Gene modification therapies for hereditary diseases in the fetus
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DOI: 10.1002/pd.6347
Abstract: Proof‐of‐principle disease models have demonstrated the feasibility of an intrauterine gene modification therapy (in utero gene therapy (IUGT)) approach to hereditary diseases as diverse as coagulation disorders, haemoglobinopathies, neurogenetic disorders, congenital metabolic, and pulmonary diseases.… read more here.
Keywords: gene modification; pathology; modification therapies; gene ... See more keywords