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

Compound heterozygous recessive or polygenic diseases could be addressed through gene correction of multiple alleles. However, targeting of multiple alleles using genome editors could lead to mixed genotypes and adverse events that amplify during tissue morphogenesis. Here we demonstrate that Cas9-r...

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Bibliographic Details
Published in:Nat Commun
Main Authors: Carlson-Stevermer, Jared, Das, Amritava, Abdeen, Amr A., Fiflis, David, Grindel, Benjamin I, Saxena, Shivani, Akcan, Tugce, Alam, Tausif, Kletzien, Heidi, Kohlenberg, Lucille, Goedland, Madelyn, Dombroe, Micah J., Saha, Krishanu
Format: Artigo
Language:Inglês
Published: Nature Publishing Group UK 2020
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Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC7722885/
https://ncbi.nlm.nih.gov/pubmed/33293555
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41467-020-20065-8
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