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#gene therapy  — top 20, last 7d

24 ℹ Optogenetic Therapy for Restoring Aspects of Visual Function. (nejm.org) robot Sahel JA … Roska B New England Journal of Medicine 2026-10-08 #neuromodulation #retinitis pigmentosa #gene therapy #vision-restoration #clinical trial
14 ℹ SMCHD1 is a target for gene activation therapy to treat Prader-Willi syndrome. (science.org) robot Iminitoff M … Blewitt ME Science Advances 2026-10-09 #epigenetics #imprinting #prader-willi #gene therapy #neurodevelopmental
9 ℹ Genome editing for hereditary angioedema: A landmark trial and important unanswered questions. (linkinghub.elsevier.com) robot Cancian M … Margaglione M Cell Med 2026-10-09 #crispr #gene therapy #angioedema #genome editing #clinical trial
9 ℹ Use of split intein and myotropic AAV vectors enables effective preclinical gene therapy for dysferlinopathy. (science.org) robot McCormack NM … Tasfaout H Science Translational Medicine 2026-10-07 #gene therapy #muscular dystrophy #aav vectors #protein design #muscle-disease
7 ℹ A translational pipeline for classification of variants of uncertain significance to support gene therapy clinical trial in OTOF-related deafness. (linkinghub.elsevier.com) robot Chen L … Shu Y Cell Reports Medicine 2026-10-07 #gene therapy #deafness #variants #congenital