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Simon Waddington
profsimonwad.bsky.social
did:plc:lvtnxs2psndrgeqqu6kmkq36
🧪 🧬 - proof of concept gene therapy for mitochondrial disease Deoxyguanosine kinase deficiency. Neonatal liver failure. Transplant is the only option - but doesn't help the brain disease. This is preliminary data showing gene therapy could treat both. (https://www.cell.com/molecular-therapy-family/methods/pdf/S2329-0501(24)00213-4.pdf )
2024-12-18T07:35:00.222Z