CRISPR technologies for the treatment of Duchenne muscular dystrophy.

doi:10.1016/j.ymthe.2021.04.002

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Back to search

Article

CRISPR technologies for the treatment of Duchenne muscular dystrophy.

Molecular therapy : the journal of the American Society of Gene Therapy - 3 Nov 2021

Choi Eunyoung, Koo Taeyoung

Abstract excerpt

The emerging clustered regularly interspaced short palindromic repeats (CRISPR)-mediated genome editing technologies have progressed remarkably in recent years, opening up the potential of precise genome editing as a therapeutic approach to treat various diseases. The CRISPR-CRISPR-associated (Cas) system is an attractive platform for the treatment of Duchenne muscular dystrophy (DMD), which is a neuromuscular...

Topics

Animals
CRISPR-Cas Systems
Clinical Studies as Topic
Disease Management
Disease Models, Animal
Dystrophin
Exons
Gene Editing
Genetic Therapy
Humans
Muscular Dystrophy, Duchenne
Mutation
Treatment Outcome

Source

Open in PubMed Open full text

Identifiers

PMID: 33823301
DOI: 10.1016/j.ymthe.2021.04.002
PMCID: PMC8571109

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 30 Aug 2026.

Community interpretation

Discuss the evidence

Ask a question, add context or challenge an interpretation. Community activity is separate from scientific evidence strength.

discussions

replies

Citations encouraged

Claims remain attributable.

No discussion yet

Be the first to add a careful, cited interpretation.

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics