#gene therapy — Wednesday, October 07, 2026 (2 items)
| 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)
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Cell Reports Medicine 2026-10-07 | #gene therapy #deafness #variants #congenital | |
| 9 | ℹ |
Use of split intein and myotropic AAV vectors enables effective preclinical gene therapy for dysferlinopathy.
(science.org)
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Science Translational Medicine 2026-10-07 | #gene therapy #muscular dystrophy #aav vectors #protein design #muscle-disease |