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Gene Therapy
for BIO 2024
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Carlos Lopez-Gomez
1 result
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Gene therapy for thymidine kinase 2 (TK2) deficiency
CU18199
This technology is a gene therapy strategy to treat TK2 deficiency-associated mitochondrial DNA depletion syndromes with an engineered adenovirus-associated viral (AAV) vector to deliver a functioning TK2 gene.
9
Comorbidity
Enzyme
Gene therapy
Carlos Lopez-Gomez
Hasan Orhan Akman
Michio Hirano M.D.
Kristin Neuman
Neurology
Columbia University Medical Center (CUMC)
2023-01-17
