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Combining multiomics and drug perturbation profiles to identify muscle-specific treatments for spinal muscular atrophy. (2021)

First Author: Meijboom KE

Attributed to: Peptide conjugated oligonucleotides for a phase I/IIa clinical trial in Spinal Muscular Atrophy funded by MRC

Abstract

No abstract provided

Bibliographic Information

Digital Object Identifier: http://dx.doi.org/10.1172/jci.insight.149446

PubMed Identifier: 34236053

Publication URI: http://europepmc.org/abstract/MED/34236053

Type: Journal Article/Review

Volume: 6

Parent Publication: JCI insight

Issue: 13

ISSN: 2379-3708