Design of efficacious somatic cell genome editing strategies for recessive and polygenic diseases

Gene correction of multiple alleles is a promising therapeutic strategy. Here the authors present bi-allelic correction of Pompe disease and provide an in silico model for predicting in vivo somatic editing outcomes.

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Autores principales: Jared Carlson-Stevermer, Amritava Das, Amr A. Abdeen, David Fiflis, Benjamin I Grindel, Shivani Saxena, Tugce Akcan, Tausif Alam, Heidi Kletzien, Lucille Kohlenberg, Madelyn Goedland, Micah J. Dombroe, Krishanu Saha
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2020
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Acceso en línea:https://doaj.org/article/9ea7446371264a6ca5170a5851e05415
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