A new study shows the feasibility of using gene therapy to treat the progressive neurodegenerative disorder chronic traumatic encephalopathy (CTE). The study, which demonstrated the effectiveness of direct delivery of gene ...
Jan 3, 2020
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Before gene therapy can be used to treat renal diseases, delivery of therapeutic genes to the kidney must become much more efficient. A novel approach in which three different gene delivery vectors were injected intravenously ...
Dec 31, 2019
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Scientists have developed a new gene-therapy technique by transforming human cells into mass producers of tiny nano-sized particles full of genetic material that has the potential to reverse disease processes.
Dec 17, 2019
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A new study has shown that a commonly used vector for large gene transfer can successfully deliver genes to retinal cells in the laboratory, but when injected subretinally into rats it provokes a robust and acute inflammatory ...
Nov 12, 2019
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Researchers are making great strides toward de-veloping gene-based strategies to treat a variety of inherited neurometabolic diseases characterized by severe neurological involvement. A review of the approaches currently ...
Oct 29, 2019
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Nearly two decades ago, a gene therapy restored vision to Lancelot, a Briard dog who was born with a blinding disease. This ushered in a period of hope and progress for the field of gene therapy aimed at curing blindness, ...
Sep 10, 2019
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Research led by Dr. Krystof Bankiewicz, who recently joined The Ohio State University College of Medicine, shows that gene replacement therapy for Niemann-Pick type A disease is safe for use in nonhuman primates and has therapeutic ...
Aug 21, 2019
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An optimized and newly engineered form of the adeno-associated vector 9 (AAV9) vector used to deliver the galactosylceramidase gene to a mouse model of the inherited neurogenerative and rapidly fatal form of Krabbe disease ...
Aug 13, 2019
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It is a remarkable proof for the concept of IOB: working closely hand in hand, our molecular and clinical researchers have developed a library of 230 adeno associated viral vectors (AAVs), each with a different synthetic ...
Jul 19, 2019
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A therapeutic gene delivered into the spinal canal of infant rhesus monkeys was still being expressed after nearly 4 years, with no evidence of acute or chronic neuronal toxicity, according to a new study published in Human ...
Jun 17, 2019
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