Scripps Research team finds that a nontoxic molecule closely related to resveratrol can overcome barriers to delivering gene therapy into stem cells. SCRIPPS RESEARCH INSTITUTE LA JOLLA, CA – Gene therapy has broadened the treatment possibilities for those with immune system deficiencies and blood-based conditions, such as sickle cell anemia and leukemia. These diseases, which...
Tag: <span>Gene Therapy</span>
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Gene therapy for Duchenne muscular dystrophy safely preserves muscle function
by Perelman School of Medicine at the University of Pennsylvania A gene therapy being developed at Penn Medicine to treat Duchenne muscular dystrophy (DMD) successfully and safely stopped the severe muscle deterioration associated with the rare, genetic disease in both small and large animal models, according to a first-of-its-kind study from Penn Medicine researchers. The...
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NIH researchers create new viral vector for improved gene therapy in sickle cell disease
‘Forward-oriented’ design might boost treatment effectiveness and broaden use NIH/NATIONAL HEART, LUNG AND BLOOD INSTITUTE Researchers at the National Institutes of Health (NIH) have developed a new and improved viral vector–a virus-based vehicle that delivers therapeutic genes–for use in gene therapy for sickle cell disease. In advanced lab tests using animal models, the new vector was up to 10 times more efficient at incorporating corrective genes into bone marrow stem...
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Tiny capsules packed with gene-editing tools offer alternative to viral delivery of gene therapy
Posted Today New tools for editing genetic code offer hope for new treatments for inherited diseases, some cancers, and even stubborn viral infections. But the typical method for delivering gene therapies to specific tissues in the body can be complicated and may cause troubling side effects. Researchers at the University of Wisconsin–Madison have addressed many...
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GENE THERAPY RESTORES VISION IN BLIND MICE
The Rain Has Gone Thanks to a new gene therapy targeting specific cells in the eye, blind mice have regained the ability to see. A team of neuroscientists developed a treatment that re-activated the Cngb1 gene, which when disabled causes light-detecting rod cells found in the retina to deteriorate, according to research recently published in the journal JNeurosci. The recovered rod cells not only regained the...
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Response to gene-targeted drugs depends on cancer type
by Institute of Cancer Research Cancers with the same genetic weaknesses respond differently to targeted drugs depending on the tumour type of the patient, new research reveals. The study is set to prompt changes in thinking around precision medicine—because it shows that the genetics of a patient’s cancer may not always be enough to tell whether it will respond to a treatment. The researchers are already starting...
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Gene therapy may help fight tough-to-treat blood cancer
by Amy Norton, Healthday Reporter (HealthDay)—A gene therapy that tweaks the immune system might offer hope to people with blood cancer that has resisted standard treatments, a new preliminary trial suggests. The cancer, called multiple myeloma, arises in certain white blood cells. It is currently incurable, but there are treatments that can help people live with the disease for years. However, most people eventually progress, and some fail to respond to the available therapies...
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Realizing the potential of gene therapy for neurological disorders
Promising findings from preclinical animal studies show the potential of gene therapy for treating incurable neurological disorders. In new research presented today, scientists successfully used gene therapy to slow the progression and improve symptoms of disorders such as amyotrophic lateral sclerosis and Parkinson’s disease. The findings were presented at Neuroscience 2018, the annual meeting of...
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Making gene therapy delivery safer and more efficient
Viral vectors used to deliver gene therapies undergo spontaneous changes during manufacturing which affects their structure and function, found researchers from the Perelman School of Medicine at the University of Pennsylvania in a study published in Molecular Therapy. As gene therapy approaches become more common for treating disease, managing consistency of the molecular makeup of the virus particles...
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Bladder control via gene therapy?
What are the limits of gene therapy? “There are no currently available FDA-approved gene therapy treatments for overactive bladder,” reads this press release. And that’s true. But is it, like, a problem? Urovant Sciences believes so, which is why it licensed just such a gene therapy for overactive bladder. It’s called hMaxi-K, and it targets...