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Postnatal and Prenatal Therapeutic Base Editing for Metabolic Diseases — Kiran Musunuru (CHILDREN'S HOSP OF PHILADELPHIA)

PROJECT SUMMARY The potential for the development of novel therapeutic modalities has energized the genome editing field since it first emerged in the 1990s and especially since the demonstration of p

Project titlePostnatal and Prenatal Therapeutic Base Editing for Metabolic Diseases
Project number5U19NS132301-02
PI nameKiran Musunuru
InstitutionCHILDREN'S HOSP OF PHILADELPHIA
Funding mechanismU19
AgencyNINDS
Award amount$5.05M
Fiscal year2024
Project start2023-08-01T00:00:00
Project end2028-07-31T00:00:00
AbstractPROJECT SUMMARY The potential for the development of novel therapeutic modalities has energized the genome editing field since it first emerged in the 1990s and especially since the demonstration of p
Key terms<1H-Purin-6-amine><AAV vector><AAV-based vector><ANGPT5><ANGPTL3><ANGPTL3 gene><Address><Adenine><Ad

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