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
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/9ea7446371264a6ca5170a5851e05415
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spelling	oai:doaj.org-article:9ea7446371264a6ca5170a5851e054152021-12-02T14:16:14ZDesign of efficacious somatic cell genome editing strategies for recessive and polygenic diseases10.1038/s41467-020-20065-82041-1723https://doaj.org/article/9ea7446371264a6ca5170a5851e054152020-12-01T00:00:00Zhttps://doi.org/10.1038/s41467-020-20065-8https://doaj.org/toc/2041-1723Gene 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.Jared Carlson-StevermerAmritava DasAmr A. AbdeenDavid FiflisBenjamin I GrindelShivani SaxenaTugce AkcanTausif AlamHeidi KletzienLucille KohlenbergMadelyn GoedlandMicah J. DombroeKrishanu SahaNature PortfolioarticleScienceQENNature Communications, Vol 11, Iss 1, Pp 1-18 (2020)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q 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 Design of efficacious somatic cell genome editing strategies for recessive and polygenic diseases
description	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.
format	article
author	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
author_facet	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
author_sort	Jared Carlson-Stevermer
title	Design of efficacious somatic cell genome editing strategies for recessive and polygenic diseases
title_short	Design of efficacious somatic cell genome editing strategies for recessive and polygenic diseases
title_full	Design of efficacious somatic cell genome editing strategies for recessive and polygenic diseases
title_fullStr	Design of efficacious somatic cell genome editing strategies for recessive and polygenic diseases
title_full_unstemmed	Design of efficacious somatic cell genome editing strategies for recessive and polygenic diseases
title_sort	design of efficacious somatic cell genome editing strategies for recessive and polygenic diseases
publisher	Nature Portfolio
publishDate	2020
url	https://doaj.org/article/9ea7446371264a6ca5170a5851e05415
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Design of efficacious somatic cell genome editing strategies for recessive and polygenic diseases

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