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 |
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Formato: | article |
Lenguaje: | EN |
Publicado: |
Nature Portfolio
2020
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Materias: | |
Acceso en línea: | https://doaj.org/article/9ea7446371264a6ca5170a5851e05415 |
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