Allele-Selective Knockdown of MYH7 Using Antisense Oligonucleotides

Hundreds of dominant-negative myosin mutations have been identified that lead to hypertrophic cardiomyopathy, and the biomechanical link between mutation and disease is heterogeneous across this patient population. To increase the therapeutic feasibility of treating this diverse genetic population,...

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Udgivet i:Mol Ther Nucleic Acids
Main Authors: Anderson, Brian R., Jensen, Marianne L., Hagedorn, Peter H., Little, Sean C., Olson, Richard E., Ammar, Ron, Kienzle, Bernadette, Thompson, John, McDonald, Ivar, Mercer, Stephen, Vikesaa, Jonas, Nordbo, Bettina, Iben, Larry, Cao, Yang, Natale, Joanne, Dalton-Kay, Greg, Cacace, Angela, Hansen, Bo R., Hedtjärn, Maj, Koch, Troels, Bristow, Linda J.
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2020
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7033438/
https://ncbi.nlm.nih.gov/pubmed/32092825
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2020.01.012
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