This site is new - feedback and suggestions welcome via GitHub issues.
Top 1d 2d 3d 7d 14d 30d
Show 10 20 50
hot top new auto
Oct 05 Oct 06 Oct 07 Oct 08 Oct 09 Oct 10 Oct 11

#gene therapy  — top 20, last 7d

25 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