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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Pubblicato in:Mol Ther Nucleic Acids
Autori principali: 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.
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Gene & Cell Therapy 2020
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Article
Accesso online: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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