Helena Escobar
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helenascobar.bsky.social
Helena Escobar
@helenascobar.bsky.social
34 followers 14 following 1 posts
Postdoc - Spuler lab - Max Delbrück Center & Charité - Universitätsmedizin Berlin | Gene & Cell therapies for muscular dystrophy | CRISPR | Muscle Stem Cells |
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Reposted by Helena Escobar
mdc-berlin.bsky.social
A #CRISPR therapy for muscular dystrophy developed at #mdcBerlin and Charité Berlin is showing promise in mice. The study was published in @naturecomms.bsky.social.

First author: @helenascobar.bsky.social (Simone Spuler Lab)

Further information: www.mdc-berlin.de/news/press/d...
Developing a CRISPR therapy for muscular dystrophy
Researchers at the Experimental and Clinical Research Center in Berlin are developing a targeted treatment for muscular dystrophy with the help of gene-editing. Preclinical research led by the Spuler ...
www.mdc-berlin.de
January 7, 2025 at 1:40 PM
helenascobar.bsky.social
Very happy and proud to finally see our paper on rescuing dysferlin deficient muscular dystrophy with muscle stem cells & CRISPR published in @naturecomms.bsky.social. Big thanks to all co-authors for their great work! www.nature.com/articles/s41... @mdc-berlin.bsky.social
Gene-editing in patient and humanized-mice primary muscle stem cells rescues dysferlin expression in dysferlin-deficient muscular dystrophy - Nature Communications
Dysferlin-deficient muscular dystrophy is a devastating and untreatable disease. Using Cas9, the authors restored dysferlin in muscle stem cells from patients ex vivo and show proof-of-concept for aut...
www.nature.com
January 3, 2025 at 2:09 PM