#gene therapy — Wednesday, September 30, 2026 (2 items)
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Cell-autonomous restoration of splicing homeostasis and RP11 phenotype in patient-derived RPE and retinal organoids by PRPF31.AAV gene therapy.
(nature.com)
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Nature Communications 2026-09-30 | #splicing #gene therapy #retinitis pigmentosa #organoid #spliceosome | |
| 1 | ℹ |
Integrating clinical diagnostics with interventional genetics for neurodevelopmental disorders.
(linkinghub.elsevier.com)
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American Journal of Human Genetics 2026-09-30 | #neurodevelopmental #sequencing #gene therapy #ipsc #genetics |