A Novel Small NPC1 Promoter Enhances AAV-Mediated Gene Therapy in Mouse Models of Niemann-Pick Type C1 Disease (2023)

First Author: Hughes M
Attributed to:  The development of gene therapy for Niemann-Pick type C disease funded by MRC

Abstract

No abstract provided

Bibliographic Information

Digital Object Identifier: http://dx.doi.org/10.3390/cells12121619

Publication URI: http://dx.doi.org/10.3390/cells12121619

Type: Journal Article/Review

Parent Publication: Cells

Issue: 12