plans a personalized supply chain, develops customized operating procedures, trains all personnel who come in contact with the shipments from lab to clinic, and works with airline personnel and international customs agents to make sure everyone knows what is at stake and how to handle the shipments.

World Courier planned and executed all logistics for a developer of immuno-oncology products during a Phase II clinical trial.Under evaluation was an autologous, dendritic cell-based therapy for cancer patients, to extend remission time and possibly overall survival.The therapy was granted an orphan drug designation by the FDA and EMA, and received fast track designation by the FDA after the Phase II trial.

The therapy owners manufacturing sites were in Europe and in Australia. Shipments of starting cell material had 24 hours to travel from the clinical sites in Europe and Asia to the central manufacturing sites. Over a period of 20 months, World Courier delivered 245 shipments containing more than 2,000 kilograms of materials to treat 63 patients.Therapeutic materials travelled by air freight and road, and were hand carried to their final destination.

Our clinical sample delivery success rate was 100%.

The best logistics partner for your clinical trial is one who has been down that road. Download World Couriers e-book Tomorrows Medicine: Curing One Patient at a Time to find out more.

See the original post here:
Global logistics strategies for cell and gene therapies - BioPharma Dive

Philadelphia Business Journal

Why Spark's CEO says 2017 may be a 'historic year' for his gene therapy company Philadelphia Business Journal We look forward to continued innovation, execution and growth in 2017, which may be a historic year for Spark Therapeutics and for patients as we potentially deliver what has been unimaginable before the first gene therapy in the United States for a ... Spark Therapeutics Reports 2016 Financial Results and Business HighlightsGlobeNewswire (press release) all 3 news articles »

Go here to see the original:
Why Spark's CEO says 2017 may be a 'historic year' for his gene therapy company - Philadelphia Business Journal

For decades, some unluckydog lovers have welcomeda bundle of barking joy into their homes, only to see them perish from a mysterious disease mere weeks after their birth. The pups seemingly healthy muscles had literally wasted away in front of their owners eyes until they could no longer stand and breathe.

It wasnt until 2010 that a French research team isolated the genetic cause of this specific muscle-wasting disease in a group of Labrador Retrievers; these dogs were suffering from a single mutation that left them unable to produce an essential protein known asmyotubularin.Whats more, it was the exact kind of mutation and disease also long found in male human babies, too. That made the researchers wonder if these unfortunate puppiescould help us study the disease and even someday find a way to saveboth pets and people.

Now, years down the road, it appearsthey were right, thanks to a cutting-edgegene therapy treatment.

An international group of researchers, including some from the original French team, gathered together 10-week-old puppies with the mutation to take part in a randomized controlled trial. The dogs who were given a treatment that repaired their defectivemyotubularingene avoided the crippling muscle degeneration that killed the placebo-treated dogs by week 17. And by the ninth month of study, the saved puppies muscle and neurological function continued to match readings from healthy dogs, particularly forthose that got the highest doses.

The findings, building on an earlier proof-of-concept study of dogs and mice by the researchers, signal that a scaled-up treatment could save the lives of boys with the same sort of genetic flaw.

I believe that the dog study will be about as close as we will ever get to a human study, senior author Dr. Martin Childers of the University of Washington told Vocativ in an email. Because we found evidence that the gene therapy product spread throughout the entire skeletal musculature of adult dogs after a single infusion, it seems reasonable to expect a similar result in human patients.

Gene therapy has received plenty of attention for its potential to treat otherwise irreparable DNA defects, but according to the researchers, theres been little focus on bone- and muscle-relatedgenetic disorders. The condition treated in the current study, called x-linked myotubular myopathy, affects around one in every 50,000 boys, with most sufferers living no more than a few years. And though theres no true tally of how often it affects dogs, case reports of similar-sounding diseases have been published stretching back decades.

There will undoubtedly be hurdles to climb before the treatment Childers and his team developed, or a similar one, can be tested in people, Childers said. It is always possible that humans might respond differently, thus, clinical trials will be conducted with extraordinary care and oversight, he explained. And though the dogs suffered little adverse effects from the therapy delivered via a harmless virus researchers will still have to watch out for any possible toxicity in people.

That said, the treatment offers hope for both man and mutts. The changes seen after a single treatment have lasted for several years in the small sample of dogs the team has raised. So its possible that people wont need repeated doses or they would be infrequent, Childers said a big positive, given how expensive gene therapy is today.

And its also likely that these treatments, within the larger field of regenerative medicine, will find a place for dogs and other animals sooner than it will for people.

Veterinary medicine is ahead of human medicine in some cases with respect to regenerative technologies, Childers said. Stem cell infusions, for example, have been given to pets and horses for more than a decade.

But people may not have to wait so long for the promise of gene therapy either. Childers is hopeful that Audentes Therapeutics, a San Francisco biomedical company hes collaborating with (and which partially funded the current study), will begin their first human trials of a gene therapy treatment for x-linked myotubular myopathy, based on his teams research, later this year.

The teams findings were published earlier this February in Molecular Therapy.

More:
Gene Therapy Saves Puppies From A Fatal DiseaseAnd Maybe Us Next - Vocativ

Gene therapy technologies are described in detail including viral vectors, nonviral vectors and cell therapy with genetically modified vectors. Gene therapy is an excellent method of drug delivery and various routes of administration as well as targeted gene therapy are described. There is an introduction to technologies for gene suppression as well as molecular diagnostics to detect and monitor gene expression.

Clinical applications of gene therapy are extensive and cover most systems and their disorders. Full chapters are devoted to genetic syndromes, cancer, cardiovascular diseases, neurological disorders and viral infections with emphasis on AIDS. Applications of gene therapy in veterinary medicine, particularly for treating cats and dogs, are included.

Research and development is in progress in both the academic and the industrial sectors. The National Institutes of Health (NIH) of the US is playing an important part. As of 2015, over 2050 clinical trials have been completed, are ongoing or have been approved worldwide.A breakdown of these trials is shown according to the geographical areas and applications.

The markets for gene therapy are difficult to estimate as there is only one approved gene therapy product and it is marketed in China since 2004. Gene therapy markets are estimated for the years 2016-2026. The estimates are based on epidemiology of diseases to be treated with gene therapy, the portion of those who will be eligible for these treatments, competing technologies and the technical developments anticipated in the next decades. In spite of some setbacks, the future for gene therapy is bright.The markets for DNA vaccines are calculated separately as only genetically modified vaccines and those using viral vectors are included in the gene therapy markets

Profiles of 188 companies involved in developing gene therapy are presented along with 233 collaborations. There were only 44 companies involved in this area in 1995. In spite of some failures and mergers, the number of companies has increased more than 4-fold within a decade. These companies have been followed up since they were the topic of a book on gene therapy companies by the author of this report.

Key Topics Covered:

Part I: Technologies & Markets

1. Introduction

2. Gene Therapy Technologies

3. Clinical Applications of Gene Therapy

4. Gene Therapy of Genetic Disorders

5. Gene Therapy of Cancer

6. Gene Therapy of Neurological Disorders

7. Gene Therapy of Cardiovascular Disorders

8. Gene therapy of viral infections

9. Research, Development and Future of Gene Therapy

10. Regulatory, Safety and Ethical Issues of Gene Therapy

11. Markets for Gene Therapy

12. References

Part II: Companies

13. Companies involved in Gene Therapy

For more information about this report visit http://www.researchandmarkets.com/research/jtwqds/gene_therapy

Media Contact:

Laura Wood, Senior Manager press@researchandmarkets.com

For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900

U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/global-gene-therapy-technologies-markets-and-companies-2016-2026---research-and-markets-300407996.html

SOURCE Research and Markets

http://www.researchandmarkets.com

View original post here:
Global Gene Therapy Technologies, Markets and Companies 2016-2026 - Research and Markets - PR Newswire (press release)

By: PTI | Updated: February 20, 2017 6:53 pm Not only did mRNA technique make the mouse glow, it also later ran around, completely unaware of the complex series of events that had just taken place within its body, researchers said. ( Image for representation, Source: Youtube)

Stanford scientists have successfully developed glow-in-the-dark mice using compounds that create proteins responsible for lighting up fireflies, an advance that may pave the way for new gene therapies.

Timothy Blake, a postdoctoral fellow at Stanford University in the US refined compounds that carry instructions for assembling the protein that makes fireflies light up and delivered them into the cells of an anaesthetised mouse.

Watch all our videos from Express Technology

Not only did the technique make the mouse glow, it also later woke up and ran around, completely unaware of the complex series of events that had just taken place within its body, researchers said.

This success could mark a significant step forward for gene therapy. It is hard enough getting these protein instructions, called messenger RNA (mRNA), physically into a cell. It is another hurdle altogether for the cell to actually use them to make a protein. If the technique works in people, it could provide a new way of inserting therapeutic proteins into diseased cells.

Its almost a childlike enthusiasm we have for this, said Robert Waymouth, a professor at Stanford. The code for an insect protein is put into an animal and that protein is not only synthesised in the cells but its folded and it becomes fully functional, capable of emitting light, said Waymouth. Although the results are impressive, this technique is remarkably simple and fast. Unlike traditional gene therapy that permanently alters the genetic makeup of the cell, mRNA is short-lived and its effects are temporary.

The transient nature of mRNA transmission opens up special opportunities, such as using these compounds for vaccination or cancer immunotherapy. Gene therapy is a decades-old field of research that usually focuses on modifying DNA, the fundamental genetic code. That modified DNA then produces a modified mRNA, which directs the creation of a modified protein.

Also Read:Gene-editing cell therapy saves two babies from cancer

The current work skips the DNA and instead just delivers the proteins instructions. They used a novel, deceptively straightforward creation, called charge-altering releasable transporters (CARTs). What distinguishes this polycation approach from the others, which often fail, is the others dont change from polycations to anything else, said Paul Wender, professor at Stanford.

Whereas, the ones that were working with will change from polycations to neutral small molecules. That mechanism is really unprecedented, Wender said. As part of their change from polycations to polyneutrals, CARTs biodegrade and are eventually excreted from the body.

One application of this technology is vaccination. At present, vaccines require introducing part of a virus or an inactive virus into the body in order to elicit an immune response. CARTs could potentially cut out the middleman, directly instructing the body to produce its own antigens.

Read this article:
Stanford scientists create glow-in-the-dark mice, may advance gene therapies - The Indian Express

CTVNews.ca Staff Published Friday, February 17, 2017 10:00PM EST Last Updated Friday, February 17, 2017 10:28PM EST

A small gene therapy trial involving several Canadian patients is offering new hope to people living with hemophilia, a rare and potentially fatal genetic disorder.

Hemophilia patients can suffer prolonged or uncontrollable bleeding, even after minor injuries. That's because they lack blood clotting factors, or proteins.

There are two types of hemophilia, A and B, and both are very rare disorders. Hemophilia A affects an estimated 2,500 Canadians, while hemophilia B affects about 600 Canadians, according to the Canadian Hemophilia Society.

A new gene therapy developed at the Children's Hospital in Philadelphia has produced very encouraging results. Preliminary research suggests that a single dose of the experimental therapy may help patients with hemophilia B, which involves a deficiency of blood clotting factor IX.

The therapy involves using a gene engineered to replace the faulty one in people with hemophilia. The engineered gene is placed into an inactivated virus and then infused into the liver, where it helps the body produce a clotting factor that prevents bleeding.

"It poses the possibility for a one-time treatment that would bepotentially life-altering for the patient," Dr. Lindsey George, a hematologist at The Children's Hospital of Philadelphia, told CTV News.

Scientists found that after just one dose, none of the nine patients involved in the trial suffered any bleeding for up to a year. Although more research and larger studies are needed to confirm the benefits of the therapy, researchers are very encouraged.

"It is very exciting, certainly from my standpoint as a clinical investigator," Dr. George said.

Dr. Jerry Teitel, the medical director of the Hemophilia Treatment Centre at St. Michael's Hospital in Toronto, who collaborated with the Philadelphia researchers, called it "a revolutionary therapy."

The results so far are wonderful, in fact even better than what we had dared to hope," he said.

Of the nine patients who've received the treatment, four are Canadian.

Among them is John Konduros, a 52-year-old bakery owner in Cambridge, Ont. As a lifelong hemophiliac, he has always lived in fear of any bumps, cuts or bruises that could cause internal bleeding, disability -- and even death.

"It has probably affected every single part of my life, from being a kid to now," he told CTV News. "If you ever saw me as a kid, I was never in a group. I was always on the sides."

Konduros said it was common for him to miss two or three weeks of school if another kid happened to kick him in the leg while they were playing. In one class photo, he is seen with a big bruise under his right eye - another side effect of his condition.

But since Konduros received the experimental treatment about eight months ago, he has not had any dangerous bleeds.

"I'm extremely happy in the sense of massive relief. I feel like I don't have to be as vigilant or worrisome about everything and anything that's going on around me," he said.

So far, the immune systems of two trial patients have reacted to the treatment, but scientists say there were no serious side effects. Konduros has had no problems with the therapy.

"If the doctors wanted me to go down every weekend for more tests to accelerate anything I would say 'sure' because the improvement it gives anyone who has hemophilia is huge," he said.

Dr. Teitel said scientists have "a long way to go" in developing a therapy that can help more hemophilia patients.

"We need to show that in large numbers, the results do hold up," he said. "We need to show that the results last for a long period of time, not necessarily last a lifetime."

With files from CTV's medical specialist Avis Favaro and producer Elizabeth St. Philip

Link:
'Exciting' new therapy shows promising results for hemophilia ... - CTV News

Sessions and Tracks

Cancer is a class of diseases characterized by out-of-control cell growth. There are more than 100 distinct sorts of cancer and each is ordered by the kind of cell which is first influenced. Malignancy is thought to be one of the main sources of grimness and mortality around the world. More than 575,000 individuals bite the dust of tumor and more than 1.5 million individuals are determined to have disease every year in the US. A restorative expert who hones in the field of cancer and cancer related diseases is an oncologist.

In addition to the multidisciplinary talks, keynote sessions and lectures relevant to cancer science & therapy, the Cancer therapy 2017 is a complete 3 days event with panel discussions, open Q & A to generate a prime learning knowledge between participants.

Scientific Session of the Conference includes:

Cancer Cell Biology

Cancer Metastasis

Cancer Genetics

Tumor & Cancer Immunology

Cancer : Genomics & Metabolomics

Targeted Cancer Therapy

Stem Cell Therapy

Cancer Biomarkers

Cancer Case Reports

Novel Approaches to Cancer Therapeutics

Precision Medicine & Cancer Therapy

Cancer Management & Prevention

Cancer Pharmacology

Organ Specific Cancers

Radiation Oncology

Surgical Oncology

Cancer Drugs

Complementary and Alternative Cancer Treatment

Cancer Clinical Trials

Cancer & Lifestyle

Cancer: Psychological & Social Aspects

Cancer Diagnostics & Diagnostic Market

1. Cancer Cell Biology

Cancerous tumors are threatening, which implies they can spread into, or attack adjacent tissues. What is more, as these tumors develop, some cancer cells can sever and go to distant places in the body through the blood or the lymph framework and shape new tumors a long way from the first tumor. Cancer cells emerge from the body's own particular tissues. Cancer Cell Biology incorporates the molecular, biochemical and cell-based ways to deal with better comprehend cancer pathogenesis.

2. Cancer Metastasis

Metastasis is the spread of a cancer or other infection from one organ or part of the body to another without being straightforwardly associated with it. At the point when cancer cells split far from a tumor, they can go to different territories of the body through the circulatory system or the lymph framework. The lungs, liver, brain and bones are the most well-known metastasis areas from solid tumors. Treatment and survival is resolved, by regardless of whether a cancer stays confined or spreads to different areas in the body.

3. Cancer Genetics

Cancer is a hereditary sickness and is brought about by specific changes to qualities that control the way our cells work, particularly how they develop and separate. These progressions incorporate transformations in the DNA that makes up our qualities. A few sorts of cancers keep running in specific families, yet most tumors are not unmistakably connected to the qualities we acquire from our folks. Quality changes that begin in a solitary cell throughout a man's life cause generally malignancies. A few people are hereditarily inclined to building up specific sorts of cancers. These individuals have a higher danger of building up the malady than those in the overall population. Hereditary testing is currently accessible for some inherited cancers. Genetic testing includes a straightforward blood test and might be utilized to get a more exact gauge of your growth hazard. Now and again, Genetic testing should be possible on put away tissue tests from deceased relatives.

4. Tumor & Cancer Immunology

Tumor immunology depicts the cooperation between cells of the invulnerable framework with tumor cells. Understanding these interactions is imperative for the improvement of new treatments for tumor treatment. In many people the resistant framework perceives and disposes of Tumor cells. Cancer immunology is a branch of immunology that reviews collaborations between the resistant framework and cancer cells (likewise called tumors or malignancies). It is a field of research that plans to find cancer immunotherapies to treat and retard movement of the disease. The immune response, including the recognition of cancer-specific antigens, forms the basis of targeted therapy, (such as vaccines and antibody therapies) and tumor marker-based diagnostic tests.

5. Cancer genomics & metabolomics

Cancer genomics is the study of the totality of DNA sequence and gene expression differences between tumor cells and normal host cells. It aims to understand the genetic basis of tumor cell proliferation and the evolution of the cancer genome under mutation and selection by the body environment, the immune system and therapeutic interventions. The metabolites within a cell or biological system are being used to analyze cancer metabolism on a system-wide scale, painting a broad picture of the altered pathways and their interactions with each other. Cancer metabolomics involves chemical analysis by a range of analytical platforms through targeted/untargeted approaches. The application of metabolomics towards cancer research has led to a renewed appreciation of metabolism in cancer development and progression.

Tumor cell proliferation

Genomic Studies

Cancer Genome Atlas (TCGA)

Genomics Tools

Metabolic Technologies

Data Interpretations

Metabolomics as Biomarker

6. Targeted Cancer Therapy

Targeted Cancer therapy is a newer type of cancer treatment that uses drugs or other substances to more precisely identify and attack cancer cells, usually while doing little damage to normal cells. Targeted therapy is a growing part of many cancer treatment regimens. Targeted therapy or molecularly targeted therapy is one of the major modalities of medical treatment (pharmacotherapy) for cancer. The Drugs work by targeting specific genes or proteins. These genes and proteins are found in cancer cells or in cells related to cancer growth, like blood vessel cells. As a form of molecular medicine, targeted therapy blocks the growth of cancer cells by interfering with specific targeted molecules needed for carcinogenesis and tumor growth, rather than by simply interfering with all rapidly dividing cells.

Therapeutic Monoclonal Antibodies

Small Molecule Drugs

Tyrosine-kinase inhibitors

Implications of Targeted Therapy

Targeted Cancer Therapy & Health Economics

Hormone Therapies

7. Stem Cell Therapy

Stem Cells and Tumors Cancer Cells also have the characteristic that is associated with normal stems cells. Stem Cell Therapy is used to prevent the disease. The most common stem cells therapy is bone marrow transplantation. Stems cells transplant is used to treat cancers like leukemia, multiple myeloma of lymphoma. Cord Blood Stem and Cancer cord blood contains hematopoietic (blood) stem cell. These cells make different types of cells like red blood cells, white blood cells, Hematopoietic stem cells, purified from bone marrow or blood, have long been used in stem cell treatments for leukemia, blood and bone marrow disorders when chemotherapy is used.

Cancer Stem Cells

Stem Cells and Tumors

Stem Cell Transplantation

Bone Marrow Transplantation

Cord Blood Stem Cells and Cancer

Stem Cell Research

8. Cancer Biomarkers

A cancer biomarker is an element or procedure that indicates the presence of cancer in the body. A biomarker may be any molecule released by the presence of a tumor or a specific indication of the body to the presence of cancer. Cancer biomarkers are usually biological molecules found in blood, other body fluids, or tissues that are a sign of a normal or abnormal process, or of a condition or disease.

Imaging Biomarkers

Clinical Biomarkers

Genetic Biomarkers

Predictive Cancer Biomarkers

Molecular Biomarkers

Cell Free Biomarkers

9. Cancer Case Reports

A case report signifies the detailed report of symptoms, signs, diagnosis, treatment and follow-up of an individual patient of a particular disease. Cancer Case reports have been playing a pivotal role in medical education, providing a structure for case-based learning and implementation throughout the world.

Unexpected/Unusual Conditions

Rare Surgical Condition of a cancer case

Novel Surgical Procedure

Adverse Effects

Innovative in Cancer Surgery

10. Novel Approaches to Cancer Therapeutics

The Normal treatment modalities are associated with severe side effects and high toxicity which in turn lead to low quality of life. This review encompasses novel strategies for more effective chemotherapeutic delivery aiming to generate better prognosis. Currently, cancer treatment is a highly dynamic field and significant advances are being made in the development of novel cancer treatment strategies. In contrast to conventional cancer therapeutics, novel approaches such as ligand or receptor based targeting, intracellular drug targeting, gene delivery, cancer stem cell therapy, magnetic drug targeting and ultrasound-mediated drug delivery, have added new modalities for cancer treatment.

Cancer Epigenetics

Molecular Profiling Techniques

New Biologics & Vaccines

Chemical Proteomics

Combination Strategies in Immuno-oncology

Novel Biomarker Discovery

11. Precision Medicine & Cancer Therapy

Precision medicine also known as Personalized Medicine is a phrase that is often used to describe how genetic information about a persons disease is being used to diagnose or treat their disease. The deeper understanding of how cancer forms and grows has ushered in a new era of precision cancer care, where tailored treatments target abnormalities that may be found in each tumors DNA profile. This exciting innovation marks a shift, from traditional treatments designed for the average patient based on their success with a representative sample of people with similar cancers, towards more precise therapies.

Genomics Mutations

Molecular Diagnostics

Non-Genetic Characteristics

Targeted Drug Therapies

Clinical Trials of Personalized Medicine

12. Cancer Management & Prevention

Cancers that are closely linked to certain behaviors are the easiest to prevent. Many complementary health approaches are also found to combat the risks of cancers like, for example, herbal and other dietary supplements, acupuncture, massage and yoga.

Lifestyle changes

Diet & Cancer

Vaccinations

Natural Therapy

Psychological & Social Aspects

13. Cancer Pharmacology

Cancer pharmacology plays a key role in drug development. In both the laboratory and the clinic, cancer pharmacology has had to adapt to the changing face of drug development by establishing experimental models and target orientated approaches.

Tumor Targeting Strategies

Hormonal & Biological Agents

Continue reading here:
Cancer Therapy Conference | October 2017 | Baltimore | USA

Work on gene therapy is showing significant progress for restoring muscle strength and prolonging lives in dogs with a previously incurable, inherited neuromuscular disease. UW Medicine Institute for Stem Cell and Regenerative Medicine scientists are leading the multi-institutional research effort.

The disease arises from a mutation in genes that normally make a protein, called myotubularin, essential for proper muscle function. Puppies with this naturally occurring mutation exhibit several features of babies with the same defective gene. The rare disorder, called myotubular myopathy, or MTM, affects only males. It causes fatal muscle wasting. Both dogs and boys with the disease typically succumb in early life due to breathing difficulties.

For decades, researchers have struggled to find suitable treatments for genetic muscle diseases like this one. Four collaborating research groups in the United States and France found a way to safely replace the disease-causing MTM gene with a healthy gene throughout the entire musculature of affected dogs.

Their most recent findings were published online this week in Molecular Therapy.

Their paper reports that diseased dogs treated with a single infusion of the corrective therapy were indistinguishable from normal animals one year later.

"This regenerative technology allowed dogs that otherwise would have perished to complete restoration of normal health," said Dr. Martin K. "Casey" Childers, UW Medicine researcher and physician. Childers is a professor of rehabilitation medicine at the University of Washington School of Medicine and co-director of the Institute for Stem Cell and Regenerative Medicine.

Gene therapy holds the promise to treat many inherited diseases. To date, this approach has not been widely translated into treatment of skeletal muscle disorders.

"We report here a gene therapy dose-finding study in a large animal model of a severe muscle disease where a single treatment resulted in dramatic rescue," said Childers. The findings demonstrate potential application across a wide range of diseases and broadly translate to human studies. The data supports the development of gene therapy clinical trials for myotubular myopathy, the researchers concluded.

UW Medicine researchers David Mack, Melissa Goddard, Jessica Snyder, Matthew Elverman, and Valerie Kelly co-authored the report, "Systemic AAV8-mediated gene therapy drives whole-body correction of myotubular myopathy in dogs." This study was conducted in collaboration with Harvard University, Medical College of Wisconsin, Virginia Tech, INSERM, and Genethon.

Story Source:

Materials provided by University of Washington Health Sciences/UW Medicine. Original written by Barbara Rodriguez. Note: Content may be edited for style and length.

Read this article:
Gene therapy treats muscle-wasting disease in dogs: Single infusion ... - Science Daily

Timothy Blake, a postdoctoral fellow in the Waymouth lab, was hard at work on a fantastical interdisciplinary experiment. He and his fellow researchers were refining compounds that would carry instructions for assembling the protein that makes fireflies light up and deliver them into the cells of an anesthetized mouse. If their technique worked, the mouse would glow in the dark.

Not only did the mouse glow, but it also later woke up and ran around, completely unaware of the complex series of events that had just taken place within its body. Blake said it was the most exciting day of his life.

This success, the topic of a recent paper in Proceedings of the National Academy of Sciences, could mark a significant step forward for gene therapy. It's hard enough getting these protein instructions, called messenger RNA (mRNA), physically into a cell. It's another hurdle altogether for the cell to actually use them to make a protein. If the technique works in people, it could provide a new way of inserting therapeutic proteins into diseased cells.

"It's almost a childlike enthusiasm we have for this," said chemistry Professor Robert Waymouth. "The code for an insect protein is put into an animal and that protein is not only synthesized in the cells but it's folded and it becomes fully functional, capable of emitting light."

Although the results are impressive, this technique is remarkably simple and fast. And unlike traditional gene therapy that permanently alters the genetic makeup of the cell, mRNA is short-lived and its effects are temporary. The transient nature of mRNA transmission opens up special opportunities, such as using these compounds for vaccination or cancer immunotherapy.

Making a protein

Gene therapy is a decades-old field of research that usually focuses on modifying DNA, the fundamental genetic code. That modified DNA then produces a modified mRNA, which directs the creation of a modified protein. The current work skips the DNA and instead just delivers the protein's instructions.

Previous work has been successful at delivering a different form of RNA -- called short interfering RNA, or siRNA -- but sending mRNA through a cell membrane is a much bigger problem. While both siRNA and mRNA have many negative charges -- so-called polyanions -- mRNA is considerably more negatively charged, and therefore more difficult to sneak through the positively charged cell membrane.

What the researchers needed was a positively charged delivery method -- a polycation -- to complex, protect and shuttle the polyanions. However, this alone would only assure that the mRNA made it through the cell membrane. Once inside, the mRNA needed to detach from the transporter compound in order to make proteins.

The researchers addressed this twofold challenge with a novel, deceptively straightforward creation, which they call charge-altering releasable transporters (CARTs).

"What distinguishes this polycation approach from the others, which often fail, is the others don't change from polycations to anything else," said chemistry Professor Paul Wender, co-author of the paper. "Whereas, the ones that we're working with will change from polycations to neutral small molecules. That mechanism is really unprecedented."

As part of their change from polycations to polyneutrals, CARTs biodegrade and are eventually excreted from the body.

The power of collaboration

This research was made possible through coordination between the chemists and experts in imaging molecules in live animals, who rarely work together directly. With this partnership, the synthesis, characterization and testing of compounds could take as little as a week.

"We are so fortunate to engage in this kind of collaborative project between chemistry and our clinical colleagues. It allowed us to see our compounds go from very basic building blocks -- all the way from chemicals we buy in a bottle -- to putting a firefly gene into a mouse," said Colin McKinlay, a graduate student in the Wender lab and co-lead author of the study.

Not only did this enhanced ability to test and re-test new molecules lead to the discovery of their charge-altering behavior, it allowed for quick optimization of their properties and applications. As different challenges arise in the future, the researchers believe they will be able to respond with the same rapid flexibility.

After showing that the CARTs could deliver a glowing jellyfish protein to cells in a lab dish, the group wanted to find out if they worked in living mice, which was made possible through the expertise of the Contag lab, run by Christopher Contag, professor of pediatrics and of microbiology and immunology. Together, the multidisciplinary team showed that the CARTs could effectively deliver mRNA that produced glowing proteins in the thigh muscle or in the spleen and liver, depending on where the injection was made.

A bright future ahead

The researchers said CARTs could move the field of gene therapy forward dramatically in several directions.

"Gene therapy has been held up as a silver bullet because the idea that you could pick any gene you want is so alluring," said Jessica Vargas, co-lead author of the study, who was a PhD student in the Wender lab during this research. "With mRNA, there are more limitations because the protein expression is transient, but that opens up other applications where you wouldn't use other types of gene therapy."

One especially appropriate application of this technology is vaccination. At present, vaccines require introducing part of a virus or an inactive virus into the body in order to elicit an immune response. CARTs could potentially cut out the middleman, directly instructing the body to produce its own antigens. Once the CART dissolves, the immunity remains without any leftover foreign material present.

The team is also working on applying their technique to another genetic messenger that would produce permanent effects, making it a complementary option to the temporary mRNA therapies. With the progress already made using mRNA and the potential of their ongoing research, they and others could be closer than ever to making individualized therapeutics using a person's own cells. "Creating a firefly protein in a mouse is amazing but, more than that, this research is part of a new era in medicine," said Wender.

Read the rest here:
Glowing mice suggest new gene therapy technique -- ScienceDaily - Science Daily

Newswise PHILADELPHIA -- Penn Medicines Orphan Disease Center (ODC) announces a new partnership with FAST (Foundation for Angelman Syndrome Therapeutics) to study gene therapy approaches to treat Angelman syndrome (AS). FAST will provide funding to establish a gene therapy research program led by ODC.

Angelman syndrome is a rare neurological disorder that affects about one in 15,000 people, totaling about 490,000 worldwide. Individuals with AS have balance issues, motor impairment, and seizures, among other symptoms. Typical characteristics are not usually evident at birth, and people with the disorder develop feeding difficulties as infants and delayed development at about six to 12 months. In most cases, AS is not inherited and is often misdiagnosed as autism or cerebral palsy.

The Orphan Disease Center is delighted to launch a new collaboration with FAST on the development of gene therapy for Angelman syndrome, said ODC director James M. Wilson, MD, PhD, who is also a professor of Medicine and Pediatrics in the Perelman School of Medicine at the University of Pennsylvania. Combining ODCs experience in novel therapeutics with the tremendous progress made by FAST and its families, caregivers, and scientists has set the stage for an aggressive and exciting research plan.

Since its inception, ODC has aligned its mission to address the unmet needs of the rare disease community. ODC focuses on making rare disease research a priority and is committed to ensuring that the best science is accessible to the global community and to patients across all populations.

Currently, there are no treatments for AS, which is caused by mutations in the UBE3A gene and the loss of UBE3A protein expression. In the brain, UBE3A is primarily expressed by the maternal copy of the gene through a biological process known as paternal imprinting. UBE3A is an enzyme that targets proteins for removal from the cell, although it is not known how the loss of UBE3A in the brain leads to AS. Developing a gene therapy for AS will focus on replacing this gene in children who are lacking a functional copy.

We are excited to launch a new effort in Angelman syndrome in collaboration with the Angelman community and FAST, said Ashley Winslow, PhD, ODC senior director. Advancements in the understanding of AS make therapeutic approaches like gene therapy a natural fit for treating Angelman syndrome.

All of the board members of FAST are parents who are working toward breakthrough treatments for our children, said FAST chief scientific officer Allyson Berent, DVM, DACVIM. In making a commitment to develop an AS-specific gene therapeutic, Dr. Wilson and his research team further confirm our belief that Angelman syndrome is a curable disorder. To have an accomplished visionary researcher developing a potential gene therapy treatment for AS indicates we are closer than ever to this ultimate goal. Dr. Wilson and the team at Penn have such a successful track record in the field of gene therapy, and we are beyond enthusiastic that, for our children, the time is now.

The Orphan Disease Center is expanding its emphasis on neurodevelopmental disorders, such as AS, and through this effort hopes to leverage expertise across closely related disorders to accelerate therapeutic development.

Penn Medicine is one of the world's leading academic medical centers, dedicated to the related missions of medical education, biomedical research, and excellence in patient care. Penn Medicine consists of the Raymond and Ruth Perelman School of Medicine at the University of Pennsylvania (founded in 1765 as the nation's first medical school) and the University of Pennsylvania Health System, which together form a $5.3 billion enterprise. The Perelman School of Medicine has been ranked among the top five medical schools in the United States for the past 18 years, according to U.S. News & World Report's survey of research-oriented medical schools. The School is consistently among the nation's top recipients of funding from the National Institutes of Health, with $373 million awarded in the 2015 fiscal year. The University of Pennsylvania Health System's patient care facilities include: The Hospital of the University of Pennsylvania and Penn Presbyterian Medical Center -- which are recognized as one of the nation's top "Honor Roll" hospitals by U.S. News & World Report -- Chester County Hospital; Lancaster General Health; Penn Wissahickon Hospice; and Pennsylvania Hospital -- the nation's first hospital, founded in 1751. Additional affiliated inpatient care facilities and services throughout the Philadelphia region include Chestnut Hill Hospital and Good Shepherd Penn Partners, a partnership between Good Shepherd Rehabilitation Network and Penn Medicine. Penn Medicine is committed to improving lives and health through a variety of community-based programs and activities. In fiscal year 2015, Penn Medicine provided $253.3 million to benefit our community.

View original post here:
Penn Orphan Disease Center Partners with Foundation ... - Newswise - Newswise (press release)

February 16, 2017 by Taylor Kubota Colin McKinlay and Jessica Vargas are co-lead authors of research that could mark a significant step forward for gene therapy by providing a new way of inserting therapeutic proteins into diseased cells. Credit: L.A. Cicero

Timothy Blake, a postdoctoral fellow in the Waymouth lab, was hard at work on a fantastical interdisciplinary experiment. He and his fellow researchers were refining compounds that would carry instructions for assembling the protein that makes fireflies light up and deliver them into the cells of an anesthetized mouse. If their technique worked, the mouse would glow in the dark.

Not only did the mouse glow, but it also later woke up and ran around, completely unaware of the complex series of events that had just taken place within its body. Blake said it was the most exciting day of his life.

This success, the topic of a recent paper in Proceedings of the National Academy of Sciences, could mark a significant step forward for gene therapy. It's hard enough getting these protein instructions, called messenger RNA (mRNA), physically into a cell. It's another hurdle altogether for the cell to actually use them to make a protein. If the technique works in people, it could provide a new way of inserting therapeutic proteins into diseased cells.

"It's almost a childlike enthusiasm we have for this," said chemistry Professor Robert Waymouth. "The code for an insect protein is put into an animal and that protein is not only synthesized in the cells but it's folded and it becomes fully functional, capable of emitting light."

Although the results are impressive, this technique is remarkably simple and fast. And unlike traditional gene therapy that permanently alters the genetic makeup of the cell, mRNA is short-lived and its effects are temporary. The transient nature of mRNA transmission opens up special opportunities, such as using these compounds for vaccination or cancer immunotherapy.

Making a protein

Gene therapy is a decades-old field of research that usually focuses on modifying DNA, the fundamental genetic code. That modified DNA then produces a modified mRNA, which directs the creation of a modified protein. The current work skips the DNA and instead just delivers the protein's instructions.

Previous work has been successful at delivering a different form of RNA - called short interfering RNA, or siRNA - but sending mRNA through a cell membrane is a much bigger problem. While both siRNA and mRNA have many negative charges - so-called polyanions - mRNA is considerably more negatively charged, and therefore more difficult to sneak through the positively charged cell membrane.

What the researchers needed was a positively charged delivery method - a polycation - to complex, protect and shuttle the polyanions. However, this alone would only assure that the mRNA made it through the cell membrane. Once inside, the mRNA needed to detach from the transporter compound in order to make proteins.

The researchers addressed this twofold challenge with a novel, deceptively straightforward creation, which they call charge-altering releasable transporters (CARTs).

"What distinguishes this polycation approach from the others, which often fail, is the others don't change from polycations to anything else," said chemistry Professor Paul Wender, co-author of the paper. "Whereas, the ones that we're working with will change from polycations to neutral small molecules. That mechanism is really unprecedented."

As part of their change from polycations to polyneutrals, CARTs biodegrade and are eventually excreted from the body.

The power of collaboration

This research was made possible through coordination between the chemists and experts in imaging molecules in live animals, who rarely work together directly. With this partnership, the synthesis, characterization and testing of compounds could take as little as a week.

"We are so fortunate to engage in this kind of collaborative project between chemistry and our clinical colleagues. It allowed us to see our compounds go from very basic building blocks - all the way from chemicals we buy in a bottle - to putting a firefly gene into a mouse," said Colin McKinlay, a graduate student in the Wender lab and co-lead author of the study.

Not only did this enhanced ability to test and re-test new molecules lead to the discovery of their charge-altering behavior, it allowed for quick optimization of their properties and applications. As different challenges arise in the future, the researchers believe they will be able to respond with the same rapid flexibility.

After showing that the CARTs could deliver a glowing jellyfish protein to cells in a lab dish, the group wanted to find out if they worked in living mice, which was made possible through the expertise of the Contag lab, run by Christopher Contag, professor of pediatrics and of microbiology and immunology. Together, the multidisciplinary team showed that the CARTs could effectively deliver mRNA that produced glowing proteins in the thigh muscle or in the spleen and liver, depending on where the injection was made.

A bright future ahead

The researchers said CARTs could move the field of gene therapy forward dramatically in several directions.

"Gene therapy has been held up as a silver bullet because the idea that you could pick any gene you want is so alluring," said Jessica Vargas, co-lead author of the study, who was a PhD student in the Wender lab during this research. "With mRNA, there are more limitations because the protein expression is transient, but that opens up other applications where you wouldn't use other types of gene therapy."

One especially appropriate application of this technology is vaccination. At present, vaccines require introducing part of a virus or an inactive virus into the body in order to elicit an immune response. CARTs could potentially cut out the middleman, directly instructing the body to produce its own antigens. Once the CART dissolves, the immunity remains without any leftover foreign material present.

The team is also working on applying their technique to another genetic messenger that would produce permanent effects, making it a complementary option to the temporary mRNA therapies. With the progress already made using mRNA and the potential of their ongoing research, they and others could be closer than ever to making individualized therapeutics using a person's own cells. "Creating a firefly protein in a mouse is amazing but, more than that, this research is part of a new era in medicine," said Wender.

Explore further: Don't kill the messenger RNA

More information: Colin J. McKinlay et al. Charge-altering releasable transporters (CARTs) for the delivery and release of mRNA in living animals, Proceedings of the National Academy of Sciences (2017). DOI: 10.1073/pnas.1614193114

FedEx, UPS, DHLwhen it comes to sending packages, choices abound. But the most important delivery service you may not have heard of? mRNA. That's short for messenger RNA, which is how your DNA sends blueprints to the protein-assembly ...

Katie Whitehead, assistant professor of chemical engineering at Carnegie Mellon University, recently received the Young Faculty Award (YFA) from the Defense Advanced Research Projects Agency (DARPA). The YFA is a prestigious ...

Alternative splicing significantly expands the form and function of the genome of organisms with limited gene numbers and is especially important for several stages of mouse spermatogenesis.

A tiny therapeutic delivery system that can control the body's ability to manufacture proteins has been developed by Saudi Arabia's King Abdullah University of Science and Technology (KAUST) researchers.

A new scientific study conducted by a team of leading geneticists has characterized how cells know when to stop translating DNA into proteins, a critical step in maintaining healthy protein levels and cell function. In the ...

The progressive dementia suffered by patients with Alzheimer's disease (AD) affects millions of people globally each year and puts considerable strain on healthcare services. The mechanisms behind AD are not yet clear, and ...

New DNA-based research provides compelling evidence that a group of strange-looking fish living near the mouth of the Congo River are evolving due to the intense hydraulics of the river's rapids and deep canyons. The study, ...

(Phys.org)A team of researchers with members from the University of Arizona and New Mexico State University has discovered how a species of moth is able to repair oxidative muscle damage without consuming antioxidants. ...

At what point on the journey along the branches of the evolutionary tree does a population become its own, unique species? And is a species still distinct, if it mates with a different, but closely related species? Evolutionary ...

A new University of Queensland-led study could help scientists more accurately predict and explain patterns of diversity in nature.

It's well known that when the DNA in a cell is damaged, the cell responds by activating specific genes that help defend the integrity of its genome. But less well studied is the fact that the cell actually shuts down the ...

Climate change from political and ecological standpoints is a constant in the media and with good reason, said a Texas A&M AgriLife Research scientist, but proof of its impact is sometimes found in unlikely places.

Adjust slider to filter visible comments by rank

Display comments: newest first

No picture of the glowing mouse? How disappointing.

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

See the original post here:
In a possible step forward for gene therapy, researchers made mice glow like fireflies - Phys.Org

DUBLIN--(BUSINESS WIRE)--Research and Markets has announced the addition of Jain PharmaBiotech's new report "Gene Therapy - Technologies, Markets and Companies" to their offering.

Gene therapy technologies are described in detail including viral vectors, nonviral vectors and cell therapy with genetically modified vectors. Gene therapy is an excellent method of drug delivery and various routes of administration as well as targeted gene therapy are described. There is an introduction to technologies for gene suppression as well as molecular diagnostics to detect and monitor gene expression.

Clinical applications of gene therapy are extensive and cover most systems and their disorders. Full chapters are devoted to genetic syndromes, cancer, cardiovascular diseases, neurological disorders and viral infections with emphasis on AIDS. Applications of gene therapy in veterinary medicine, particularly for treating cats and dogs, are included.

Research and development is in progress in both the academic and the industrial sectors. The National Institutes of Health (NIH) of the US is playing an important part. As of 2015, over 2050 clinical trials have been completed, are ongoing or have been approved worldwide. A breakdown of these trials is shown according to the geographical areas and applications.

The markets for gene therapy are difficult to estimate as there is only one approved gene therapy product and it is marketed in China since 2004. Gene therapy markets are estimated for the years 2016-2026. The estimates are based on epidemiology of diseases to be treated with gene therapy, the portion of those who will be eligible for these treatments, competing technologies and the technical developments anticipated in the next decades. In spite of some setbacks, the future for gene therapy is bright. The markets for DNA vaccines are calculated separately as only genetically modified vaccines and those using viral vectors are included in the gene therapy markets

Profiles of 188 companies involved in developing gene therapy are presented along with 233 collaborations. There were only 44 companies involved in this area in 1995. In spite of some failures and mergers, the number of companies has increased more than 4-fold within a decade. These companies have been followed up since they were the topic of a book on gene therapy companies by the author of this report.

Key Topics Covered:

Part I: Technologies & Markets

1. Introduction

2. Gene Therapy Technologies

3. Clinical Applications of Gene Therapy

4. Gene Therapy of Genetic Disorders

5. Gene Therapy of Cancer

6. Gene Therapy of Neurological Disorders

7. Gene Therapy of Cardiovascular Disorders

8. Gene therapy of viral infections

9. Research, Development and Future of Gene Therapy

10. Regulatory, Safety and Ethical Issues of Gene Therapy

11. Markets for Gene Therapy

12. References

Part II: Companies

13. Companies involved in Gene Therapy

For more information about this report visit http://www.researchandmarkets.com/research/npn4n6/gene_therapy

The rest is here:
Gene Therapy Technologies, Markets and Companies 2017 - Research and Markets - Business Wire (press release)

February 15, 2017 In a study replacing the mutated gene responsible for myotubular myopathy with a healthy gene throughout the entire musculature of affected dogs, researchers observed a relationship between dosage and survival. Credit: Martin Childer lab/UW Medicine

Work on gene therapy is showing significant progress for restoring muscle strength and prolonging lives in dogs with a previously incurable, inherited neuromuscular disease. UW Medicine Institute for Stem Cell and Regenerative Medicine scientists are leading the multi-institutional research effort.

The disease arises from a mutation in genes that normally make a protein, called myotubularin, essential for proper muscle function. Puppies with this naturally occurring mutation exhibit several features of babies with the same defective gene. The rare disorder, called myotubular myopathy, or MTM, affects only males. It causes fatal muscle wasting. Both dogs and boys with the disease typically succumb in early life due to breathing difficulties.

For decades, researchers have struggled to find suitable treatments for genetic muscle diseases like this one. Four collaborating research groups in the United States and France found a way to safely replace the disease-causing MTM gene with a healthy gene throughout the entire musculature of affected dogs.

Their most recent findings were published online this week in Molecular Therapy.

Their paper reports that diseased dogs treated with a single infusion of the corrective therapy were indistinguishable from normal animals one year later.

"This regenerative technology allowed dogs that otherwise would have perished to complete restoration of normal health," said Dr. Martin K. "Casey" Childers, UW Medicine researcher and physician. Childers is a professor of rehabilitation medicine at the University of Washington School of Medicine and co-director of the Institute for Stem Cell and Regenerative Medicine.

Gene therapy holds the promise to treat many inherited diseases. To date, this approach has not been widely translated into treatment of skeletal muscle disorders.

"We report here a gene therapy dose-finding study in a large animal model of a severe muscle disease where a single treatment resulted in dramatic rescue," said Childers. The findings demonstrate potential application across a wide range of diseases and broadly translate to human studies. The data supports the development of gene therapy clinical trials for myotubular myopathy, the researchers concluded.

UW Medicine researchers David Mack, Melissa Goddard, Jessica Snyder, Matthew Elverman, and Valerie Kelly co-authored the report, "Systemic AAV8-mediated gene therapy drives whole-body correction of myotubular myopathy in dogs." This study was conducted in collaboration with Harvard University, Medical College of Wisconsin, Virginia Tech, INSERM, and Genethon.

Explore further: Gene therapy leads to robust improvements in animal model of fatal muscle disease

More information: Molecular Therapy, http://www.cell.com/molecular-therapy-family/molecular-therapy/fulltext/S1525-0016(17)30056-4 , DOI: 10.1016/j.ymthe.2017.02.004

Preclinical studies show that gene therapy can improve muscle strength in small- and large-animal models of a fatal congenital pediatric disease known as X-linked myotubular myopathy. The results, appearing in the Jan. 22 ...

Muscular dystrophy, which affects approximately 250,000 people in the U.S., occurs when damaged muscle tissue is replaced with fibrous, fatty or bony tissue and loses function. For years, scientists have searched for a way ...

A collaborative research team including a Medical College of Wisconsin (MCW) pediatric neuropathologist successfully mitigated some of the effects of a muscular disease by using a new targeted enzyme replacement therapy strategy ...

Researchers have developed a new strategy using lung-targeted gene therapy that may lead to improved treatments for inherited diseases including emphysema.

Zebrafish with very weak muscles helped scientists decode the elusive genetic mutation responsible for Native American myopathy, a rare, hereditary muscle disease that afflicts Native Americans in North Carolina.

University of Michigan researchers have discovered a new cause of congenital myopathy: a mutation in a previously uncharacterized gene, according to research published this month in the American Journal of Human Genetics.

Work on gene therapy is showing significant progress for restoring muscle strength and prolonging lives in dogs with a previously incurable, inherited neuromuscular disease. UW Medicine Institute for Stem Cell and Regenerative ...

Purdue University and Indiana University School of Medicine scientists were able to force an epigenetic reaction that turns on and off a gene known to determine the fate of the neural stem cells, a finding that could lead ...

Just before Rare Disease Day 2017, a study from the Monell Center and collaborating institutions provides new insight into the causes of trimethylaminura (TMAU), a genetically-transmitted metabolic disorder that leads to ...

Monash University and Danish researchers have discovered a gene in worms that could help break the cycle of overeating and under-exercising that can lead to obesity.

Most of us would be lost without Google maps or similar route-guidance technologies. And when those mapping tools include additional data about traffic or weather, we can navigate even more effectively. For scientists who ...

A new study shows how errors in a specific gene can cause growth defects associated with a rare type of dwarfism.

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

See the rest here:
Gene therapy treats muscle-wasting disease in dogs - Medical Xpress

DUBLIN, Feb. 15, 2017 /PRNewswire/ --

Research and Markets has announced the addition of Jain PharmaBiotech's new report "Gene Therapy - Technologies, Markets and Companies" to their offering.

Gene therapy technologies are described in detail including viral vectors, nonviral vectors and cell therapy with genetically modified vectors. Gene therapy is an excellent method of drug delivery and various routes of administration as well as targeted gene therapy are described. There is an introduction to technologies for gene suppression as well as molecular diagnostics to detect and monitor gene expression.

Clinical applications of gene therapy are extensive and cover most systems and their disorders. Full chapters are devoted to genetic syndromes, cancer, cardiovascular diseases, neurological disorders and viral infections with emphasis on AIDS. Applications of gene therapy in veterinary medicine, particularly for treating cats and dogs, are included.

Research and development is in progress in both the academic and the industrial sectors. The National Institutes of Health (NIH) of the US is playing an important part. As of 2015, over 2050 clinical trials have been completed, are ongoing or have been approved worldwide.A breakdown of these trials is shown according to the geographical areas and applications.

The markets for gene therapy are difficult to estimate as there is only one approved gene therapy product and it is marketed in China since 2004. Gene therapy markets are estimated for the years 2016-2026. The estimates are based on epidemiology of diseases to be treated with gene therapy, the portion of those who will be eligible for these treatments, competing technologies and the technical developments anticipated in the next decades. In spite of some setbacks, the future for gene therapy is bright.The markets for DNA vaccines are calculated separately as only genetically modified vaccines and those using viral vectors are included in the gene therapy markets

Profiles of 188 companies involved in developing gene therapy are presented along with 233 collaborations. There were only 44 companies involved in this area in 1995. In spite of some failures and mergers, the number of companies has increased more than 4-fold within a decade. These companies have been followed up since they were the topic of a book on gene therapy companies by the author of this report.

Key Topics Covered:

Part I: Technologies & Markets

1. Introduction

2. Gene Therapy Technologies

3. Clinical Applications of Gene Therapy

4. Gene Therapy of Genetic Disorders

5. Gene Therapy of Cancer

6. Gene Therapy of Neurological Disorders

7. Gene Therapy of Cardiovascular Disorders

8. Gene therapy of viral infections

9. Research, Development and Future of Gene Therapy

10. Regulatory, Safety and Ethical Issues of Gene Therapy

11. Markets for Gene Therapy

12. References

Part II: Companies

13. Companies involved in Gene Therapy

For more information about this report visit http://www.researchandmarkets.com/research/jtwqds/gene_therapy

Media Contact:

Laura Wood, Senior Manager press@researchandmarkets.com

For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900

U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716

SOURCE Research and Markets

RELATED LINKS http://www.researchandmarkets.com

Excerpt from:
Global Gene Therapy Technologies, Markets and Companies 2016-2026 - Research and Markets - PR Newswire UK (press release)

CRISPR: gene editing made easy

Ella Maru Studio/SPL

By Jessica Hamzelou

While gene editing is already saving lives, for now, the technique shouldnt be used to edit embryos or create changes that will be passed on through the generations. So say the authors of a new report on editing the human genome.

However, such germline editing could be permitted in the future, if properly regulated and with public approval, concludes the report. It was compiled by the Committee on Human Genome Editing, a group of 22 researchers, lawyers and ethicists.

Gene therapy isnt new, but the development of the CRISPR Cas-9 technique has made it much easier to change a genome. The technique enables researchers to specifically target a region of DNA and add or remove genes both a useful tool for research, and a technique that can treat diseases in people.

But gene editing treatments are not without some risk. Theres a chance, for instance, that a therapy will have off-target effects, changing other genes. The risks will depend on the disorder and the treatment, and regulators must weigh up the risks against treatment benefits on a case-by-case basis, the authors say.

The risks are higher when it comes to germline editing. Beyond off-target effects, theres a chance that attempts to perform gene editing on an embryo will create a mosaic of treated and untreated cells. Its the most common problem in mouse studies, says Robin Lovell-Badge of the Francis Crick Institute in London, who co-authored the report.

Lovell-Badge and his colleagues concluded that germline editing could be performed in humans, but only after much more research to minimise the risks and weigh them up against any benefits. Even then, the public must have a say, and any trials must be performed under strict oversight.

The report is also not in favour of gene editing techniques to enhance people, or create designer babies but only for the time being. Its the thing that worries people the most, because it is felt to be unfair, says Lovell-Badge. Its the same as using drugs to cheat.

But the boundary between treatment and enhancement is often blurred. If you were able to lower a persons cholesterol for example, where would the cut-off be? In the future, some aspects of enhancement might be considered acceptable, says Lovell-Badge. We may need to modify aspects of our physiology to adapt to climate change, but thats being speculative, he says. Were not saying it should never be done but not now.

Based on what we already know about genes and health, it might be possible to boost a persons muscle mass, for instance, using gene editing. But for many other features including intelligence hundreds or thousands of genes are involved. Using gene editing to enhance these features isnt currently feasible.

Read more: Why banning CRISPR gene editing would be unnecessarily cautious

More on these topics:

Follow this link:
Human genome editing shouldn't be used for enhancement yet - New Scientist

25 years have passed since the publication of the first work on solid lipid nanoparticles (SLNs) and nanostructured lipid carriers (NLCs) as a system for delivering drugs. So the European Journal of Pharmaceutics and Biopharmaceutics has prepared a special edition for which it asked the PharmaNanoGene group of the UPV/EHU-University of the Basque Country to produce a piece of work reviewing the application of SLNs and NLCs in gene therapy since the group's significant contributions made in this area have been included in various international scientific publications.

Lipid nanoparticles (SLNs and NLCs) are regarded as highly promising systems for delivering nucleic acids in gene therapy. Until now, viral systems have been the most effective method for delivering genetic matter but they pose significant safety problems. "Non-viral vectors, including SLNs and NLCs, are less effective but much safer even though their effectiveness has increased significantly in recent years," pointed out Alicia Rodrguez, Mara ngeles Solins and Ana del Pozo, authors of the article published in the European Journal of Pharmaceutics and Biopharmaceutics.

This review article describes these systems and their main advantages in gene therapy, such as their capacity to protect the gene material against degradation, to facilitate cell and nucleus internalisation and to boost the transfection process. "What is more, the nanoparticles are made up of biocompatible, biodegradable materials, they are easy to produce on a large scale, they can be sterilised and freeze-dried and are very stable both in biological fluids and in storage," explained the researchers.

This review also includes the main diseases in which lipid nanoparticles are being applied, generally on the preclinical level: degenerative diseases of the retina, infectious diseases, metabolic disorders, and cancer, among others. "At PharmaNanoGene we are working on the design and evaluation of SLNs for treating some of these diseases using gene therapy. We are studying the relationship between formulation factors and the processes involving the intracellular internalisation and disposition of the genetic material that condition the effectiveness of the vectors and which is essential in the optimisation process, and for the first time we have demonstrated the capacity of SLNs to induce the synthesis of a protein following their intravenous administration in mice," they stressed.

The publication also includes other pieces of work by this UPV/EHU research group on the application of SLNs in the treatment of rare diseases, such as chromosome-X-linked juvenile retinoschisis, a disorder in which the retina becomes destructured due to a deficiency in the protein retinoschisin. "One of the main achievements of our studies in this field has been to demonstrate, also for the first time, the capacity of a non-viral vector to transfect the retina of animals lacking the gene that encodes this protein and partially restore its structure, showing than non-viral gene therapy is a viable, promising therapeutic tool for treating degenerative disorders of the retina," specified the researchers.

The application of SLNs for treating Fabry disease, a serious, multi-system metabolic disorder of a hereditary nature, has also been studied at PharmaNanoGene. "This is a monogenic disease linked to the X-chromosome which is caused by various gene mutations in the gene that encodes the a-galactosidase A (a-Gal A) enzyme. In cell models of this disease we have demonstrated the capacity of SLNs to induce the synthesis of a-Gal A." They have also reviewed the application of lipid nanoparticles to the treatment of infectious diseases: "Our work in this field shows that SLNs with RNA interference are capable of inhibiting a replicon of the hepatitis C virus in vitro, which was used as proof-of-concept of the use of SLN-based vectors as a new therapeutic strategy for treating this infection and others related to it."

Story Source:

Materials provided by University of the Basque Country. Note: Content may be edited for style and length.

Read the original post:
Lipid nanoparticles for gene therapy -- ScienceDaily - Science Daily