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Journal Article
What is CRISPR/Cas9?
2016
Overview
Two similar studies have described using the CRISPR/Cas9 system in vivo to increase expression of the dystrophin gene and improve muscle function in mouse models of DMD. 6 7 Other studies have used CRISPR/Cas9 to target duplication of exons in the human dystrophin gene in vitro and have shown that this approach can lead to production of full-length dystrophin in the myotubules of an individual with DMD. 8 CRISPR/Cas9 could also be used to treat haemoglobinopathies. After further refinement, the authors suggest their findings may enable gene therapies or transplantation of genetically altered bone marrow stem cells or inducible pluripotent stem cells to eradicate HIV infection. 10 Engineering somatic cells ex vivo to treat malignancy or other diseases There has been increasing interest in the possibility of using CRISPR/Cas9 to modify patient-derived T-cells and stem/progenitor cells which can then be reintroduced into patients to treat disease.
Publisher
BMJ Publishing Group LTD,BMJ Publishing Group
Subject
/ Child
/ Clustered Regularly Interspaced Short Palindromic Repeats - immunology
/ CRISPR-Associated Proteins - genetics
/ CRISPR-Cas Systems - genetics
/ CRISPR-Cas Systems - immunology
/ Editing
/ Female
/ Genomes
/ HIV
/ Human immunodeficiency virus
/ Humans
/ Infant
/ Male
/ Mutation
/ Patients
/ Proteins
/ Studies
