A Novel Small NPC1 Promoter Enhances AAV-Mediated Gene Therapy in Mouse Models of Niemann–Pick Type C1 Disease.

Autor: Hughes, Michael Paul1 (AUTHOR) h.nelvagal@ucl.ac.uk, Nelvagal, Hemanth Ramesh1 (AUTHOR) oliver.coombe-tennant.21@ucl.ac.uk, Coombe-Tennant, Oliver1 (AUTHOR) giulia.massaro.13@ucl.ac.uk, Smith, Dave2 (AUTHOR) dave.smith@pharm.ox.ac.uk, Smith, Claire2 (AUTHOR) claire.smith@pharm.ox.ac.uk, Massaro, Giulia1 (AUTHOR), Poupon-Bejuit, Laura1 (AUTHOR), Platt, Frances Mary2 (AUTHOR) frances.platt@pharm.ox.ac.uk, Rahim, Ahad Abdul1 (AUTHOR) a.rahim@ucl.ac.uk
Zdroj: Cells (2073-4409). Jun2023, Vol. 12 Issue 12, p1619. 21p.
Databáze: Academic Search Ultimate
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