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AAV-Mediated Somatic Gene Editing for Cardiac and Skeletal Muscle in a Large Animal Model

  • Technical University of Munich
  • Partner Site Munich Heart Alliance

Research output: Chapter in Book/Report/Conference proceedingChapterpeer-review

3 Scopus citations

Abstract

Here we describe a protocol to produce a recombinant adeno-associated viral vector (rAAV)-based system to deliver the CRISPR-Cas9 complex into porcine skeletal muscle and myocardial cells. We initially describe the genomic composition of the rAAV-CRISPR vectors used in our lab. Furthermore, we give a step-by-step instruction into the production of recombinant viral vectors with high yields and purity. Lastly we describe the minimally invasive injection regimes to target the myocardium in a pig.

Original languageEnglish
Title of host publicationMethods in Molecular Biology
PublisherHumana Press Inc.
Pages63-74
Number of pages12
DOIs
StatePublished - 2022

Publication series

NameMethods in Molecular Biology
Volume2573
ISSN (Print)1064-3745
ISSN (Electronic)1940-6029

Keywords

  • CRISPR
  • Cas9
  • Duchenne muscular dystrophy
  • Gene editing
  • Gene vector
  • rAAV

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