MeSH tree: E05.393.420.301
Researchers in the WashU Medicine Department of Ophthalmology & Visual Sciences have discovered key neuroprotective genes that could lead to the development of gene therapies to treat retinitis pigmentosa, an inherited form ...
May 11, 2026
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Thousands of times per year, a family's moment of joy turns to unexpected grief. A seemingly healthy infant stops smiling or making eye contact. Their limbs grow weak. The tiny child suffers seizures and breathing problems.
May 8, 2026
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An inherited form of blindness directly comparable to a common inherited optic nerve disease in humans has been discovered in rhesus macaques at the California National Primate Research Center at the University of California, ...
Apr 28, 2026
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A Virginia Tech team has identified a promising new therapeutic strategy for idiopathic pulmonary fibrosis (IPF), showing that blocking two proteins can halt and even reverse lung scarring in pre-clinical models.
Apr 28, 2026
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US health officials on Thursday greenlit a first-of-its-kind gene therapy to treat a rare form of hereditary hearing loss, a breakthrough that could pave the way for other such hearing impairment treatments.
Apr 24, 2026
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A new international study co-led by investigators from Mass General Brigham and the Eye & ENT Hospital of Fudan University shows that a gene therapy for a rare form of genetic deafness successfully restored hearing in most ...
Apr 22, 2026
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University of Virginia School of Medicine researchers have used an advanced gene-editing technique to correct the underlying cause of a severe form of epilepsy in lab mice. This breakthrough could one day lead to new treatments ...
Apr 7, 2026
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Dr. Donald Kohn has been developing gene therapies for rare pediatric immune disorders for over 30 years. This week, his role in a clinical trial has culminated in the first-ever U.S. Food and Drug Administration–approved ...
Mar 28, 2026
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Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal muscle disease in children. The study shows in animal models that a single treatment is sufficient ...
Feb 26, 2026
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The Food and Drug Administration plans to drop its longtime standard of requiring two rigorous studies to win approval for new drugs, the latest change from Trump administration officials vowing to speed up the availability ...
Feb 19, 2026
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