#gene therapy — Friday, June 26, 2026 (2 items)
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Re-administration of AAV-mediated gene therapy for OTOF-related deafness: a single-arm trial.
(nature.com)
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Nature Medicine 2026-06-26 | #gene therapy #deafness #clinical trial #aav #immunogenicity | |
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Cell type-specific contextualisation of the human phenome: towards the systematic treatment of all rare diseases.
(link.springer.com)
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Genome Medicine 2026-06-26 | #transcriptomics #rare-diseases #phenotyping #cell-type #gene therapy |