In vivo gene editing of CAMKIID: out with the bad and in with the good
- PMID: 38165033
- PMCID: PMC10760942
- DOI: 10.1172/JCI176672
In vivo gene editing of CAMKIID: out with the bad and in with the good
Abstract
The ability to change an organism's DNA through gene editing is of great importance for the prevention and treatment of genetic and acquired diseases. Rapid progress has been made during the last decade due to the discovery and refinement of CRISPR/Cas9 as an accurate, fast, and reliable genome editing technique. In this issue of the JCI, Lebek et al. present the culmination from a line of work in the Olson laboratory focused on in vivo gene editing of CAMK2D. The paper presents a combined state-of-the-art gene therapy approach that demonstrates how gene therapy can yield cardioprotection in a mouse model and takes notable steps toward potential applicability in patients.
Conflict of interest statement
Comment on
-
CRISPR-Cas9 base editing of pathogenic CaMKIIδ improves cardiac function in a humanized mouse model.J Clin Invest. 2024 Jan 2;134(1):e175164. doi: 10.1172/JCI175164. J Clin Invest. 2024. PMID: 37856214 Free PMC article.
Similar articles
-
Gene Therapy with CRISPR/Cas9 Coming to Age for HIV Cure.AIDS Rev. 2017 Oct-Dec;19(3):167-172. AIDS Rev. 2017. PMID: 29019352
-
Therapeutic Genome Editing and In Vivo Delivery.AAPS J. 2021 Jun 2;23(4):80. doi: 10.1208/s12248-021-00613-w. AAPS J. 2021. PMID: 34080099 Review.
-
CRISPR/Cas9 system: a powerful technology for in vivo and ex vivo gene therapy.Sci China Life Sci. 2017 May;60(5):468-475. doi: 10.1007/s11427-017-9057-2. Epub 2017 Apr 20. Sci China Life Sci. 2017. PMID: 28534255 Review.
-
Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Therapeutics.AAPS J. 2018 Oct 10;20(6):108. doi: 10.1208/s12248-018-0267-9. AAPS J. 2018. PMID: 30306365 Free PMC article. Review.
-
CRISPR/Cas9 System and its Research Progress in Gene Therapy.Anticancer Agents Med Chem. 2019;19(16):1912-1919. doi: 10.2174/1871520619666191014103711. Anticancer Agents Med Chem. 2019. PMID: 31633477 Review.
References
Publication types
MeSH terms
Substances
Grants and funding
LinkOut - more resources
Full Text Sources
Research Materials