A Novel Small NPC1 Promoter Enhances AAV-Mediated Gene Therapy in Mouse Models of Niemann-Pick Type C1 Disease (2023)
Attributed to:
MICA: Towards clinical trial readiness of gene therapy for Niemann-Pick disease type c
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