Literature DB >> 35194186

CRISPR editing as a therapeutic strategy for Duchenne muscular dystrophy-anti-Cas9 immune response casts its shadow over safety and efficacy.

James J Dowling1,2.   

Abstract

Entities:  

Year:  2022        PMID： 35194186     DOI： 10.1038/s41434-022-00323-8

Source DB:  PubMed          Journal:  Gene Ther        ISSN： 0969-7128            Impact factor:   5.250


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  1 in total

1.  A humanized knockin mouse model of Duchenne muscular dystrophy and its correction by CRISPR-Cas9 therapeutic gene editing.

Authors:  Yu Zhang; Hui Li; Takahiko Nishiyama; John R McAnally; Efrain Sanchez-Ortiz; Jian Huang; Pradeep P A Mammen; Rhonda Bassel-Duby; Eric N Olson
Journal:  Mol Ther Nucleic Acids       Date:  2022-08-01       Impact factor: 10.183
  1 in total

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