A novel computational platform developed by researchers from the University of Pittsburgh School of Medicine identifies top-performing viral vectors that could deliver gene therapies to the retina with maximum efficiency ...
Oct 19, 2021
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65
Autosomal dominant polycystic kidney disease (ADPKD), a genetic disorder, causes fluid-filled cysts to develop on the kidneys, which can impair their function. As part of the growth of cysts, the kidneys develop inflammation ...
Oct 13, 2021
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76
A common mutation in gliomas sensitizes them to immunotherapy, a finding that researchers believe could have broader therapeutic implications for all glioma patients.
Sep 29, 2021
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51
Genetic muscle diseases lead to progressive muscle wasting and often early death, with few treatment options and no cure. Some gene therapies that use a harmless virus to deliver a functioning copy of a disease-causing gene ...
Sep 9, 2021
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244
A new study from Linköping University has shown that the tumor-inhibiting gene TET2 is silenced in a large fraction of cases of acute lymphoblastic leukemia (ALL) in children. The scientists show that the gene can be reactivated ...
Aug 25, 2021
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294
In a major advancement in the field of gene therapy for rare and devastating diseases, researchers at Children's Hospital of Philadelphia (CHOP) have developed a "dimmer switch" system that can control levels of proteins ...
Jul 28, 2021
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294
A form of gene therapy protects optic nerve cells and preserves vision in mouse models of glaucoma, according to research supported by NIH's National Eye Institute. The findings suggest a way forward for developing neuroprotective ...
Jul 22, 2021
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196
A chemical modification that occurs in some RNA molecules as they carry genetic instructions from DNA to cells' protein-making machinery may offer protection against non-alcoholic fatty liver, a condition that results from ...
Jul 19, 2021
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86
A novel method of gene therapy is helping children born with a rare genetic disorder called AADC deficiency that causes severe physical and developmental disabilities. The study, led by researchers at The Ohio State University ...
Jul 12, 2021
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46
Research at Baylor College of Medicine, the Texas Heart Institute and collaborating institutions is moving a novel promising gene therapy to treat heart failure closer to the clinic.
Jun 30, 2021
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