Sensitive and reliable evaluation of single-cut sgRNAs to restore dystrophin by a GFP-reporter assay

Most Duchenne muscular dystrophy (DMD) cases are caused by deletions or duplications of one or more exons that disrupt the reading frame of DMD mRNA. Restoring the reading frame allows the production of partially functional dystrophin proteins, and result in less severe symptoms. Antisense oligonucl...

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Detalles Bibliográficos
Autores principales: Lyu, Pin, Yoo, Kyung Whan, Yadav, Manish Kumar, Atala, Anthony, Aartsma-Rus, Annemieke, van Putten, Maaike, Duan, Dongsheng, Lu, Baisong
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2020
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7514106/
https://www.ncbi.nlm.nih.gov/pubmed/32970732
http://dx.doi.org/10.1371/journal.pone.0239468

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7514106/
https://www.ncbi.nlm.nih.gov/pubmed/32970732
http://dx.doi.org/10.1371/journal.pone.0239468

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