Archive for the ‘Gene Therapy Research’ Category

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Dave Blanchard

OPB | May 22, 2014 12:06 p.m. | Updated: May 22, 2014 1:51 p.m.

An egg cell's nucleus is extracted by apipette.

OHSU

This March, Oregon Health & Science University (OHSU) created a new Center for Embryonic Cell and Gene Therapy. The facility will be focused in part on advancing the work of Shoukhrat Mitalipov, one of the worlds leading researchers on embryonic stem cells. Mitalipov has been working for years on two promising areas of stem cellscience.

The first research area is a gene therapy for women with diseases stored in DNA located in their mitochondria. Mitalipovs lab has developed a technique to extract the nucleus from a cell with damaged mitochondrial DNA, and implant it in a cell with healthy mitochondria. The process would allow most of the mothers DNA to be inherited by her child, without the risk of the mitochondrial diseases. Mitalipov hopes to begin clinical trials of the procedure, and the FDA is in the process of deciding whether to approve the technique soon. Some critics have ethical and medical concerns about creating an embryo with DNA from three differentpeople.

The second area, which has garnered even more attention, is the field of embryonic stem cell cloning. Last May, Mitalipovs lab became the first team to create human embryonic stem cells by cloning a breakthrough that was highlighted by Nature, Discover, Science, and National Geographic as one of the most significant science stories of the year. Now Miltalipovs lab is trying to figure out how to further that field ofresearch.

Well check in with Mitalipov to hear about his hopes for his areas of research, and where he thinks the future holds for stem cell science and genetherapy.

Rose E. Tucker Charitable Trust

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OHSU Scientist Pushes Forward With Stem Cell Research

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Newswise PHILADELPHIA The University of Pennsylvanias Center for Orphan Disease Research and Therapy will host a symposium on Friday, May 2 detailing developing therapeutics for rare/orphan diseases, as well as a poster session showcasing rare disease research at the Perelman School of Medicine and The Childrens Hospital of Philadelphia. Following the Symposium, on Saturday May 3, the Center is sponsoring the Million Dollar Bike Ride for rare diseases research with a starting/finish line on the Penn Campus at 31 & Chestnut Street. The funds raised from over 350 cyclists at the Ride will be used for the rare disease grants program sponsored by the Center.

When: Friday, May 2, 2014, 8:30am 5:00pm

Where: Smilow Center for Translational Research 3400 Civic Center Boulevard Philadelphia PA, 19104

9:00 - 9:15 AM Welcome and Opening Remarks H. Lee Sweeney, PhD Director, Center for Orphan Disease Research and Therapy 9:15 - 10:00 AM Emil D. Kakkis, MD, PhD Chief Executive Officer and President of Ultragenyx Pharmaceutical, Inc. Improving the Process of Rare Disease Treatment Development 10:00 - 10:45 AM Jerry R. Mendell, MD Director, Center for Gene Therapy, The Research Institute at Nationwide Childrens Hospital Progress Toward Molecular Based Therapies for Neuromuscular Disease 10:45 - 11:00 AM BREAK 11:00 - 11:45 PM Forbes D. Porter, MD, PhD Senior Investigator, Program Head and Clinical Director, NICHD, NIH Development of a 2-hydroxypropyl--cyclodextrin therapeutic trial for Niemann-Pick disease, type C1 11:45 - 12:30 PM Akshay K. Vaishnaw, MD, PhD Executive Vice-President & Chief Medical Officer, Alnylam Pharmaceuticals Inc. Development of a Novel RNAi Therapeutic, Patisiran, for the Treatment of TTRmediated Familial Amyloidotic Polyneuropathy (FAP) 12:30 - 2:00 PM LUNCH 2:00 - 2:45 PM Gwyneth Jane Farrar, PhD Professor of Genetics, Smurfit Institute of Genetics, School of Genetics and Microbiology, Trinity College Dublin, Ireland Exploration of AAV-Mediated Gene therapies for Inherited Ocular Disorders 2:45- 3:30 PM Edward G.D. Tuddenham MD Emeritus Professor of Haemophilia UCL Katharine Dormandy Haemophilia Centre, Royal Free Hospital "Gene Therapy for Haemophilia B - UCL/St Jude's Trial Update at 4 Years" Agenda and other details can be found on the Center for Orphan Disease Research and Therapy site. Register for this free symposium here.

### 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 $4.3 billion enterprise. The Perelman School of Medicine has been ranked among the top five medical schools in the United States for the past 17 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 $392 million awarded in the 2013 fiscal year. The University of Pennsylvania Health System's patient care facilities include: The Hospital of the University of Pennsylvania -- recognized as one of the nation's top "Honor Roll" hospitals by U.S. News & World Report; Penn Presbyterian Medical Center; Chester County Hospital; 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 2013, Penn Medicine provided $814 million to benefit our community.

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International Rare Disease Symposium Brings Together Academia, Industry, and Government at Penn Medicine

Researchers at UNSW Australia have for the first time used electrical pulses delivered from a cochlear implant to deliver gene therapy, thereby successfully regrowing auditory nerves.

The research also heralds a possible new way of treating a range of neurological disorders, including Parkinson's disease, and psychiatric conditions such as depression through this novel way of delivering gene therapy.

The research is published today (Thursday 24 April) in the journal Science Translational Medicine.

"People with cochlear implants do well with understanding speech, but their perception of pitch can be poor, so they often miss out on the joy of music," says UNSW Professor Gary Housley, who is the senior author of the research paper.

"Ultimately, we hope that after further research, people who depend on cochlear implant devices will be able to enjoy a broader dynamic and tonal range of sound, which is particularly important for our sense of the auditory world around us and for music appreciation," says Professor Housley, who is also the Director of the Translational Neuroscience Facility at UNSW Medicine.

The research, which has the support of Cochlear Limited through an Australian Research Council Linkage Project grant, has been five years in development.

The work centres on regenerating surviving nerves after age-related or environmental hearing loss, using existing cochlear technology. The cochlear implants are "surprisingly efficient" at localised gene therapy in the animal model, when a few electric pulses are administered during the implant procedure.

"This research breakthrough is important because while we have had very good outcomes with our cochlear implants so far, if we can get the nerves to grow close to the electrodes and improve the connections between them, then we'll be able to have even better outcomes in the future," says Jim Patrick, Chief Scientist and Senior Vice-President, Cochlear Limited.

It has long been established that the auditory nerve endings regenerate if neurotrophins -- a naturally occurring family of proteins crucial for the development, function and survival of neurons -- are delivered to the auditory portion of the inner ear, the cochlea.

But until now, research has stalled because safe, localised delivery of the neurotrophins can't be achieved using drug delivery, nor by viral-based gene therapy.

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Hearing quality restored with bionic ear technology used for gene therapy: Re-growing auditory nerves

MADRID, SPAIN - Spanish scientists have for the first time used gene therapy to reverse memory loss in mice with Alzheimer's, an advance that could lead to new drugs to treat the disease, they said Wednesday.

The Autonomous University of Barcelona team injected a gene which causes the production of a protein that is blocked in patients with Alzheimer's into the hippocampus -- a region of the brian essential to memory processing -- in mice that were in the initial stages of the disease.

"The protein that was reinstated by the gene therapy triggers the signals needed to activate the genes involved in long-term memory consolidation," the university said in a statement.

Gene therapy involves transplanting genes into a patient's cells to correct an otherwise incurable disease caused by a failure of one or another gene.

The finding was published in The Journal of Neuroscience and it follows four years of research.

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Scientists reverse memory loss in Alzheimer's-afflicted mice

WASHINGTON - Australian researchers are trying a novel way to boost the power of cochlear implants: They used the technology to beam gene therapy into the ears of deaf animals and found the combination improved hearing.

The approach reported Wednesday isn't ready for human testing, but it's part of growing research into ways to let users of cochlear implants experience richer, more normal sound.

Normally, microscopic hair cells in a part of the inner ear called the cochlea detect vibrations and convert them to electrical impulses that the brain recognizes as sound. Hearing loss typically occurs as those hair cells are lost, whether from aging, exposure to loud noises or other factors.

Cochlear implants substitute for the missing hair cells, sending electrical impulses to directly activate auditory nerves in the brain. They've been implanted in more than 300,000 people. While highly successful, they don't restore hearing to normal, missing out on musical tone, for instance.

The idea behind the project: Perhaps a closer connection between the implant and the auditory nerves would improve hearing. Those nerves' bush-like endings can regrow if exposed to nerve-nourishing proteins called neurotrophins. Usually, the hair cells would provide those.

Researchers at Australia's University of New South Wales figured out a new way to deliver one of those growth factors.

They injected a growth factor-producing gene into the ears of deafened guinea pigs, animals commonly used as a model for human hearing. Then they adapted an electrode from a cochlear implant to beam in a few stronger-than-normal electrical pulses.

That made the membranes of nearby cells temporarily permeable, so the gene could slip inside. Those cells began producing the growth factor, which in turn stimulated regrowth of the nerve fibers closing some of the space between the nerves and the cochlear implant, the team reported in the journal Science Translational Medicine.

The animals still needed a cochlear implant to detect sound but those given the gene therapy had twice the improvement, they concluded.

Senior author Gary Housley estimated small studies in people could begin in two or three years.

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Researchers add gene therapy to cochlear implants in deaf animals

Research assistant Natalie Thomas pulls a slice of a cancerous tumor for analysis at the Anschutz Medical Campus. (Andy Cross, The Denver Post)

Ellen Smith received a death sentence for her advanced lung cancer five years ago, but it was commuted by a revolution in human genetics, drug therapies and clinical approaches unfolding at the University of Colorado Hospital.

The advances have saved her life, by her reckoning, four times.

The accelerating speed of DNA sequencing, drug development and data analysis has led UCHealth, the University of Colorado Medical School and Children's Hospital Colorado to join in an effort to fundamentally change the way they care for patients.

The partnership will invest more than $63 million over the next five years to create a new division, adding clinicians, genetic counselors, researchers and advanced practice nurses and also expanding a DNA bank and advanced data warehouse. It's called the Center for Personalized Medicine and Biomedical Informatics.

The pioneering field of personalized medicine uses molecular analysis to determine a patient's predisposition to developing certain diseases and to deliver tailored medical treatment.

"There is no doubt in my mind that this will change how we treat disease, how we teach our students, how physicians work, how we raise our kids and how we conduct public health policy," Dr. David Schwartz, chair of the CU Department of Medicine, said of the center.

The DNA bank, Schwartz said, probably will require a year of discussion with physicians, academicians, lawyers, ethicists and patient advocates about what it really means to secure patients' genetic blueprints and how they should be used.

While the center will be based on the Anschutz Medical Campus in Aurora, it will serve UCHealth's five hospitals and Children's Hospital. The DNA bank would sequence and analyze samples from around the region.

The benefits of personalized medicine have been evident for several years in cancer treatment, said Dr. Dan Theodorescu, director of the CU Cancer Center. It's why the center's survival rates are significantly better for certain types of cancers than the average national outcomes, he said. The new center will bring these kinds of lifesaving therapies to all disease fronts while providing more laboratory and analytical power to evaluate cancer DNA, he said.

Link:
CU system resets health care with $63M personalized medicine division

The Harvard-MIT genomic science institute stays mute on how it will assert control over the tools expected to speed cures and change gene therapy.

One of the most important genetic technologies developed in recent years is now patented, and researchers are wondering what they will and wont be allowed to do with the powerful method for editing the genome.

On Tuesday, the Broad Institute of MIT and Harvard announced that it had been granted a patent covering the components and methodology for CRISPRa new way of making precise, targeted changes to the genome of a cell or an organism. CRISPR could revolutionize biomedical research by giving scientists a more efficient way of re-creating disease-related mutations in lab animals and cultured cells; it may also yield an unprecedented way of treating disease (see Genome Surgery).

The patent, issued just six months after its application was filed, covers a modified version of the CRISPR-Cas9 system found naturally in bacteria, which microbes use to defend themselves against viruses. The patent also covers methods for designing and using CRISPRs molecular components.

The inventor listed on the patent is Feng Zhang, an MIT researcher and core faculty member of the Broad. Zhang was an MIT Technology Review Innovator Under 35 in 2013.

The patent describes how the tools could be used to treat diseases, and lists many specific conditions from epilepsy, to Huntingtons, to autism, and macular degeneration. One of the most exciting possibilities for CRISPR is its potential to treat genetic disorders by directly correcting mutations on a patients chromosomes. That would enable doctors to treat diseases that cannot be addressed by more traditional methods, a goal already set by a startup cofounded by Zhang called Editas Medicine (see New Genome-Editing Method Could Make Gene Therapy More Precise and Effective).

Another founder of Editas, Jennifer Doudna, and her institute, the University of California, have a pending patent application for CRISPR technology. How that west coast application will be affected is not yet clear. Its also unclear what impact the Broads claims on the technology will have on its commercial use and on basic research.

Chelsea Loughran, an intellectual property litigation lawyer who has been following CRISPR over the last year, says that lots of people are already using CRISPR and its not clear if it will now become harder for them to do that. All of that is in the hands of MIT and the Broad, she says.

While MIT, Harvard, and the Broad all jointly own the CRISPR patents announced yesterday, the Broads technology licensing office is managing decisions about who will get licenses to use the technology, says Lita Nelsen, director of the MIT Technology Licensing Office. (Licenses areformal permissions to use a patented technology, often in exchange for money.)

A spokesperson for the Broad says that specific details around licensing arent available at this time, but the Broad does intend to make this technology broadly available to scientists.

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Broad Institute Gets Patent on Revolutionary Gene-Editing Method

Genetics and the BAP1 Gene in Mesothelioma
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PUBLIC RELEASE DATE:

21-Mar-2014

Contact: Meng Zhao eic@nrren.org 86-138-049-98773 Neural Regeneration Research

Preliminary basic research and clinical findings have demonstrated that electroacupuncture therapy exhibits positive effects in ameliorating depression. However, most studies of the underlying mechanism are at the single gene level; there are few reports regarding the mechanism at the whole-genome level. Using a rat genomic gene-chip, Dr. Dongmei Duan and co-workers from General PLA Hospital in China profiled hippocampal gene expression changes in rats after electroacupuncture therapy. Electroacupuncture therapy alleviated depression-related manifestations in the model rats. Using gene-chip analysis, electroacupuncture at Baihui (DU20) and Yintang (EX-HN3) regulates the expression of 21 genes. Real-time PCR showed that the genes Vgf, Igf2, Tmp32, Loc500373, Hif1a, Folr1, Nmb, and Rtn were upregulated or downregulated in depression and that their expression tended to normalize after electroacupuncture therapy. These results, published in the Neural Regeneration Research (Vol. 9, No. 1, 2014), indicate that electroacupuncture modulates depression by regulating the expression of particular genes.

###

Article: " Hippocampal gene expression in a rat model of depression after electroacupuncture at the Baihui and Yintang acupoints," by Dongmei Duan1, Xiuyan Yang2, Tu Ya3, Liping Chen1 (1 Department of Traditional Chinese Medicine of South Building, Chinese PLA General Hospital, Beijing 100853, China; 2 Institute of Health Maintenance, Beijing University of Chinese Medicine, Beijing 100029, China; 3 School of Acupuncture and Moxibustion, Beijing University of Chinese Medicine, Beijing 100037, China)

Duan DM, Yang XY, Ya T, Chen LP. Hippocampal gene expression in a rat model of depression after electroacupuncture at the Baihui and Yintang acupoints. Neural Regen Res. 2014;9(1):76-83.

Contact:

Meng Zhao eic@nrren.org 86-138-049-98773 Neural Regeneration Research http://www.nrronline.org/

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Electroacupuncture effect on depression and variation of polygenes expression

10 hours ago by Lynn Yarris With the new SIF-seq technique, mouse embryonic stem cells can be used to identify human embryonic stem cell enhancers even when the human enhancers are not present in the mouse genome. Credit: Axel Visel, Berkeley Lab

An international team led by researchers with the Lawrence Berkeley National Laboratory (Berkeley Lab) has developed a new technique for identifying gene enhancers - sequences of DNA that act to amplify the expression of a specific gene in the genomes of humans and other mammals. Called SIF-seq, for site-specific integration fluorescence-activated cell sorting followed by sequencing, this new technique complements existing genomic tools, such as ChIP-seq (chromatin immunoprecipitation followed by sequencing), and offers some additional benefits.

"While ChIP-seq is very powerful in that it can query an entire genome for characteristics associated with enhancer activity in a single experiment, it can fail to identify some enhancers and identify some sites as being enhancers when they really aren't," says Diane Dickel, a geneticist with Berkeley Lab's Genomics Division and member of the SIF-seq development team. "SIF-seq is currently capable of testing only hundreds to a few thousand sites for enhancer activity in a single experiment, but can determine enhancer activity more accurately than ChIP-seq and is therefore a very good validation assay for assessing ChIP-seq results."

Dickel is the lead author of a paper in Nature Methods describing this new technique. The paper is titled "Function-based identification of mammalian enhancers using site-specific integration." The corresponding authors are Axel Visel and Len Pennacchio, also geneticists with Berkeley Lab's Genomics Division. (See below for a complete list of authors.)

With the increasing awareness of the important role that gene enhancers play in normal cell development as well as in disease, there is strong scientific interest in identifying and characterizing these enhancers. This is a challenging task because an enhancer does not have to be located directly adjacent to the gene whose expression it regulates, but can instead be located hundreds of thousands of DNA base pairs away. The challenge is made even more difficult because the activity of many enhancers is restricted to specific tissues or cell types.

"For example, brain enhancers will not typically work in heart cells, which means that you must test your enhancer sequence in the correct cell type," Dickel says.

Currently, enhancers can be identified through chromatin-based assays, such as ChIP-seq, which predict enhancer elements indirectly based on the enhancer's association with specific epigenomic marks, such as transcription factors or molecular tags on DNA-associated histone proteins. Visel, Pennacchio, Dickel and their colleagues developed SIF-seq in response to the need for a higher-throughput functional enhancer assay that can be used in a wide variety of cell types and devel-opmental contexts.

"We've shown that SIF-seq can be used to identify enhancers active in cardiomyocytes, neural progenitor cells, and embryonic stem cells, and we think that it has the potential to be expanded for use in a much wider variety of cell types," Dickel says. "This means that many more types of enhancers could potentially be tested in vitro in cell culture."

In SIF-seq, hundreds or thousands of DNA fragments to be tested for enhancer activity are coupled to a reporter gene and targeted into a single, reproducible site in embryonic cell genomes. Every embryonic cell will have exactly one potential enhancer-reporter. Fluorescence-activated sorting is then used to identify and retrieve from this mix only those cells that display strong reporter gene expression, which represent the cells with the most active enhancers.

"Unlike previous enhancer assays for mammals, SIF-seq includes the integration of putative enhancers into a single genomic locus," says co-corresponding author Visel. "Therefore, the activity of enhancers is assessed in a reproducible chromosomal context rather than from a transiently expressed plasmid. Furthermore, by making use of embryonic stem cells and in vitro differentiation, SIF-seq can be used to assess enhancer activity in a wide variety of disease-relevant cell types."

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New technique for identifying gene-enhancers

Mark Mercola, a scientist who studies heart disease at Sanford-Burnham Medical Research Institute.

A team including Sanford-Burnham Medical Research scientists has identified a form of RNA that plays a key role in inducing heart failure.

Called miR-25, the molecule is a short fragment of RNA, called microRNA. In a model of heart failure in mice, increasing the level of miR-25 reduced the efficiency of heart muscle contraction. Inhibiting miR-25 halted heart failure that had already been established.

The study was published Wednesday in the journal Nature. (If link is not live, check later). Scientists from the Icahn School of Medicine at Mount Sinai and UC San Diego collaborated with Sanford-Burnham scientists in the study.

The microRNA molecule blocks activity of a gene called SERCA2a, which regulates the flow of calcium ions into cardiac tissue. The gene has been identified in another study as a target for gene therapy in heart failure.

In this study, led by Sanford-Burnham heart disease researcher Mark Mercola, the gene activity was boosted by using antisense technology to inactivate miR-25. Antisense RNA molecules form a complementary sequence to the specific RNA molecule, called the "sense" molecule, they inactivate. The antisense molecules bind to the targeted RNA molecules, which prevents them from serving as a template for protein production.

The culprit molecule was identified with a functional screening system developed at Sanford-Burnham Mercola said in a Sanford-Burnham press release. Mercola is a professor in the Development, Aging, and Regeneration Program at Sanford-Burnham and a professor of bioengineering at UC San Diego Jacobs School of Engineering.

"Before the availability of high-throughput functional screening, our chance of teasing apart complex biological processes involved in disease progression like heart failure was like finding a needle in a haystack," Mercola said in the press release. "The results of this study validate our approach to identifying microRNAs as potential therapeutic targets with significant clinical value."

The screen searched through all human microRNAs to find those linked to heart failure. Colleagues at the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai found that injecting the antisense complement to miR-25 stopped heart-failure progression in mice, improved cardiac function and survival.

Heart failure is a progressive loss of the heart's ability to pump blood. It can be caused by heart attacks, high blood pressure, diabetes and other conditions. As heart failure worsens, patients become increasingly restricted in their physical activities. The disease affects nearly six million Americans.

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MicroRNA therapy may help heart failure

PUBLIC RELEASE DATE:

5-Mar-2014

Contact: Steve Graff stephen.graff@uphs.upenn.edu 215-349-5653 University of Pennsylvania School of Medicine

PHILADELPHIAUniversity of Pennsylvania researchers have successfully genetically engineered the immune cells of 12 HIV positive patients to resist infection, and decreased the viral loads of some patients taken off antiretroviral drug therapy (ADT) entirelyincluding one patient whose levels became undetectable. The study, appearing today in the New England Journal of Medicine, is the first published report of any gene editing approach in humans.

The phase I study was co-authored by researchers at Penn Medicine, the Albert Einstein College of Medicine and scientists from Sangamo BioSciences, which developed the zinc finger nuclease (ZFN) technology, the T cell therapy approach used in the clinical trial.

"This study shows that we can safely and effectively engineer an HIV patient's own T cells to mimic a naturally occurring resistance to the virus, infuse those engineered cells, have them persist in the body, and potentially keep viral loads at bay without the use of drugs," said senior author Carl H. June, MD, the Richard W. Vague Professor in Immunotherapy in the department of Pathology and Laboratory Medicine at Penn's Perelman School of Medicine. "This reinforces our belief that modified T cells are the key that could eliminate the need for lifelong ADT and potentially lead to functionally curative approaches for HIV/AIDS."

June and his colleagues, including Bruce L. Levine, PhD, the Barbara and Edward Netter Associate Professor in Cancer Gene Therapy in the department of Pathology and Laboratory Medicine and the director of the Clinical Cell and Vaccine Production Facility at Penn, used the ZFN technology to modify the T cells in the patientsa "molecular scissors," of sorts, to mimic the CCR5-delta-32 mutation. That rare mutation is of interest because it provides a natural resistance to the virus, but in only 1 percent of the general population. By inducing the mutations, the scientists reduced the expression of CCR5 surface proteins. Without those, HIV cannot enter, rendering the patients' cells resistant to infection.

For the study, the team infused the modified cells known as SB-728-Tinto two cohorts of patients, all treated with single infusionsabout 10 billion cellsbetween May 2009 and July 2012. Six were taken off antiretroviral therapy altogether for up to 12 weeks, beginning four weeks after infusion, while six patients remained on treatment.

Infusions were deemed safe and tolerable, the authors report, and modified T cells continued to persist in the patients when tested during follow up visits. One week after the initial infusion, testing revealed a dramatic spike in modified T cells inside the patients' bodies. While those cells declined over a number of weeks in the blood, the decrease of modified cells was significantly less than that of unmodified T cells during ADT treatment interruption. Modified cells were also observed in the gut-associated lymphoid tissue, which is a major reservoir of immune cells and a critical reservoir of HIV infection, suggesting that the modified cells are functioning and trafficking normally in the body.

The study also shows promise in the approach's ability to suppress the virus. The viral loads (HIV-RNA) dropped in four patients whose treatment was interrupted for 12 weeks. One of those patients' viral loads dropped below the limit of detection; interestingly, it was later discovered that the patient was found to be heterozygous for the CCR5 delta-32 gene mutation.

Originally posted here:
Gene therapy locks out HIV, paving the way to control virus without antiretroviral drug

GWEN IFILL: For all of the progress made in the fight against AIDS, it still takes a terrible toll. More than 35 million people are infected with HIV around the world. More than two million people are newly infected each year. And well over a million die from it annually.

But research released at an AIDS conference this week is raising hope about new inroads into treating it and preventing infections.

Jeffrey Brown has the story.

JEFFREY BROWN: Three reports attracted attention. One involved injections of drugs into monkeys that helped stop infections. A second revealed promising news of a baby born with the virus and given aggressive treatment. A third concerned so-called gene editing, altering cells to resist HIV.

The NIHs Institute of Allergy and Infectious Diseases has been funding much of this work. Dr. Anthony Fauci is its longtime director, and he joins me now.

And welcome back.

So, lets walk through some of this. First, the injections of long-lasting drugs into monkeys, explain the work and why its so important.

DR. ANTHONY FAUCI, National Institutes of Health: Well, the reason the reason the work is important is that we know, in human studies, several human studies, that if you give a drug to an uninfected person whos practicing risk behavior, we call it preexposure prophylaxis, that if they take the drug every day, it absolutely works and prevents infection in over 90 percent of the people.

The problem with the approach is that people dont like to take medicine every day or before or after a sexual encounter. So, a modality of prevention that you know works 90-plus percent doesnt work that well, purely because people dont adhere.

The experiments that have been reported recently now show that, in a monkey model, if you take a long-acting drug, a drug thats used in a different form to treat HIV infection, in a monkey model, and give an injection every so often, like every couple of months, you can actually prevent challenging that monkey with infection with the monkey version of HIV by exposing them rectally or vaginally.

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Injections, gene therapy and treatment for infants raise hope for fighting AIDS

Monica Coenraads and Dr. Neul discuss the genetics and nuances behind a Rett diagnosis
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Minecraft - Attack of The B-Team - Ep.9 : Advanced Genetics! w/ BdoubleO
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After having the genetic material in their white blood cells modified, some HIV patients saw their virus levels drop.

Gene editing a process used in a recent study targeting HIV is precisely what it sounds like and may hold new hope for patients battling the virus that causes AIDS.

Researchers involved in the study removed white blood cells the soldiers of the bodys immune system from the blood of12 HIV patients, then cut into the genes in those cellsand sewed in new pieces of DNA. They thengrew the modified immune cells and reinserted them into the patients.

[READ:Genetic Mutations Could Shrink Risk of Type 2 Diabetes]

The modified cells blocked the virus from fulfilling its mission: hijacking the immune system and leaving patients susceptible to myriad diseases.

"This study shows that we can safely and effectively engineer an HIV patient's own T cells to mimic a naturally occurring resistance to the virus, infuse those engineered cells, have them persist in the body, and potentially keep viral loads at bay without the use of drugs," Dr. Carl June, a study author and a professor in immunotherapy at theUniversity of Pennsylvanias Perelman School of Medicine, said in a release.

In a sense, what the study tests is the ability to trick HIV. By modifying a gene called CCR5 that contains the code fora receptor that, when present, essentiallyopens the door of a cellto the virus, researchers aim to prevent the virus from spreading.

Researchers at Penn Medicine and Yeshiva University's Albert Einstein College of Medicine gave each of 12 patients a single infusion of roughly 10 billion modified cells between May 2009 and July 2012. Sangamo BioSciences, a California-based biopharmaceutical company, developed the technology for the therapy and donated the genetic material that was stitched into patients' genes.

After a month, half of the patients were taken off their traditional HIV treatments for 12 weeks, and four saw their virus levels drop. Onepatients viral load dropped below detection levels, and researchers discovered this patient already had a naturally occurring mutation similar to the therapy.

"Since half the subject's CCR5 genes were naturally disrupted, the gene editing approach was building on the head start provided by inheriting the mutation from one parent," saidstudy author Bruce Levine, aUniversity of Pennsylvaniaprofessor in cancer gene therapy.

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Gene Therapy for HIV Delivers Hopeful Results - US News

By Deena Beasley

Thu Mar 6, 2014 4:21pm EST

A nurse carries a child in the San Jose Hospice, in Sacatepequez, 45 km (28 miles) of Guatemala City, November 30, 2012.

Credit: Reuters/Jorge Dan Lopez

(Reuters) - Treating HIV patients first with a chemotherapy drug improved their response to an experimental gene-modifying technique for controlling the virus, according to Sangamo BioSciences.

The company presented new data from a small early-stage trial of its treatment, SB-728-T, on Thursday at the Conference on Retroviruses and Opportunistic Infections in Boston.

Shares of Sangamo were up 17 percent at $22.92 in late trading on Nasdaq. Shares of the Richmond, California company have gained about 67 percent so far this year.

On Wednesday, the New England Journal of Medicine published data from an earlier trial showed that Sangamo's strategy of genetically modifying cells from people infected with HIV could become a way to control the virus that causes AIDS without using antiviral drugs.

"Sangamo's HIV 'suppression' is promising, but very early and far from a 'cure,'" RBC Capital Markets analyst Michael Yee said in a research note. "This is very early study for technology and safety validation."

The technique is designed to disrupt a gene, CCR5, used by the human immunodeficiency virus to infect T-cells, the white blood cells that fight viral infections. A patient's cells are removed and processed to alter the DNA that codes for the CCR5 receptor. The altered cells are multiplied and tested, then infused back into the patient.

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Chemo drug helps HIV patients respond to Sangamo gene therapy

Scientists have modified genes in the blood cells of HIV patients to help them resist the AIDS virus, and say the treatment seems safe and promising. The results give hope that this approach might one day free at least some people from needing medicines to keep HIV under control, a form of cure.

The idea came from an AIDS patient who appears cured after getting a cell transplant seven years ago in Berlin from a donor with natural immunity to HIV. Only about 1 percent of people have two copies of the gene that gives this protection.

Researchers are seeking a more practical way to get similar results by using gene therapy to modify patients' own blood cells.

A study of this in 12 patients was led by Dr. Carl June at the University of Pennsylvania. Results are in Thursday's the New England Journal of Medicine. These are the first published results from this method, which also has been tried in several smaller studies of patients in California.

HIV usually infects blood cells through a protein on their surface, a "docking station" called CCR5. A California company, Sangamo BioSciences Inc., makes a treatment that can knock out a gene that makes CCR5.

The 12 HIV patients had their blood filtered to remove some of their cells. The gene-snipping compound was added in the lab, and the cells were infused back into the patients.

Four weeks later, half of the patients were temporarily taken off AIDS medicines to see the gene therapy's effect. The virus returned in all but one of them, but the modified cells seemed to be protected from HIV infection and were more likely to survive than the cells that had not been treated.

"We knew that the virus was going to come back in most of the patients," but the hope is that the modified cells eventually will outnumber the rest and give the patient a way to control viral levels without medicines, said Dr. Pablo Tebas, one of the Penn researchers. That would be what doctors call a "functional cure," because the virus would still be present but held in check without treatment.

The lone patient whose HIV did not return turned out to have one copy of the protective gene, so "nature had done half of the job already," Tebas said.

The National Institute of Allergy and Infectious Diseases sponsored the work with Sangamo and Penn.

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Gene Therapy Seems Safe, May Help Control HIV - ABC News

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Dr. Max Gomez: HIV Gene Therapy
Genetically engineered white blood cells could mark the beginning of a cure for AIDS. CBS 2 #39;s Dr. Max Gomez has more. Subscribe Here: http://www.youtube.com/...

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Dr. Max Gomez: HIV Gene Therapy - Video

By Amy Norton HealthDay Reporter

WEDNESDAY, March 5, 2014 (HealthDay News) -- In an early step toward drug-free HIV therapy, researchers are reporting the first success in genetically "editing" T-cells in patients' immune systems to become resistant to the virus.

The findings, published in the March 6 issue of the New England Journal of Medicine, are based on only 12 patients. But experts were cautiously optimistic about what the study accomplished.

Specifically, researchers were able to take T-cells from the HIV patients' blood, then "knock out" a gene known as CCR5, which controls a protein that allows HIV to enter a cell.

The scientists then infused the genetically altered T-cells back into patients' blood, where they expanded in number. What's more, a few patients were taken off their HIV drugs temporarily and saw their virus levels decrease.

"This is impressive," said Rowena Johnston, director of research for amfAR, the Foundation for AIDS Research.

The altered T-cells "actually seem to be doing exactly what [the researchers] wanted them to," said Johnston, who was not involved in the study.

Still, she said, there are plenty of questions left and much research ahead. The investigators on the study agreed.

"This was a first-in-human study," said researcher Bruce Levine, an associate professor of cancer gene therapy at the University of Pennsylvania School of Medicine, in Philadelphia.

That means the trial was designed to see whether it's even safe to use this approach in people with HIV -- and not whether it's an effective therapy.

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Gene Therapy for Controlling HIV Shows Early Promise

Two doses of gene therapy restore vision to three women who were born nearly blind

Garretttaggs55, Wikimedia Commons

Gene therapy has markedly improved vision in both eyes in three women who were born virtually blind. The patients can now avoid obstacles even in dim light, read large print and recognize people's faces. The operation, researchers predict, should work even better in children and adolescents blinded by the same condition.

The advance, reported in the February 8 issue of Science Translational Medicine, extends earlier work by the same group. Between 2008 and 2011, Jean Bennett of the University of Pennsylvania's Mahoney Institute of Neurological Sciences and her colleagues used gene therapy to treat blindness in 12 adults and children with Leber's congenital amaurosis (LCA), a rare inherited eye disease that destroys vision by killing photoreceptorslight-sensitive cells in the retina at the back of the eye. Typically, afflicted children start life with poor vision, which worsens as more and more photoreceptors die.

The treatment grew out of the understanding that people with the disorder become blind because of genetic mutations in retinal cells. One mutated gene that causes the disorder is named RPE65. An enzyme encoded by RPE65 helps break down a derivative of vitamin A called retinol into a substance that photoreceptors need to detect light and send signals to the brain. Mutant forms of RPE65 prevent the production of this enzyme in a "nursery" layer of cells called the retinal pigment epithelium, which is attached to the retina and nourishes photoreceptors by breaking down retinol, among other cellular services.

In the initial study, retina specialist and Bennett's co-author Albert Maguire of Penn Medicine injected a harmless virus carrying normal copies of RPE65 into an area of the retinal pigment epithelium, which subsequently began producing the enzyme. In each of the 12 patients, Maguire treated one eyethe one with worse vision. Six patients improved so much they no longer met the criteria for legal blindness.

In the new study, Maguire injected the functional genes into the previously untreated eye in three of the women from the first group. Bennett followed the patients for six months after their surgeries. The women's vision in their previously untreated eye improved as soon as two weeks after the operation: They could navigate an obstacle course, even in dim light, avoiding objects that had tripped them up before, as well as recognize people's faces and read large signs. Bennett showed that not only were the women's eyes much more sensitive to light, their brains were much more responsive to optical input as well. Functional magnetic imaging showed regions of their visual cortices that had remained offline before gene therapy began to light up.

Surprisingly, Bennett reports, the second round of gene therapy further strengthened the brain's response to the initially treated eye as well as the newly treated one. "That wasn't something we had been expecting, but it makes sense because the two eyes act in concert, and some aspects of vision rely on binocularity." In the new paper, the authors suggest that neuroplasticity plays a role: It is possible that regions of the visual cortex responding to the newly flowing channel of information from the second eye bolster activity in areas of the visual cortex responding to the initially treated eye.

An institutional review board required that Bennett work with adults in the follow-up study, but she thinks the therapy will work even better in younger patients who have not lost as many photoreceptors. She says the results "really bode well" for restoring meaningful vision to people with LCA and other forms of inherited blindness.

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Sight Seen: Gene Therapy Restores Vision in Both Eyes

Engineering a patients own immune cells to resist HIV could eliminate the need for lifelong antiretroviral therapies.

The immune cells of HIV patients can be genetically engineered to resist infection, say researchers. In a small study in humans, scientists report that by creating a beneficial mutation in T cells, they may be able to nearly cure patients of HIV.

In a study published in the New England Journal of Medicine on Wednesday, researchers report that they can use genome editing to re-create the rare mutations responsible for protecting about 1 percent of the population from the virus in infected patients. They report that some of the patients receiving the genome-modifying treatment showed decreased viral loads during a temporary halt of their antiretroviral drugs. In one patient, the virus could no longer be detected in his blood.

Zinc-finger nucleases are one of a few genome-editing tools that researchers use to create specific changes to the genomes of living organisms and cells (see Genome Surgery). Scientists have previously used genome-editing techniques to modify DNA in human cells and nonhuman animals, including monkeys (see Monkeys Modified with Genome Editing). Now, the NEJM study suggests the method can also be safely used in humans.

From each participating patient, the team harvested immune cells from the blood of the patients. They then used a zinc finger nuclease to break copies of the CCR5 gene that encodes for proteins on the surface of immune cells that are a critical entry point of HIV. The cells were then infused back into each patients bloodstream. The modification process isnt perfect, so only some of the cells end up carrying the modification. About 25 percent of the cells have at least one of the CCR5 genes interrupted, says Edward Lanphier, CEO of Sangamo Biosciences, the Richmond, California, biotech company that manufactures zinc finger nucleases.

Because the cells are a patients own, there is no risk of tissue rejection. The modified T cells are more resistant to infection by HIV, say the researchers.

One week after the infusion, researchers were able to find modified T cells in the patients blood. Four weeks after the infusion, six of the 12 patients in the study temporarily stopped taking their antiretroviral drugs so the researchers could assess the effect of the genome-editing treatment on the amount of the virus in the patients bodies. In four of these patients, the amount of HIV in the blood dropped. In one patient, the virus could no longer be detected at all. The team later discovered that this best responder had naturally already had one mutated copy of the CCR5 gene.

Patients who carry one broken copy of the CCR5 progress to AIDS more slowly than those who dont, says Bruce Levine, a cell and gene therapy researcher at the University of Pennsylvania School of Medicine and coauthor on the study. Because all of the cells in that best-responder patient already carried one disrupted copy of CCR5, the modification by the zinc finger nuclease led to T cells with no functional copies of the gene. That means the cells are fully resistant to HIV infection. The team is now working to increase the number of immune cells that end up carrying two broken copies of CCR5.

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In a small trial, researchers have successfully used gene therapy to boost the immune system of 12 patients with HIV to resist infection. They removed the patients' white blood cells to edit a gene in them, then infused them back into the patients. Some of the patients who showed reduced viral loads were off HIV drugs completely.

In fact, one of the patients showed no detectable trace of HIV at all after therapy. The researchers, who report their phase I study in the New England Journal of Medicine believe theirs is the first published account of using gene editing in humans.

The team included researchers from the University of Pennsylvania (Penn), PA, Albert Einstein College of Medicine, Bronx, NY, and Sangamo BioSciences, Richmond, CA, the company that developed the gene editing technology.

Carl H. June, senior author of the study and professor at Penn's Perelman School of Medicine, says:

"This study shows that we can safely and effectively engineer an HIV patient's own T cells to mimic a naturally occurring resistance to the virus, infuse those engineered cells, have them persist in the body, and potentially keep viral loads at bay without the use of drugs."

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Gene therapy used to block HIV without drugs

The approach involves using enzymes to destroy a gene in the immune cells of people with HIV, thereby increasing resistance to the virus

Scanning electron micrograph of a human T cell from the immune system of a healthy donor. Credit:NIAID/NIH - Wikimedia Commons

A clinical trial has shown that a gene-editing technique can be safe and effective in humans. For the first time, researchers used enzymes called zinc-finger nucleases (ZFNs) to target and destroy a gene in the immune cells of 12 people with HIV, increasing their resistance to the virus. The findings were published March 5 in The New England Journal of Medicine.

This is the first major advance in HIV gene therapy since it was demonstrated that the Berlin patient Timothy Brown was free of HIV, says John Rossi, a molecular biologist at the Beckman Research Institute of the City of Hope National Medical Center in Duarte, California. In 2008, researchers reported thatBrown gained the ability to control his HIV infectionafter they treated him with donor bone-marrow stem cells that carried a mutation in a gene calledCCR5. Most HIV strains use a protein encoded byCCR5as a gateway into the T cells of a hosts immune system. People who carry a mutated version of the gene, including Brown's donor, are resistant to HIV.

But similar treatment isnot feasible for most people with HIV: it is invasive, and the body is likely to attack the donor cells. So a team led by Carl June and Pablo Tebas, immunologists at the University of Pennsylvania in Philadelphia, sought to create the beneficialCCR5 mutation in a persons own cells, using targeted gene editing.

Personalized medicine The researchers drew blood from 12 people with HIV who had been taking antiretroviral drugs to keep the virus in check. After culturing blood cells from each participant, the team used a commercially available ZFN to target theCCR5gene in those cells. The treatment succeeded in disrupting the gene in about 25% of each participants cultured cells; the researchers then transfused all of the cultured cells into the participants. After treatment, all had elevated levels of T cells in their blood, suggesting that the virus was less capable of destroying them.

Six of the 12 participants then stopped their antiretroviral drug therapy, while the team monitored their levels of virus and T cells. Their HIV levels rebounded more slowly than normal, and their T-cell levels remained high for weeks. In short, the presence of HIV seemed to drive the modified immune cells, which lacked a functionalCCR5gene, to proliferate in the body. Researchers suspect that the virus was unable to infect and destroy the altered cells.

They used HIV to help in its own demise, says Paula Cannon, who studies gene therapy at the University of Southern California in Los Angeles. They throw the cells back at it and say, Ha, now what?

Long-term action In this first small trial, the gene-editing approach seemed to be safe: Tebas says that the worst side effect was that the chemical used in the process made the patients bodies smell bad for several days.

The trial isnt the end game, but its an important advance in the direction of this kind of research, says Anthony Fauci, director of the National Institute of Allergy and Infectious Diseases in Bethesda, Maryland. Its more practical and applicable than doing a stem-cell transplant, he says, although it remains to be seen whether it is as effective.

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Gene-Editing Technique Shown to Work as HIV Treatment