Search Results - "Rok, Matthew"
-
1
Advances in CRISPR/Cas9 Genome Editing for the Treatment of Muscular Dystrophies
Published in Human gene therapy (01-05-2023)“…Muscular dystrophies (MDs) comprise a diverse group of inherited disorders characterized by progressive muscle loss and weakness. Given the genetic etiology…”
Get more information
Journal Article -
2
Targeted genome editing in vivo corrects a Dmd duplication restoring wild‐type dystrophin expression
Published in EMBO molecular medicine (07-05-2021)“…Tandem duplication mutations are increasingly found to be the direct cause of many rare heritable diseases, accounting for up to 10% of cases. Unfortunately,…”
Get full text
Journal Article -
3
Prevention of early-onset cardiomyopathy in Dmd exon 52–54 deletion mice by CRISPR-Cas9-mediated exon skipping
Published in Molecular therapy. Methods & clinical development (14-09-2023)“…Duchenne muscular dystrophy (DMD) is a disease with a life-threatening trajectory resulting from mutations in the dystrophin gene, leading to degeneration of…”
Get full text
Journal Article -
4
In Vivo Genome Engineering for the Treatment of Muscular Dystrophies
Published in Current stem cell reports (01-09-2020)“…Purpose of Review Muscular dystrophies (MDs) are a heterogeneous collection of inherited disorders which cause progressive muscle loss and weakness/hypotonia…”
Get full text
Journal Article -
5
Twitter Activity Is Associated With a Higher Research Citation Index for Academic Thoracic Surgeons
Published in The Annals of thoracic surgery (01-08-2020)“…Academic surgeons are encouraged to promote their work on social media. We hypothesized that thoracic surgeons who are active on Twitter have a higher research…”
Get full text
Journal Article