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Comment
. 2021 Apr 16;6(1):158.
doi: 10.1038/s41392-021-00576-6.

CRISPR base editor treats premature-aging syndrome

Ping Lin  1 , Jianxin Jiang  2 , Min Wu  3
Affiliations
Comment

CRISPR base editor treats premature-aging syndrome

Ping Lin et al. Signal Transduct Target Ther. .
No abstract available

PubMed Disclaimer

Conflict of interest statement

The authors declare no competing interests.

Figures

Fig. 1
Fig. 1
Programmable base editing rescues Hutchinson–Gilford progeria syndrome. a A single base mutation at 1824 locus in LMNA gene results in mis-splicing and subsequently translates into a truncated lamin A protein, termed progerin, which causes Hutchinson–Gilford progeria syndrome (HGPS). b ABEmax-VRQR corrected human LMNA mutation in two primary fibroblast cells derived from HGPS patients. Using the lentiviral delivery delivered ABEmax-VRQR and sgRNA targeting LMNA c.1824 C > T mutation, resulting in gene editing of the LMNA loci and subsequently rescuing lamin A abundance and nuclear morphology. c ABEmax-VRQR treatment in HGPS mice model. Koblan et al. used the clinical adeno-associated virus vector for co-packaged ABEmax-VRQR to make the correction of mutation in many tissues, but not all, in mouse model, resulting in improved vascular pathology and extended the HGPS mice lifespan
See this image and copyright information in PMC

Comment on

  • In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice.
    Koblan LW, Erdos MR, Wilson C, Cabral WA, Levy JM, Xiong ZM, Tavarez UL, Davison LM, Gete YG, Mao X, Newby GA, Doherty SP, Narisu N, Sheng Q, Krilow C, Lin CY, Gordon LB, Cao K, Collins FS, Brown JD, Liu DR. Koblan LW, et al. Nature. 2021 Jan;589(7843):608-614. doi: 10.1038/s41586-020-03086-7. Epub 2021 Jan 6. Nature. 2021. PMID: 33408413 Free PMC article.

References

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