Gene editing via the clustered regularly interspaced short palindromic repeats (CRISPR) system is a promising therapeutic for DMD, as it can permanently correct DMD mutations and thus restore the …

Oct 1, 2023 · Several delivery methods, including viral vectors, nanoparticles, and electroporation, have been explored to deliver CRISPR/Cas9 to the targeted cells. Despite the potential of CRISPR/Cas9 …

May 12, 2025 · Knowledge gained through those efforts ultimately led to DMD becoming the first muscular dystrophy, and one of the first human monogenic diseases, to profit from the newly-born …

Jul 26, 2025 · CRISPR-based gene editing for muscular dystrophy has shown progress in preclinical studies, demonstrating its potential to restore dystrophin expression and improve muscle function in …

Jan 7, 2025 · 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 …

Jan 17, 2025 · A CRISPR-based gene therapy for muscular dystrophy has demonstrated encouraging preclinical results, hoping to progress to clinical trials.

Duchenne muscular dystrophy (DMD) is a neuromuscular disease caused by mutations in the DMD gene. This review summarizes various therapeutic strategies that use CRISPR-Cas to correct or …

To fix the dysferlin mutation, Escobar uses CRISPR-Cas9, which is often described as "gene-editing scissors" and for which a Nobel Prize was awarded in 2020. The molecular scissors are guided to...

Using CRISPR to bypass DMD mutations, dystrophin expression has been efficiently restored in human cells and mouse models of DMD. Here, we review recent progress toward the development of …

Jan 25, 2021 · Using a gene editing technology called CRISPR, Dr. Eric Olson and his lab at UT Southwestern were able to correct a genetic flaw in Ben's cells that had caused him to face the …