A translatable RNAi-driven gene therapy silences PMP22/Pmp22 genes and improves neuropathy in CMT1A mice. (2022)
Attributed to:
Exosomal protein deficiencies: how abnormal RNA metabolism results in childhood-onset neurological diseases
funded by
MRC
Abstract
No abstract provided
Bibliographic Information
Digital Object Identifier: http://dx.doi.org/10.1172/jci159814
PubMed Identifier: 35579942
Publication URI: http://europepmc.org/abstract/MED/35579942
Type: Journal Article/Review
Volume: 132
Parent Publication: The Journal of clinical investigation
Issue: 13
ISSN: 0021-9738