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Keyword: genetherapy

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  • US approves gene therapy for cancer from Israeli-founded company (BREAKTHROUGH!)

    10/19/2017 7:41:42 AM PDT · by RoosterRedux · 9 replies ^ | Max Schindler
    American regulators granted approval on Wednesday to a gene-altering treatment for patients suffering from lymphoma, the second-ever approval for a gene therapy procedure and one that could revolutionize how we fight cancer. The drug, named Yescarta, was developed by Israeli-founded Kite Pharma and it is expected to cost some $373,000, likely generating hundreds of millions of dollars in sales. Kite was purchased by Gilead Sciences in August to the tune of $11.9 billion, and today’s breakthrough may have helped with the sale. The price of Yescarta is set below that of comparable treatments, according to Reuters, as Novartis AG’s gene...
  • Pioneering new injection to cure heart failure without need for major surgery

    08/11/2014 11:13:46 PM PDT · by Innovative · 12 replies
    UK Telegraph ^ | Aug 11, 2014 | Sarah Knapton
    The technique, which involves a simple injection, could aid the recovery of hundreds of thousands of heart failure patients - and could even consign heart transplants to history. Researchers hope to increase levels of SERCA2a, a protein in heart muscle cells that plays an important role in heart muscle contraction The technique, which involves a simple injection, could aid the recovery of hundreds of thousands of heart failure patients. Heart transplants could even be consigned to history thanks to a trial by Imperial College, London, which aims to show for the first time that gene therapy could repair failing organs....
  • Hearing quality restored with bionic ear technology used for gene therapy

    04/23/2014 1:36:03 PM PDT · by Red Badger · 26 replies ^ | 04-24-2014 | Provided by University of New South Wales
    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...
  • Virus That Evolved in the Lab Delivers Gene Therapy into the Retina

    07/04/2013 6:38:53 PM PDT · by neverdem · 9 replies
    MIT Technology Review ^ | June 12, 2013 | Susan Young
    From millions of random mutations, scientists identify a virus that could make gene therapy for inherited retinal diseases safer and more effective.Special delivery: Eight weeks after the new virus was injected into the eye of a monkey, the activity of a fluorescent gene it delivered can be seen in spots across the retina. A new delivery mechanism shuttles gene therapy deep into the eye’s retina to repair damaged light-sensing cells without requiring a surgeon to put a needle through this delicate tissue. The approach could make it substantially easier to treat inherited forms of eye disease with this approach.Although still...
  • Gene therapy cures leukaemia in eight days

    03/27/2013 7:36:28 AM PDT · by Sir Napsalot · 2 replies
    NewScientist ^ | 3-26-2013 | Andy Coghlan
    WITHIN just eight days of starting a novel gene therapy, David Aponte's "incurable" leukaemia had vanished. For four other patients, the same happened within eight weeks, although one later died from a blood clot unrelated to the treatment, and another after relapsing. The cured trio, who were all previously diagnosed with usually fatal relapses of acute lymphoblastic leukaemia, have now been in remission for between 5 months and 2 years. Michel Sadelain of the Memorial Sloan-Kettering Cancer Center in New York, co-leader of the group that designed the trial, says that a second trial of 50 patients is being readied,...
  • HIV Vector Licks Leukemia

    12/10/2012 6:20:25 PM PST · by neverdem · 25 replies
    A gene therapy that uses infusions of patients’ own T cells genetically engineered to attack their tumors enjoyed its first successful and sustained demonstration of clinical-trial success in nine of 12 leukemia patients—two of whom have been in remission for more than two years. The therapy was pioneered by the University of Pennsylvania’s Perelman School of Medicine, whose researchers will present latest results from the study today at the American Society of Hematology (ASH)'s Annual Meeting and Exposition in Atlanta.According to Penn Perelman, the results pave the way for a potential paradigm shift in the treatment of these types of...
  • DNA-swap technology almost ready for fertility clinic

    10/25/2012 10:31:45 PM PDT · by neverdem · 8 replies
    NATURE NEWS ^ | 24 October 2012 | David Cyranoski
    Mitochondrial transfer could reduce the risk of childhood disease. Researchers say that technology to shuffle genetic material between unfertilized eggs is ready to make healthy babies. The technique could allow parents to minimize the risk of a range of diseases related to defects in the energy-producing cell organelles known as mitochondria. Mitochondrial defects affect an estimated 1 in 4,000 children, and can cause rare and often fatal diseases such as carnitine deficiency, which prevents the body from using fats for energy. They are also implicated in a wide range of more common diseases affecting children and adults, such as multiple...
  • Anti-cocaine vaccine described in Human Gene Therapy Journal

    06/19/2012 11:34:35 PM PDT · by neverdem · 12 replies
    Biology News Net ^ | June 18, 2012 | NA
    A single-dose vaccine capable of providing immunity against the effects of cocaine offers a novel and groundbreaking strategy for treating cocaine addiction is described in an article published Instant Online in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc. ( The article is available free online at the Human Gene Therapy website ( "This is a very novel approach for addressing the huge medical problem of cocaine addiction," says James M. Wilson, MD, PhD, Editor-in-Chief, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia. In...
  • Spell-Checked Stem Cells Show Promise Against Liver Disease

    10/13/2011 6:56:01 PM PDT · by neverdem · 14 replies
    ScienceNOW ^ | 12 October 2011 | Jocelyn Kaiser
    Enlarge Image Gene fix. Red cells in this slice of mouse liver are making a human protein called A1At. Credit: K. YUSA ET AL., NATURE (ADVANCED ONLINE EDITION) ©2011 MACMILLAN PUBLISHERS LTD. Researchers have taken a step toward showing how stem cells might one day be used to help patients born with a deadly liver disease. The researchers corrected a DNA spelling error in patient skin cells that had been converted into so-called induced pluripotent stem (iPS) cells, then coaxed the cells to form liver cells that seemed to function normally in mice. The approach is still a long...
  • Gene therapy and stem cells unite

    10/13/2011 6:30:34 PM PDT · by CutePuppy · 5 replies
    BBC ^ | October 12, 2011 | James Gallagher
    Two of the holy grails of medicine - stem cell technology and precision gene therapy - have been united for the first time in humans, say scientists.It means patients with a genetic disease could, one day, be treated with their own cells. A study in Nature corrected a mutation in stem cells made from a patient with a liver disease. Researchers said this was a "critical step" towards devising treatments, but safety tests were still needed. At the moment, stem cells created from a patient with a genetic illness cannot be used to cure the disease as those cells would...
  • Breakthrough in fight against fatal Ebola as new drug saves 100% of monkeys tested! (Praise God!)

    05/29/2010 4:21:54 PM PDT · by Niuhuru · 29 replies · 708+ views
    Daily Mail ^ | 10:57 PM on 29th May 2010 | Daily Mail Reporter
    A gene silencing approach can save monkeys from high doses of the most lethal strain of Ebola virus in what researchers call the most viable route yet to treating the deadly and frightening infection. They used small interfering RNAs or siRNAs, a new technology being developed by a number of companies, to hold the virus at bay for a week until the immune system could take over. Tests in four rhesus monkeys showed that seven daily injections cured 100 per cent of them. U.S. government researchers and a small Canadian biotech company, Tekmira Pharmaceuticals, worked together to develop the new...
  • Lego-like gene delivery system is child's play

    05/14/2010 10:03:45 AM PDT · by neverdem · 1 replies · 171+ views
    Highlights in Chemical Biology ^ | 12 May 2010 | Philippa Ross
    Functionalised gold nanoparticles make a more efficient and versatile delivery system for use in gene therapy and cancer treatment, claim Korean scientists. Gold nanoparticles are non-toxic and easily made and so provide an effective way to deliver therapeutic antisense DNA to disease causing genes. Antisense DNA that is covalently attached to the nanoparticles will bind to messenger RNA of a disease causing gene, preventing translation and therefore inactivating it. But previous systems could deliver only antisense DNA that was covalently cross-linked to gold nanoparticles, which needed to be individually synthesized for each gene of interest. Now, Kangseok Lee and Min Su Han at...
  • Gene Therapy Cures Canines of Inherited Form of Day Blindness, Penn Veterinary Researchers Say

    04/21/2010 10:00:39 AM PDT · by decimon · 4 replies · 139+ views
    Veterinary ophthalmology researchers from the University of Pennsylvania have used gene therapy to restore retinal cone function and day vision in two canine models of congenital achromatopsia, also called rod monochromacy or total color blindness. Achromatopsia is a rare autosomal recessive disorder with an estimated prevalence in human beings of about 1 in 30,000 to 50,000. It primarily affects the function of the cone photoreceptors in the retina and serves as a representative model for other more common inherited retinal disorders affecting cones. Cone function is essential for color vision, central visual acuity and most daily visual activities, which underlines...
  • Second Dose Of Gene Therapy For Inherited Blindness Proves Safe In Animal Study

    03/06/2010 10:41:26 PM PST · by neverdem · 4 replies · 271+ views
    Gene therapy for a severe inherited blindness, which produced dramatic improvements last year in 12 children and young adults who received the treatment in a clinical trial, has cleared another hurdle. The same research team that conducted the human trial now reports that a study in animals has shown that a second injection of genes into the opposite, previously untreated eye is safe and effective, with no signs of interference from unwanted immune reactions following the earlier injection. These new findings suggest that patients who benefit from gene therapy in one eye may experience similar benefits from treatment in the...
  • Longevity Tied to Genes That Preserve Tips of Chromosomes

    11/11/2009 4:03:13 PM PST · by decimon · 35 replies · 1,016+ views
    (BRONX, NY) — A team led by researchers at Albert Einstein College of Medicine of Yeshiva University has found a clear link between living to 100 and inheriting a hyperactive version of an enzyme that rebuilds telomeres — the tip ends of chromosomes. The findings appear in the latest issue of the Proceedings of the National Academy of Sciences. Telomeres play crucial roles in aging, cancer and other biological processes. Their importance was recognized last month, when three scientists were awarded the 2009 Nobel Prize in Physiology and Medicine for determining the structure of telomeres and discovering how they protect...
  • Brain disease treated by gene therapy - A treatment based on HIV finds first success in...

    11/08/2009 8:58:34 PM PST · by neverdem · 6 replies · 685+ views
    Nature News ^ | 5 November 2009 | Lizzie Buchen
    A treatment based on HIV finds first success in humans.Researchers have halted a fatal brain disease by delivering a therapeutic gene to the stem cells that mature into blood cells. The gene was transferred using a virus derived from HIV, a technique that researchers have pursued for more than a decade but has not been successful in humans until now. Together with his colleagues, paediatric neurologist Patrick Aubourg at INSERM — France's main biomedical research agency — and at the Saint-Vincent de Paul Hospital in Paris, developed the system to treat X-linked adrenoleukodystrophy (ALD), a neurodegenerative disease that affects young...
  • Gene therapy could remedy Parkinson's

    10/17/2009 10:31:51 PM PDT · by neverdem · 21 replies · 1,608+ views
    Nature News ^ | 14 October 2009 | Elie Dolgin
    Introducing three genes corrects motor defects in monkeys.A potential gene therapy for Parkinson's disease can correct motor deficits in monkeys without causing the jerky, involuntary movements that often accompany long-term treatments for the disease. The approach is undergoing preliminary testing in a handful of human patients, who have all shown promising signs of improvement.At present, the most common remedy for Parkinson's disease involves replacing dopamine — the neurotransmitter that is depleted in patients with the disease — by administering the dopamine precursor levodopa, or L-DOPA. Most patients initially regain near-normal motor control, but after several years on L-DOPA the majority...
  • Colour blindness corrected by gene therapy - Treated monkeys can now see in technicolour.

    09/17/2009 2:01:52 PM PDT · by neverdem · 15 replies · 747+ views
    Nature News ^ | 16 September 2009 | Elie Dolgin
    Dalton, a squirrel monkey treated with gene therapy, enjoys his new colour sense.Neitz Laboratory Researchers have used gene therapy to restore colour vision in two adult monkeys that have been unable to distinguish between red and green hues since birth — raising the hope of curing colour blindness and other visual disorders in humans."This is a truly amazing study," says András Komáromy, a vision researcher and veterinary ophthalmologist at the University of Pennsylvania in Philadelphia, who was not involved in the research. "If we can target gene expression specifically to cones [in humans] then this has a tremendous implication."About 1...
  • Embryonic-like Cells Advance Toward Disease Treatment

    06/02/2009 9:24:12 PM PDT · by neverdem · 8 replies · 576+ views
    ScienceNOW Daily News ^ | 1 June 2009 | Constance Holden
    Enlarge ImageOn track. Colonies of genetically corrected cells taken from Fanconi anemia patients show red and yellow, markers associated with pluripotency. Credit: Juan Carlos Izpisúa Belmonte Two papers published this week appear to bring closer the day when embryonic-like stem cells can be used to treat human diseases. One study describes what scientists say is the safest method yet to produce these cells. The other reports success in using the cells to begin correcting a rare genetic disorder known as Fanconi anemia. Induced pluripotent stem (iPS) cells were first reported in 2006 by Shinya Yamanaka, a researcher at Kyoto...
  • Designer Antibodies Derail Monkey AIDS Virus

    05/21/2009 1:33:20 AM PDT · by neverdem · 2 replies · 496+ views
    ScienceNOW Daily News ^ | 18 May 2009 | Jon Cohen
    Enlarge ImageDesigner genes. Monkeys given a virus that holds the DNA for lab-made antibodies dodged SIV.Credit: Malcolm Linton Researchers are reporting that a new antiviral strategy powerfully protects monkeys from SIV, the simian cousin of HIV. The approach combines elements of vaccines and gene therapy, and experts say the development could eventually lead to a vaccinelike weapon against AIDS--a goal that has thus far proved elusive. Vaccines work by priming the "adaptive" immune system to recognize and attack a specific invader. But despite 2 decades of research, several potential AIDS vaccines have failed to teach the immune system to...