Genome editing in lysosomal disorders.
| Autor: | Pimentel-Vera LN; Postgraduate Program in Genetics and Molecular Biology, UFRGS, Porto Alegre, Brazil; Gene Therapy Center, HCPA, Porto Alegre, Brazil., Poletto E; Postgraduate Program in Genetics and Molecular Biology, UFRGS, Porto Alegre, Brazil; Gene Therapy Center, HCPA, Porto Alegre, Brazil., Gonzalez EA; Postgraduate Program in Genetics and Molecular Biology, UFRGS, Porto Alegre, Brazil; Gene Therapy Center, HCPA, Porto Alegre, Brazil., de Oliveira Poswar F; Medical Genetics Service, HCPA, Porto Alegre, Brazil., Giugliani R; Postgraduate Program in Genetics and Molecular Biology, UFRGS, Porto Alegre, Brazil; Gene Therapy Center, HCPA, Porto Alegre, Brazil; Medical Genetics Service, HCPA, Porto Alegre, Brazil; INAGEMP, Porto Alegre, Brazil; Biodiscovery Research Group, Experimental Research Center, HCPA, Porto Alegre, Brazil; Department of Genetics, UFRGS, Porto Alegre, Brazil., Baldo G; Postgraduate Program in Genetics and Molecular Biology, UFRGS, Porto Alegre, Brazil; Gene Therapy Center, HCPA, Porto Alegre, Brazil; Biodiscovery Research Group, Experimental Research Center, HCPA, Porto Alegre, Brazil; Department of Physiology, UFRGS, Porto Alegre, Brazil. Electronic address: gbaldo@hcpa.edu.br. |
|---|---|
| Jazyk: | angličtina |
| Zdroj: | Progress in molecular biology and translational science [Prog Mol Biol Transl Sci] 2021; Vol. 182, pp. 289-325. Date of Electronic Publication: 2021 Feb 22. |
| DOI: | 10.1016/bs.pmbts.2021.02.001 |
| Abstrakt: | Lysosomal disorders are a group of heterogenous diseases caused by mutations in genes that encode for lysosomal proteins. With exception of some cases, these disorders still lack both knowledge of disease pathogenesis and specific therapies. In this sense, genome editing arises as a technique that allows both the creation of specific cell lines, animal models and gene therapy protocols for these disorders. Here we explain the main applications of genome editing for lysosomal diseases, with examples based on the literature. The ability to rewrite the genome will be of extreme importance to study and potentially treat these rare disorders. (Copyright © 2021 Elsevier Inc. All rights reserved.) |
| Databáze: | MEDLINE |
| Externí odkaz: |
|