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...

Descripción completa

Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7722885/
https://www.ncbi.nlm.nih.gov/pubmed/33293555
http://dx.doi.org/10.1038/s41467-020-20065-8

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7722885/
https://www.ncbi.nlm.nih.gov/pubmed/33293555
http://dx.doi.org/10.1038/s41467-020-20065-8

Ejemplares similares