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    News and Articles on Gene Therapy

    Archives: Gene Therapy

    A look into the culture of performance enhancing drugs  Jul 18, 2008
    The one thing we explore in the movie is gene therapy, which could be termed 'gene doping. If you look at gene doping, it's like where is it going to end because you can change someone's genetics and basically tell their body to make more human growth hormone every single day by injecting them with a single virus that you only have to do once. (Juneau Empire)

    IAVI statement in response to NIAID decision not to move forward with PAVE 100 HIV Vaccine Trial  Jul 18, 2008
    "The AIDS vaccine field does not suffer from a lack of ideas about how to move forward. Yes, significant progress remains to be accomplished before an effective, preventive vaccine is within our grasp. But researchers are working apace on such projects as solving the HIV neutralizing antibody problem, determining how elite controllers remain disease-free for decades, determining how live-attenuated SIV vaccines work, developing replicating vectors for use in AIDS vaccines, developing AIDS... (EurekAlert!)

    AMT Appoints Director Global Marketing and Sales  Jul 17, 2008
    AMSTERDAM, The Netherlands, July 17 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today the appointment of Mrs. Janneke de Wal, M.D., as Director Global Marketing and Sales ... About Amsterdam Molecular Therapeutics AMT has a unique gene therapy platform that to date appears to circumvent many if not all of the obstacles that have prevented gene therapy from becoming a mainstay of clinical medicine. (PR Newswire)

    DNA 'Palindromes' Linked To Disease  Jul 17, 2008
    (June 1, 2007) Researchers say they have the ability to study the potential cause of genomic instability in sporadic cancers using a recombinant adeno-associated virus, a virus commonly used for gene therapy. (Mar. (Science Daily)

    AMT Starts Collaboration With St. Jude Children's Research Hospital on Gene Therapy for Hemophilia B. Company Accesses Exclusive Commercial Rights to Final Therapy  Jul 15, 2008
    AMSTERDAM, The Netherlands, July 15 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today announced the start of a collaboration with St. Jude Children's Research Hospital in Memphis, Tennessee, USA, on the development of a gene therapy treatment for Hemophilia B. Under the deal, AMT will receive the exclusive commercial rights to the final product. The combination of this gene with AMT's proprietary adeno-associated virus... (PR Newswire)

    Researchers Locate And Image Prostate Cancer As It Spreads To Lymph Nodes  Jul 15, 2008
    (May 23, 2001) Researchers at UCLA s Jonsson Cancer Center have shown for the first time that immunotherapy delivered via gene therapy may prove to be a potent weapon in the fight against locally advanced. . (Science Daily)

    Gene therapy opens new frontier in fight against Huntington's  Jul 15, 2008
    Gene therapy opens new frontier in fight against Huntington's - Yahoo ... Gene therapy opens new frontier in fight against Huntington's ... PARIS (AFP) - French researchers on Tuesday said that gene therapy, tested on lab animals, showed promise in combatting the tragic neurogenerative disease known as Huntington's. (Yahoo News -- Top Stories)

    Newsweek: Good scents boost mood  Jul 14, 2008
    Gilbert even speculates that gene therapy will one day help the elderly, whose sense of smell is diminished. If that day ever comes, it will be like recovering a whiff of youth. (MSNBC -- Health)

    First DNA Molecule Made Almost Entirely Of Artificial Parts  Jul 8, 2008
    The finding could lead to improvements in gene therapy, futuristic nano-sized computers, and other high-tech advances, they say ... The discovery could be used in the fields of gene therapy and other futuristic high-tech advances, such as nano-sized computers. (Science Daily)

    Chemists claim 1st almost all artificial DNA molecule  Jul 8, 2008
    BEIJING, July 8 (Xinhuanet) -- Japanese chemists say they have created the world's first DNA molecule made almost entirely of artificial parts, which could lead to improvements in gene therapy, nano-sized computers and other high-tech advances. Scientists have tried for years to develop artificial versions of DNA in order to take advantage of its amazing information storage capabilities. (Xinhuanet, China)

    A New President at the Head of the Genethon Laboratory  Jul 7, 2008
    Genethon is the promoter of the first gene therapy trial in humans for gamma-sarcoglycan deficiency. It possesses two clinical batch production units for vectors and is engaged in the development of a pre-industrial scale BMP production site to go onstream in 2010. (Canada Newswire)

    CFTR and Wnt/beta-catenin signaling in lung development  Jul 7, 2008
    Adenoviruses containing full-length CFTR, a short anti-sense CFTR gene fragment, or a reporter gene as control were used in an intra-amniotic gene therapy procedure to transiently modify CFTR expression in the fetal lung. Results. (BioMed Central)

    Artificial DNA can power future comps  Jul 7, 2008
    The finding could lead to improvements in gene therapy, futuristic nano-sized computers, and other high-tech advances, the researchers say. DNA, popularly illustrated as a double helix, holds the blueprints of life and controls what every living organism becomes and how it functions. (India Times, India -- Health/Science)

    10,000 people in world-first cerebral palsy study  Jul 3, 2008
    "In the future, gene therapy may allow doctors to alter the aberrant genes in a mother or fetus, or specific drugs could be used to counter the effect of genetic mutations and ultimately prevent a child from developing cerebral palsy before birth. "Knowledge of a patient's genetic makeup and tailored administration of anti-inflammatory drugs before and during pregnancy may be possible. Immunisation against viral infections also may be a future option when this preventative therapy is available,"... (EurekAlert!)

    Introgen stock rises as company seeks FDA approval of cancer drug  Jul 1, 2008
    "This is an important milestone in the clinical application of gene therapy for cancer patients," says Dr. Jack Roth, inventor of Advexin and professor at in Houston. "With the use of p53 biomarkers, Advexin will provide more effective and less toxic treatment for head and neck cancer patients who have limited treatment options.". (Austin Business Journal, TX)

    The Good And The Bad Of A Potential Alzheimer's Target  Jun 30, 2008
    26, 2003) A molecule that naturally degrades a protein linked to Alzheimer's disease appears to reduce the levels of that protein by nearly 50 percent when delivered by gene therapy, researchers at the. . (Science Daily)

    Thompson: Biotech still an achievable dream  Jun 29, 2008
    Your Connection to the. Web Search powered by YAHOO. (Athens Banner-Herald)

    New Research May Lead To Safer, More Effective Gene Therapy  Jun 28, 2008
    ScienceDaily (June 27, 2008) The potential of gene therapy has long been hampered by the risks associated with using viruses as vectors to deliver healthy genes, but a new University of Georgia study helps bring scientists closer to a safe and efficient gene delivery method that doesn t involve viruses ... Gene therapy involves replacing abnormal, disease-causing genes with normal genes ... 17, 2006) MIT scientists are reporting synthesis and laboratory tests of a promising new group of... (Science Daily)

    The tiniest things  Jun 27, 2008
    More promising but also more distant and still unproven are approaches like somatic gene therapy, said Dr. Salvatore DiMauro, a mitochondrial specialist at The Neurological Institute of New York and Columbia University ... The track record for gene therapy is mixed; most efforts thus far have fallen short of expectations or failed altogether. (San Diego Union-Tribune)

    Biotech: Repairing damage to brain may be nearer  Jun 26, 2008
    Previous research trying to produce neurons from embryonic stem cells has resulted in the creation of glial cells, a type of brain cell that provides support to neurons, said Steven Goldman, a neurologist who is chief of the division of Cell and Gene Therapy at the University of Rochester Medical Center in New York. Goldman said Lipton's team has created a very general group of neurons. (San Diego Union-Tribune)

    Math Could Help Cure Leukemia  Jun 24, 2008
    ScienceDaily (June 23, 2008) When kids complain that math homework won't help them in real life, a new answer might be that math could help cure cancer. In a recent study that combined math and medicine, researchers have shown that patients with chronic myelogenous leukemia (CML) may be cured of the disease with an optimally timed cancer vaccine, where the timing is determined based on their own immune response. (Science Daily)

    Stem Cells Correct Defect In Child’s Fatal Skin Disease  Jun 24, 2008
    Her laboratory performed the first genetic studies on the Liao family and determined the exact molecular basis for their individual case of RDEB. She has studied several gene therapy approaches for RDEB in the past before turning her attention to circulating stem cells. This is the first time physicians have approached the treatment of RDEB or any similar skin disease, from a systemic perspective, using marrow-derived stem cells as a means to replace the missing protein, collagen type VII,... (Science Daily)

    Malaccas bio-city to provide 5,000 jobs  Jun 20, 2008
    ABM, a joint venture between Actis Biologics and Malaysian investors, has in-licensed technology platforms in areas such as gene therapy and vaccines. The BioCity was launched Thursday at the 2008 BIO International Convention here by Mohd Ali. (The Star Online, Malaysia)

    First gene therapy for heart failure offered at NewYork-Presbyterian/Columbia  Jun 19, 2008
    Physician-scientists are setting out to answer that question in a first-ever clinical trial of gene therapy to treat severe heart failure ... Patients enrolled in the multicenter CUPID trial (Calcium Up-Regulation by Percutaneous Administration of Gene Therapy in Cardiac Disease) will undergo a minimally invasive cardiac catheterization procedure that will introduce a specially engineered gene that stimulates production of an enzyme necessary for the heart to pump more efficiently ... Gene... (EurekAlert!)

    Gene Therapy Improves Survival And Quality Of Life Of Dogs With Cancer: Could It Also Help Humans?  Jun 17, 2008
    Gene Therapy Improves Survival And Quality Of Life Of Dogs With Cancer: Could It Also Help Humans ... Gene Therapy Improves Survival And Quality Of Life Of Dogs With Cancer: Could It Also Help Humans ... 17, 2008) A new gene therapy has helped pet dogs with cancer live longer and could potentially improve the quality of life and survival of people with cancer, said the therapy's developers. (Science Daily)

    Eating Fatty Fish Can Help Aging Eyes  Jun 13, 2008
    Early research is showing some promise for gene therapy designed to stop the wet form of the disease in its tracks. . (WebMD)

    AMT Prepares for Submission of Marketing Authorization Dossier for AMT-011 (Glybera(R))  Jun 13, 2008
    AMSTERDAM, June 13 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today that it has concluded enrollment and treated the last patient in the pivotal trial for it's lead product AMT-011 (Glybera(R)) for Hyperlipoproteinemia, a seriously debilitating and potentially lethal disease ... AMT-011 is a gene therapy product designed to insert a healthy gene into the patient's body to replace the defective gene ... About... (PR Newswire)

    Carbon Monoxide May Reduce Skeletal Muscle Injuries, Mouse Study Suggests  Jun 12, 2008
    5, 2006) University of Pittsburgh School of Medicine researchers have successfully used gene therapy to accelerate muscle regeneration in experimental animals with muscle damage. They suggest that this. (Science Daily)

    WADA concerned with possibility of gene doping  Jun 12, 2008
    Although there's no hard evidence that athletes are doing it, WADA is concerned about the potential for gene doping - the illegal use of gene therapy, which typically alters a person's DNA to fight diseases - to enhance performance ... WADA vice president Arne Ljungqvist said scientists working on gene therapy are being approached by sports figures interested in performance enhancement ... "We need to make sure that athletes know the dangers associated with these technologies and, for those who... (SportsIllustrated.CNN -- More)

    Plainsboro resident tapped for prominent post  Jun 12, 2008
    His laboratory and clinical studies focus on tumor immunology, gene therapy and the development of vaccines for cancer treatment ... He has written more than 100 scientific publications and is the editor of "Gene Therapy of Cancer.". (NJ.com -- Times)

    Inhalable Form Of Gene-therapy Takes Aim At Lung Cancer And Inflammatory Lung Disease  Jun 11, 2008
    10, 2008) A new inhalable form of gene therapy -- based on technology recognized in the 2006 Nobel medicine prize, shows increasing promise for treating lung cancer, infectious diseases and inflammatory lung disease, scientists have concluded after an exhaustive review of worldwide research on the topic ... 17, 2005) According to a University of Pittsburgh study presented at the 47th Annual Meeting of the American Society for Therapeutic Radiology and Oncology, Oct. 16 to 20, gene therapy could... (Science Daily)

    How The Brain Can Protect Against Cancer  Jun 11, 2008
    (May 3, 1999) NIH scientists are attacking chronic pain with a novel form of gene therapy that targets the spinal cord. Though still in the animal testing stage, this approach has overcome one of the major. (Science Daily)

    VERDUCCI: The declines of old vets, draft trends and more  Jun 11, 2008
    Another well-connected clubhouse source gave me this interpretation of what's going on: the climate has definitely calmed down, but in two years or so you'll see bodies begin to blow up again as the watchdog atmosphere wanes and as new advances in performance enhancers, including gene therapy, are discovered by early adapters. Despite your assertion that , isn't it premature to assign "top" status to young pitchers without knowing their future contributions. (SportsIllustrated.CNN)

    AMT Makes Important Advance in the Development of its Gene Therapy for Hemophilia B -Company Accesses Technology to Prevent Immune Response  Jun 10, 2008
    AMSTERDAM, The Netherlands, June 10 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today announced that it obtained a license from 'TIGET' San Raffaele Telethon Institute For Gene Therapy, Italy, to use their micro-RNA technology to prevent immune responses against gene therapy for Hemophilia B. This technology enables AMT to accelerate the development of AMT-060 (AMT's AAV-based gene therapy for this seriously... (PR Newswire)

    Health InfoCenter  Jun 10, 2008
    To read the study on using gene therapy for Leber's Congenital Amaurosis. For more on carboxitherapy. (WCAX.com, VT)

    Gene Therapy Involving Antibiotics May Help Patients With Usher Syndrome  Jun 9, 2008
    8, 2008) A new approach to treating vision loss caused by Type 1 Usher syndrome (USH1), the most common condition affecting both sight and hearing, will be unveiled by a scientist at the annual conference of the European Society of Human Genetics June 3, 2008. Ms Annie Rebibo Sabbah, from the Genetics Department of the Rappaport Faculty of Medicine, Technion, Haifa, Israel, will tell the conference that preliminary results using a class of drugs called aminoglycosides, commonly used as... (Science Daily)

    Riken Makes Developments in Alzheimer's Disease Research  Jun 9, 2008
    In the laboratory, they treated an Alzheimer's disease model mouse with a gene therapy that boosted the activity of Neprilysin by 10 times, and as a result, were able to suppress the accumulation of the amyloid peptide. Riken is currently conducting bio-chemical and histological analysis of the brain using an Alzheimer's disease model mouse. (JCN Network, Japan)

    Saturday marks first Batten Disease Day  Jun 7, 2008
    They have also helped fund a trial for gene therapy for the infant form of the disease. The federal government provides 3 million a year for Batten Disease research, Johnston said, but that is not nearly enough since the disease encompasses 10 separate genetic disorders. (Taylor Daily Press, TX)

    New Mouse Model Mimics Hyperglycemia, Aids In Diabetes Research  Jun 7, 2008
    2, 2006) Using state-of-the-art gene therapy techniques, University of Pittsburgh investigators have successfully prevented the onset of elevated blood sugar, or hyperglycemia, in diabetes-prone mice by. (Apr. (Science Daily)

    Locomotor Training Restores Walking Function In Child With Spinal Cord Injury  Jun 6, 2008
    15, 1997) Researchers from the UCSD School of Medicine report the first successful use of gene therapy to achieve partial recovery from spinal cord. . (Science Daily)

    RIKEN Identifies Potential Drug for Alzheimer's : Video Report  Jun 6, 2008
    In the laboratory, they treated an Alzheimer's disease model mouse with a gene therapy that boosted the activity of Neprilysin by 10 times, and as a result, were able to suppress the accumulation of the amyloid beta-peptide. Somatostatin can act to reinforce memory retention, so it has the potential to be both a treatment and preventive drug for Alzheimer's disease. (JCN Network, Japan)

    QIAGEN to Present At the Goldman Sachs 29th Annual Global Healthcare Conference  Jun 6, 2008
    Such uncertainties and risks include, but are not limited to, risks associated with management of growth and international operations (including the effects of currency fluctuations and risks of dependency on logistics), variability of operating results, the commercial development of the applied testing markets, clinical research markets and proteomics markets, women's health/HPV testing markets, nucleic acid-based molecular diagnostics market, and genetic vaccination and gene therapy markets,... (Primezone Releases)

    Silver Rings To Treat Arthritis Symptoms Validated  Jun 5, 2008
    14, 2002) Northwestern University researchers have reported the first successful use of interleukin-13 (IL-13) cytokine gene therapy to treat and prevent rheumatoid arthritis in an animal. (Apr. (Science Daily)

    'Cyborg Engineering' For Coronary Bypass Grafts  Jun 4, 2008
    25, 2000) A gene therapy that packs a one-two punch may eventually lead to new treatments designed to keep arteries from re-blocking following bypass surgery, British researchers reported in Circulation. (Feb. (Science Daily)

    Drug Found To Stimulate Immune System In Patients With Prostate Cancer  Jun 4, 2008
    (May 23, 2001) Researchers at UCLA s Jonsson Cancer Center have shown for the first time that immunotherapy delivered via gene therapy may prove to be a potent weapon in the fight against locally advanced. (Jun. (Science Daily)

    Fracture - Hands On  Jun 3, 2008
    Despite the two factions taking very different routes of 'evolution', one using cybernetic enhancements and the other gene therapy, both have access to terrain deformation weaponry, which affects the very ground of the battlefield. Whilst early demos of Fracture were set in the future dustbowl of the San Francisco Bay area, this latest unveiling focused on a storm-drenched desert, offering a very different (and more sci-fi) atmosphere. (Total Video Games)

    Artificial Virus Carries Drugs Into Cancer Cells  Jun 2, 2008
    This trait is now being exploited for gene therapy, in which genes are brought into the cells of a patient to treat genetic diseases or genetic defects ... Natural viruses are extremely effective at transporting genes into cells for gene therapy; their disadvantage is that they can initiate an immune response or cause cancer ... Known as small interfering RNAs (siRNA), these sequences represent a promising approach to gene therapy. (Science Daily)

    Targeted Genetics' reports results on drug trial; shares plummet  Jun 1, 2008
    Targeted Genetics described its clinical study of its test of tgAAC94 in patients with inflammatory arthritis at the 11th annual meeting of the American Society of Gene Therapy in Seattle. In a morning press release, H. Stewart Parker, president and CEO, was quoted as saying, "although these results do not provide statistical evidence of efficacy, we believe they suggest that tgAAC94 has the potential to improve disease symptoms that are refractory to other therapies.". (Puget Sound Business Journal, WA)

    Cancer drug developed by Introgen founder meets with clinical success  Jun 1, 2008
    A gene therapy developed at the and licensed by is the first to succeed in a phase III clinical trial in the United States. The announcement was made this week at the American Society of Gene Therapy annual meeting in Boston. (Austin Business Journal, TX)

    NYP/Weill Cornell physician-scientists present latest cancer findings at ASCO meeting  Jun 1, 2008
    Weill Cornell physician-scientists have been responsible for many medical advances -- from the development of the Pap test for cervical cancer to the synthesis of penicillin, the first successful embryo-biopsy pregnancy and birth in the U.S., the first clinical trial for gene therapy for Parkinson's disease, the first indication of bone marrow's critical role in tumor growth, and, most recently, the world's first successful use of deep brain stimulation to treat a minimally-conscious... (EurekAlert!)

    Clarification on Press Release Issued Earlier Today: Targeted Genetics Confirms Positive, Interim Phase 1/2 Study Findings  May 31, 2008
    D., vice president of therapeutic development for Targeted Genetics, presented the data in an oral presentation at the 11th Annual Meeting of the American Society of Gene Therapy (ASGT) in Seattle. To clarify, the data demonstrated that a 30 percent decrease in the global visual analog scale (VAS) was experienced by 21/50 (42 percent) of subjects and 3/16 (19 percent) placebo subjects 12 weeks after the first injection. (PR Newswire)

    AMT Announces Positive Interim Clinical Data on its Lead Product AMT-011  May 30, 2008
    AMSTERDAM, May 30 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today announced that positive interim data from its pivotal clinical trial with AMT's lead product Glybera(R) (AMT-011) were presented by the Principal Investigator, professor Daniel Gaudet, at the American Society of Gene Therapy Annual Meeting in Boston on May 29th ... Study data Glybera(R) is a gene therapy product that corrects the genetic defect in... (PR Newswire)

    Oncolytics Biotech Inc. Collaborators to Present Reovirus Research at ASGT Meeting  May 30, 2008
    CALGARY, May 30 /CNW/ - Oncolytics Biotech Inc. (TSX: ONC, NASDAQ: ONCY) announced today that two presentations covering preclinical work using the reovirus are scheduled to be delivered by Dr. Richard Vile of the Mayo College of Medicine, Rochester, Minnesota, at the American Society of Gene Therapy (ASGT) annual meeting in Boston, MA. The meeting runs from May 28 to June 1, 2008. A poster presentation entitled "Combination Immunotherapy and Oncolytic Virotherapy for the Treatment of Malignant... (Canada Newswire)

    Gene Therapy Increases Survival For End-stage Head And Neck Cancer  May 30, 2008
    ScienceDaily (May 29, 2008) A gene therapy invented at The University of Texas M. D. Anderson Cancer Center is the first to succeed in a U.S. phase III clinical trial for cancer, as announced May 28 at the American Society of Gene Therapy annual meeting in Boston ... Roth's lab has been developing gene therapy for cancer since 1990 ... Roth found little interest in gene therapy from established pharmaceutical companies, so in 1994 he co-founded Introgen working through M. D. Anderson's Office of... (Science Daily)

    Q&A: The Man Behind Embryonic Stem Cells  May 29, 2008
    People thought gene therapy would be here, and it hasn't happened. Nobody ever predicted the genome project would be done at this point. (Forbes -- Business)

    New Cheaper Method For Mapping Disease Genes  May 28, 2008
    28, 2000) Gene therapy which aims to replace "bad" genes with useful ones has yet to live up to its promise largely because of problems delivering genes to the right place in the body. . (Science Daily)

    AMT's Cooperative Research and Development Agreement With NIH to Boost Production Capacity  May 26, 2008
    AMSTERDAM, The Netherlands, May 26 /PRNewswire-FirstCall/ -- - Production Increase Brings Large Disease Areas within Reach Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today announced that it obtained a Cooperative Research and Development Agreement from the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health (NIH), Bethesda, Maryland, that promises to substantially enhance the power of AMT's gene therapy... (PR Newswire)

    'Intrabody' can mop up mutant protein in Huntington's disease model  May 26, 2008
    Delivering the intrabody to brain tissues in people would be a formidable challenge, because it would require some form of gene therapy. However, it may be possible to use information about the intrabody's structure to find drugs that mimic its effects, Li says. (EurekAlert!)

    Gene Therapy  May 24, 2008
    Search post-gazette NOW. Do You Even Watch the Game. (Pittsburgh Post-Gazette, PA)

    Gene Therapy: Oral Gene Delivery System For Inflammatory Bowel Disease Works, Study Shows  May 23, 2008
    The article discusses the effectiveness of oral interleukin-10 or IL-10 (anti-inflammatory protein molecules) gene therapy for the treatment of IBD. The findings indicate that upon oral administration of NiMOS, transfection and local expression of IL-10 can not only suppress the levels of pro-inflammatory cytokines, but can also increase body weight, restore colon length and weight, and suppress inflammatory tissue response ... Over the course of eight days of oral gene therapy treatment, the... (Science Daily)

    Neural Cell Transplants May Help Those With Parkinson's Disease  May 23, 2008
    "There are currently a number of clinical trials underway using direct gene therapy approaches to deliver potent trophic factors throughout the basal ganglia.". Emborg and colleagues report that hNPCs genetically modified to over-express GDNF were able to survive transplant and produced GDNF for three months, and that functional recovery in test animals increased while no obvious negative side effects from the transplant procedure were observed. (Science Daily)

    AMT Accesses Technology for Treatment of Duchenne Muscular Dystrophy  May 22, 2008
    AMSTERDAM, The Netherlands, May 22 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today announced that it obtained a license from La Sapienza University in Rome, Italy, to their advanced small nuclear RNA (snRNA)-based exon-skipping technology for the treatment of Duchenne muscular dystrophy (DMD). The combination with AMT's proprietary adeno-associated virus (AAV) gene therapy platform potentially makes up a long-term... (PR Newswire)

    Improved Gene Therapy Agent Is 30 Times More Efficient Than Current One  May 22, 2008
    ScienceDaily (May 21, 2008) Replacing one amino acid on the surface of a virus that shepherds corrective genes into cells could be the breakthrough scientists have needed to make gene therapy a more viable option for treating genetic diseases such as hemophilia, University of Florida researchers say ... Reporting in the journal Proceedings of the National Academy of Sciences May 19, UF geneticists say they have developed a new version of the adeno-associated virus used in gene therapy that works... (Science Daily)

    Blood-clotting Protein Modified For People With Hard-to-treat Hemophilia  May 22, 2008
    ScienceDaily (May 21, 2008) Pathologists at The University of Texas Health Science Center at Houston have developed a chemically modified protein that may help people with a hard-to-treat form of a genetic bleeding disorder known as Hemophilia A.. With a shortage of the blood-clotting protein Factor VIII (FVIII), people with Hemophilia A typically receive injections of FVIII derived from plasma or produced synthetically to control potentially life-threatening episodes of bleeding. (Science Daily)

    Gene Therapy May Treat Erectile Dysfunction  May 22, 2008
    TUESDAY, May 20 (HealthDay News) -- Gene therapy may be a way to treat erectile dysfunction in men who do not respond to pills such as Viagra, researchers report. Maxi-K gene therapy is a gene transfer that improves erectile dysfunction. (MEDLINEplus)

    Gene Therapy for Retinal Degeneration  May 22, 2008
    In this issue of the Journal, two groups of investigators Bainbridge et al.1 and Maguire et al.2 describe the first results of separate clinical trials investigating the short-term safety and preliminary efficacy of gene therapy for Leber's congenital amaurosis. Both groups present short-term data (12 months and 5 months, respectively) on three patients with Leber's congenital amaurosis in each study; the patients were enrolled in trials of recombinant adeno-associated viral delivery of the... (New England Journal of Medicine)

    Effect of Gene Therapy on Visual Function in Leber’s Congenital Amaurosis  May 22, 2008
    NEJM -- Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis ... Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis. (New England Journal of Medicine)

    Dr. Gupta: The Prognosis for Kennedy  May 22, 2008
    There is also a type of gene therapy that injects custom-made viruses into tumor cells, causing them to die or at least become more susceptible to radiation. These aren't great choices, which means the Senator may have to rely as much on his own strength and optimism as on modern medicine. (Time.com)

    Frequently asked questions about MPS  May 21, 2008
    Researchers are hoping gene therapy will offer a cure for MPS someday. Comments. (San Francisco Chronicle)

    Scientists 'paint' viruses to track their fate in the body  May 21, 2008
    This breakthrough should give a much-needed boost to the efficiency of some forms of gene therapy, help track and treat viral disease and evolution, improve the efficiency of vaccines, and ultimately allow health care professionals track the movement of viral infections within the body. Specifically, the new method should make it easier to track and treat infectious diseases such as HIV/AIDS, influenza, hepatitis C, and dengue fever. (EurekAlert!)

    A WISH TO WALK: Part III: 'I can't believe that it couldn't happen'  May 20, 2008
    Gene therapy carries the potential to provide the injured spinal cord with the specific gene products, or proteins, that it needs to promote functional recovery. Gene therapy is not a current treatment for spinal cord injuries but is being studied with animal models of spinal cord injury. (Orangeburg Times and Democrat, SC)

    Vaccine Triggers Immune Response, Prevents Alzheimer's In Mice  May 20, 2008
    The Rochester scientists reported the findings in an article in the May issue of Molecular Therapy, the journal of The American Society of Gene Therapy. "Our study demonstrates that we can create a potent but safe version of a vaccine that utilizes the strategy of immune response shaping to prevent Alzheimer's-related pathologies and memory deficits," said William Bowers, associate professor of neurology and of microbiology and immunology at the Medical Center and lead author of the article. (Science Daily)

    UF researchers develop improved gene therapy agent  May 20, 2008
    Replacing one amino acid on the surface of a virus that shepherds corrective genes into cells could be the breakthrough scientists have needed to make gene therapy a more viable option for treating genetic diseases such as hemophilia, University of Florida researchers say. Reporting in the journal Proceedings of the National Academy of Sciences today (May 19), UF geneticists say they have developed a new version of the adeno-associated virus used in gene therapy that works about 30 times more... (EurekAlert!)

    Gene Drugs For Untreatable Diseases  May 15, 2008
    Previous attempts by other drug companies to harness gene therapy have, at times, had disastrous results. In one trial in 1999, an 18-year-old patient died; in a separate trial in France, a child developed leukemia. (Forbes -- Technology)

    Gene Therapy Found to Slow Progression of Fatal Brain Condition  May 15, 2008
    An experimental gene therapy had positive effects in slowing the progression of Batten disease, or Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL), a team of New York-Presbyterian Hospital/Weill Cornell Medicine Center physician-scientists showed ... The genes are incorporated within the genetic material of the cells, which are then able to produce a protein that is deficient in Batten disease, Crystal reported in a paper entitled Treatment of Late Infantile Neuronal Ceroid... (eFluxMedia)

    Gene Therapy Clinical Trial Yields Promising Results For Batten Disease  May 15, 2008
    ScienceDaily (May 14, 2008) Promising results from a team of NewYork-Presbyterian Hospital/Weill Cornell Medical Center physician-scientists show that gene therapy is both safe and effective at slowing the progression of Batten disease, or Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL), a rare, genetic, degenerative neurological disorder that usually becomes fatal in children by the age of 8 to 12 ... Dr. Crystal is a world leader and pioneer in the use of gene therapy to treat a number... (Science Daily)

    Gene Therapy Shows Promise in Rare Brain Disease  May 15, 2008
    WASHINGTON (Reuters) - An experimental gene therapy treatment appears to have helped eight children with a rare and incurable neurological disorder, although it may have been responsible for the death of one, researchers reported on Tuesday ... Like all forms of gene therapy, the hope is that the mutant cells will take up the new gene and start working normally ... "The disease is caused by mutations in the CLN2 (ceroid lipofuscinosis, neuronal 2) gene," Crystal and colleagues wrote in their... (MEDLINEplus)

    ASK THE EXPERTSHow does gene therapy work?  May 14, 2008
    How does gene therapy work ... How does gene therapy work ... and president of the American Society of Gene Therapy, responds. (Scientific American)

    Subsc00001174ribe  May 14, 2008
    Subscription Step 1 - Product Selection. Select if you are buying online access for multiple users. (Nature News Service)

    Arthritis Is A Potential Barrier To Physical Activity For Adults With Diabetes  May 11, 2008
    ScienceDaily (May 10, 2008) People with diagnosed diabetes are nearly twice as likely to have arthritis, and the inactivity caused by arthritis hinders the successful management of both diseases, according to a new Morbidity and Mortality Weekly Report (MMWR) study released May 8 by the Centers for Disease Control and Prevention (CDC). This is one of the first studies of its kind to look at the relationship between arthritis and diabetes and the outcomes associated with physical activity. (Science Daily)

    Suspected Cause Of Type 1 Diabetes Caught 'Red-handed' For The First Time  May 11, 2008
    2, 2006) Using state-of-the-art gene therapy techniques, University of Pittsburgh investigators have successfully prevented the onset of elevated blood sugar, or hyperglycemia, in diabetes-prone mice by. (May 7, 2008) Researchers in Maine report a new explanation for the mysterious link between consumption of cows' milk protein in infant formula early in life and an increased risk of later developing Type-1. (Science Daily)

    Skin Flaps Deliver Cancer-fighting Therapy, Study Reveals  May 10, 2008
    ScienceDaily (May 9, 2008) Using gene therapy, plastic surgeons have delivered cancer fighting proteins through skin flaps placed on cancerous tumors on rats with a 79 percent reduction in tumor volume, according to a study in the May issue of Plastic and Reconstructive Surgery, the official medical journal of the American Society of Plastic Surgeons ... "This new technique may allow us to reprogram skin flaps, using gene therapy, to provide a blueprint for anti-tumor agents like Interleukin-12... (Science Daily)

    Opinion: Freebie ban is insulting to docs  May 8, 2008
    So students and residents enjoy your pizza sit down, learn about a new heart valve, a new antibiotic, a new advance in gene therapy and get on with your duties to your patients. Click for related content. (MSNBC -- Terrorism)

    Naturally-occuring Protein May Be Effective In Limiting Heart Attack Injury And Restoring Function  May 8, 2008
    7, 2004) A novel gene therapy that responds specifically to oxygen-starved heart muscle may protect against further injury following a heart attack, a study by University of South Florida cardiovascular. (May 27, 2002) University of Melbourne researchers, along with doctors and scientists from eight Melbourne hospitals, have located three genes that make heart attack more likely. (Science Daily)

    Double Duty: Loss Of Protective Heart Failure Protein Causes High Blood Pressure  May 8, 2008
    D, director of the Center for Translational Medicine, they have proven that loss of the protein causes diseased hearts to fail and that the protein is a potential target for gene therapy for heart failure ... (May 24, 2007) Heart researchers have used gene therapy to reverse heart failure in animals. (Science Daily)

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