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Comment
. 2019 Aug 7;27(8):1346-1347.
doi: 10.1016/j.ymthe.2019.06.008. Epub 2019 Jun 24.

The Right Tools for the Right Job: CRISPR-pass Could Offer Safe Gene Correction for Many Disorders

David J Segal  1
Affiliations
Comment

The Right Tools for the Right Job: CRISPR-pass Could Offer Safe Gene Correction for Many Disorders

David J Segal. Mol Ther. .
No abstract available

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References

    1. Lee C., Hyun Jo D., Hwang G.H., Yu J., Kim J.H., Park S.E., Kim J.S., Kim J.H., Bae S. CRISPR-pass: Gene rescue of nonsense mutations using adenine base editors. Mol. Ther. 2019;27:1364–1371. this issue. - PMC - PubMed
    1. Malik V., Rodino-Klapac L.R., Viollet L., Wall C., King W., Al-Dahhak R., Lewis S., Shilling C.J., Kota J., Serrano-Munuera C. Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophy. Ann. Neurol. 2010;67:771–780. - PubMed
    1. McDonald C.M., Campbell C., Torricelli R.E., Finkel R.S., Flanigan K.M., Goemans N., Heydemann P., Kaminska A., Kirschner J., Muntoni F., Clinical Evaluator Training Group; ACT DMD Study Group Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2017;390:1489–1498. - PubMed
    1. Iossifov I., Ronemus M., Levy D., Wang Z., Hakker I., Rosenbaum J., Yamrom B., Lee Y.H., Narzisi G., Leotta A. De novo gene disruptions in children on the autistic spectrum. Neuron. 2012;74:285–299. - PMC - PubMed
    1. Long C., Amoasii L., Mireault A.A., McAnally J.R., Li H., Sanchez-Ortiz E., Bhattacharyya S., Shelton J.M., Bassel-Duby R., Olson E.N. Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy. Science. 2016;351:400–403. - PMC - PubMed

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