Proove Biosciences Will Present Clinical Research and Data at the American Academy of Pain Medicines 30th Annual …
Irvine, CA (PRWEB) March 06, 2014
Proove Biosciences, the leader Personalized Pain Medicine testing services, will participate and present clinical data and research at the American Academy of Pain Medicines 30th annual meeting this weekend, March 6-9, 2014. The meeting provides a comprehensive overview and review of current and cutting-edge pain medicine practice topics.
AAPM President and Proove Medical Advisory Board Member, Dr. Lynn Webster, will be moderating a workshop on safe opioid prescribing practices. The program will educate prescribers about eight evidence-based principles that will drastically reduce the number of unintentional overdose deaths from prescription medication.
"We are thrilled to be a part of the AAPMs Annual Meeting. As the premiere industry forum, we are excited to share and present our clinical data on how our Proove Genetic tests have been helping pain medicine professionals throughout the country improve the selection, dosing, and evaluation of medications. We will exhibit how Proove helps identify patients at risk for tolerance and misuse of medications, and how we have been creating efficiency within the healthcare system, while improving safety and decreasing risks associated with pain medicine therapies," stated Brian Meshkin, President of Proove Biosciences.
Proove will again be the only company presenting data on the genetics of pain medicine. During the poster session, Proove will be presenting data correlating genetic variations with co-occurring psychiatric disorders among chronic pain patients taking narcotics.
About the American Academy of Pain Medicine
The American Academy of Pain Medicine (AAPM) is the medical specialty society representing physicians practicing in the field of pain medicine. As a medical specialty society, the Academy is involved in education, training, advocacy, and research in the specialty of pain medicine.
About Proove Biosciences
Proove Biosciences is the leading Personalized Pain Medicine laboratory that provides proprietary genetic testing services to help physicians improve outcomes for patients and contain costs for insurers. With offices in Southern California and the Baltimore-Washington metropolitan area, the Company is the research leader investigating and publishing data on the genetics of pain medicine with clinical research sites across the United States. Physicians use Proove Biosciences testing to improve pain medicine selection, dosing, and evaluation of medications they prescribe. From a simple cheek swab collected in the office, Proove performs proprietary genetic tests in its CLIA-certified laboratory to identify patients at risk for misuse of prescription pain medications and evaluate their metabolism of medications. For more information, please visit http://www.proovebio.com or call toll free 855-PROOVE-BIO (855-776-6832).
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A Father's Genetic Quest Pays Off
A genetic mutation provides a clue to Hugh Rienhoff's daughter's undefined syndrome
Colston Rienhoff
Hugh Rienhoff says that his nine-year-old daughter, Bea, is a fire cracker, a tomboy and a very sassy, impudent girl. But in a forthcoming research paper, he uses rather different terms, describing her hypertelorism (wide spacing between the eyes) and bifid uvula (a cleft in the tissue that hangs from the back of the palate). Both are probably features of a genetic syndrome that Rienhoff has obsessed over since soon after Beas birth in 2003. Unable to put on much muscle mass, Bea wears braces on her skinny legs to steady her on her curled feet. She is otherwise healthy, but Rienhoff has long worried that his daughters condition might come with serious heart problems.
Rienhoff, a biotech entrepreneur in San Carlos, California, who had trained as a clinical geneticist in the 1980s, went from doctor to doctor looking for a diagnosis. He bought lab equipment so that he could study his daughters DNA himself and in the process, he became a symbol for the do-it-yourself biology movement, and a trailblazer in using DNA technologies to diagnose a rare disease (see Nature 449, 773776; 2007).
Talk about personal genomics, says GarySchroth, a research and development director at the genome-sequencing company Illumina in San Diego, California, who has helped Rienhoff in his search for clues. It doesnt get any more personal than trying to figure out whats wrong with your own kid.
Now nearly a decade into his quest, Rienhoff has arrived at an answer. Through the partial-genome sequencing of his entire family, he and a group of collaborators have found a mutation in the gene that encodes transforming growth factor-3 (TGF-3). Genes in the TGF- pathway control embryogenesis, cell differentiation and cell death, and mutations in several related genes have been associated with Marfan syndrome and LoeysDietz syndrome, both of which have symptomatic overlap with Beas condition. The mutation, which has not been connected to any disease before, seems to be responsible for Beas clinical features, according to a paper to be published in the American Journal of Medical Genetics.
Hal Dietz, a clinician at Johns Hopkins University School of Medicine in Baltimore, Maryland, where Rienhoff trained as a geneticist, isnt surprised that the genetic culprit is in this pathway. The overwhelming early hypothesis was that this was related, says Dietz, who co-discovered LoeysDietz syndrome in 2005.
Rienhoff had long been tapping experts such as Dietz for assistance. In 2005, an examination at Johns Hopkins revealed Beas bifid uvula. This feature, combined with others, suggested LoeysDietz syndrome, which is caused by mutations in TGF- receptors. But physicians found none of the known mutations after sequencing these genes individually. This was a relief: LoeysDietz is associated with devastating cardiovascular complications and an average life span of 26 years.
In 2008, Jay Flatley, chief executive of Illumina, offered Rienhoff the chance to sequence Beas transcriptome all of the RNA expressed by a sample of her cells along with those of her parents and her two brothers. After drilling into the data, Rienhoff and his collaborators found that Bea had inherited from each parent a defective-looking copy of CPNE1, a poorly studied gene that seems to encode a membrane protein. It looked like the answer.
But questions remained. The gene did not have obvious connections to Beas features, and publicly available genome data suggests that the CPNE1 mutations are present in about 1in1,000people an indication that there should be many more people like Bea.
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Clinics Offer Expensive Whole-Genome Tests for Undiagnosed Disorders
Two university-based clinics have debuted large programs that rely on sequencing to diagnose genetic disorders, including developmental disorders such as autism
Cancer.gov
Reprinted with permission fromSFARI.org, an editorially independent division of The Simons Foundation. (Find original story here.)
Over the past few years, teams of scientists have been finding genetic glitches related to a wide variety of disorders by sequencing exomes, the protein-coding portions of the genome. But these genetic tests are typically out of reach for people unless they enroll in research studies, and even then, theyre almost never privy to their individual results.
But that looks set to change: A few clinics are debuting large programs that rely on sequencing of exomes or even of whole genomes, and making the results directly available to individuals. For less than $10,000 each, the tests offer people with unexplained genetic disorders the chance to find the cause of their condition.
The first academic lab to offer clinical exome sequencing was the Whole Genome Laboratory at Baylor College of Medicine in Houston. Since November 2011, the lab has sequenced the exomes of some 1,700 individuals with undiagnosed conditions, including many children with developmental disorders. It now averages about 200 exomes a month.
"It's gone gangbusters," says Richard Gibbs, director of Baylor's Human Genome Sequencing Center, which helped establish the new lab. The researchers have pinpointed the genetic cause of about one-quarter of the 1,700 cases as mutations in known disease genes, he says.
Last week, the Harvard-affiliated Partners Healthcare Center in Boston launched a similar lab focused on sequencing whole genomes. And two private companies Ambry Genetics in Aliso Viejo, California, and GeneDx in Gaithersburg, Maryland have offered clinical exome sequencing since 2011.
Deciding which parts of the sequencing data should be divulged to individuals is far from straightforward. A few mutations are clearly associated with disease, but most are still tricky to interpret.
From a research perspective, however, the development is unequivocally exciting, experts say.
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Allele you need to know about Genetics – Video
Allele you need to know about Genetics
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Michael Parker – Moral Craft in the Genetics Clinic and Laboratory – Video
Michael Parker - Moral Craft in the Genetics Clinic and Laboratory
Parker #39;s talk will explore the moral world of the contemporary genetics profession at a key moment in its development. In particular, the talk will focus on ...
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Thanks to genetics, you are you
A genetics team from Hawaii is here on Guam for the week to meet with patients with genetic conditions.
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Building Better Genetics – CAM Ranches – Video
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Monica Coenraads and Dr. Neul discuss the genetics and nuances behind a Rett diagnosis – Video
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 – Video
Minecraft - Attack of The B-Team - Ep.9 : Advanced Genetics! w/ BdoubleO
Minecraft - Attack of The B-Team - Ep.9 : Advanced Genetics! w/ BdoubleO Leave a LIKE on this video for more! I want to have all the genes! Joined by Bdouble...
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Gene Therapy for HIV Delivers Hopeful Results – US News
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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Chemo drug helps HIV patients respond to Sangamo gene therapy
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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Gene Therapy Seems Safe, May Help Control HIV – ABC News
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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Localized Therapeutics – a localized photo-inducible gene therapy for arthritis – Video
Localized Therapeutics - a localized photo-inducible gene therapy for arthritis
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Dr. Max Gomez: HIV Gene Therapy – Video
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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Gene Therapy for Controlling HIV Shows Early Promise
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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Sight Seen: Gene Therapy Restores Vision in Both Eyes
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
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Can Gene Therapy Cure HIV?
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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Gene-Editing Technique Shown to Work as HIV Treatment
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 therapy used to block HIV without drugs
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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 Therapies Will Cure Many a Disease
See Inside
Gene therapy, once off to a rocky start, transforms medicine by getting at the root cause of many diseases
The Science Of The Next 150 Years: 50 Years in the Future
It is 2063. you walk into the doctor's office, and a nurse takes a sample of saliva, blood or a prenatal cell and applies it to a microchip the size of a letter on this page on a handheld device. Minutes later the device reads the test results. The multicolored fluorescence pattern on its display reveals the presence of DNA sequences that cause or influence any of 1,200-plus single-gene disorders. Fortunately, regulatory authorities have approved a cure for each one of these diseases: gene therapy.
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Personalized Medicine and Smart Drugs – Video
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Scientists Chafe at Restrictions on New Stem Cell Lines
The California Institute for Regenerative Medicine is rethinking its rules in the wake of a recent breakthrough involving the creation of stem cell lines from a cloned human embryo
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The announcement last month of a long-awaited breakthrough in stem-cell research the creation of stem-cell lines from a cloned human embryo has revived interest in using embryonic stem cells to treat disease. But US regulations mean that many researchers will be watching those efforts from the sidelines.
The US National Institutes of Health (NIH), which distributes the majority of federal funding for stem-cell research, prohibits research on cells taken from embryos created solely for research a category that includes the six stem-cell lines developed by Shoukhrat Mitalipov, a reproductive-biology specialist at the Oregon Health and Science University in Beaverton, and his colleagues. The team used cloning techniques to combine a donor cell with an unfertilized egg whose nucleus had been removed, creating a self-regenerating stem-cell colony that is genetically matched to the cell donor.
Mitalipovs cell lines are also off limits to researchers funded by the California Institute for Regenerative Medicine (CIRM), which was created in part to support stem-cell work that is restricted by the NIH. CIRM funds cannot be used for studies that pay women for their eggs or rely on cell lines produced using eggs from paid donors. That rules out Mitalipovs lines, because his team paid egg donors US$3,0007,000 each, says Geoffrey Lomax, senior officer to the standards working group at CIRM, which is based in San Francisco. That amount is above and beyond any out-of-pocket costs to donors, he says.
The end result, says Mitalipov, is that a dozen or so universities are struggling to negotiate material transfer agreements to receive the new cell lines without running afoul of CIRM or the NIH. Interest in the new cell lines is high, especially since the identification of errors in images and figures in Mitalipovs research paper shortly after its publication in Cell. But regulations would require laboratories to use only dedicated, privately funded equipment to study the new cells, a condition that only a fewresearchers such as George Daley, a stem-cell expert at Boston Childrens Hospital in Massachusetts will be able to meet.
That concerns Daley, who calls the NIH stem-cell policy a frustrating limitation that will preclude federal dollars being used to ask many important questions about how Mitalipovs cell lines compare with induced pluripotent stem cells (iPS), which are created by reprograming adult cells to an embryonic state. Most labs will take the path of least resistance and continue working with iPS cells unless someone shows that there is a clear and compelling reason to change course, Daley says.
Mitalipov also worries that his cell lines wont be sufficiently analyzed, which he says could hamper efforts to understand how epigenetic changes modifications to chromosomes that determine how genes are expressed affect stem cells' ability to transform into a wide array of mature cell types. We just dont have that much expertise at looking at all aspects of epigenetics, he says.
But some scientists say that the impact of US stem-cell restrictions is overestimated. Alexander Meissner, a developmental biologist at the Harvard Stem Cell Institute in Cambridge, Massachusetts, says Mitalipov's cell lines will not reveal much about how stem cells transform. That work can be done only with eggs that are easy to come by, allowing scientists to examine the reprograming process at many points. In practical terms, that means relying on eggs from mice instead of humans. Everything is over by time you derive those cell lines, he says of Mitalipovs cells. There is no signature that would tell you what has happened. Its the wrong species.
In the meantime, CIRM is re-examining the rules that govern the research its supports. The institute is not likely to alter the restrictions against funding studies that pay cell donors, but it might overturn the rules against using cell lines produced in such studies, Lomax says. The original policy was set in 2006 to address concerns that arose in the wake of fraud and ethical violations by Woo Suk Hwang, then a researcher at Seoul National University.
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Scientists Chafe at Restrictions on New Stem Cell Lines
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