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

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

doi:10.1038/s41467-020-20065-8

Nature Communications (Dec 2020)

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

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

Affiliations

Jared Carlson-Stevermer: Wisconsin Institute for Discovery, University of Wisconsin-Madison
Amritava Das: Wisconsin Institute for Discovery, University of Wisconsin-Madison
Amr A. Abdeen: Wisconsin Institute for Discovery, University of Wisconsin-Madison
David Fiflis: Wisconsin Institute for Discovery, University of Wisconsin-Madison
Benjamin I Grindel: Wisconsin Institute for Discovery, University of Wisconsin-Madison
Shivani Saxena: Wisconsin Institute for Discovery, University of Wisconsin-Madison
Tugce Akcan: Department of Surgery, University of Wisconsin-Madison
Tausif Alam: Department of Surgery, University of Wisconsin-Madison
Heidi Kletzien: Department of Biomedical Engineering, University of Wisconsin-Madison
Lucille Kohlenberg: Wisconsin Institute for Discovery, University of Wisconsin-Madison
Madelyn Goedland: Wisconsin Institute for Discovery, University of Wisconsin-Madison
Micah J. Dombroe: Wisconsin Institute for Discovery, University of Wisconsin-Madison
Krishanu Saha: Wisconsin Institute for Discovery, University of Wisconsin-Madison

DOI: https://doi.org/10.1038/s41467-020-20065-8
Journal volume & issue: Vol. 11, no. 1
pp. 1 – 18

Abstract

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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.

Published in Nature Communications

ISSN: 2041-1723 (Online)
Publisher: Nature Portfolio
Country of publisher: United Kingdom
LCC subjects: Science
Website: https://www.nature.com/ncomms/

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