Gene editing strategies to treat lysosomal disorders: The example of mucopolysaccharidoses.

Animals Gene Editing Mucopolysaccharidoses / genetics Lysosomal Storage Diseases / therapy Enzyme Replacement Therapy / methods Lysosomes
CRISPR/Cas Gene delivery methods Gene editing Mucopolysaccharidoses TALEN Zinc finger

Journal

Advanced drug delivery reviews
ISSN: 1872-8294
Titre abrégé: Adv Drug Deliv Rev
Pays: Netherlands
ID NLM: 8710523

Informations de publication

Date de publication:
12 2022
Historique:
received: 13 02 2022
revised: 20 09 2022
accepted: 02 11 2022
pubmed: 11 11 2022
medline: 2 12 2022
entrez: 10 11 2022
Statut: ppublish

Résumé

Lysosomal storage disorders are a group of progressive multisystemic hereditary diseases with a combined incidence of 1:4,800. Here we review the clinical and molecular characteristics of these diseases, with a special focus on Mucopolysaccharidoses, caused primarily by the lysosomal storage of glycosaminoglycans. Different gene editing techniques can be used to ameliorate their symptoms, using both viral and nonviral delivery methods. Whereas these are still being tested in animal models, early results of phase I/II clinical trials of gene therapy show how this technology may impact the future treatment of these diseases. Hurdles related to specific hard-to-reach organs, such as the central nervous system, heart, joints, and the eye must be tackled. Finally, the regulatory framework necessary to advance into clinical practice is also discussed.

Identifiants

DOI: 10.1016/j.addr.2022.114616 PMID: 36356930
pubmed: 36356930
pii: S0169-409X(22)00506-3
doi: 10.1016/j.addr.2022.114616
pii:
doi:

Types de publication

Journal Article Review Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

114616

Informations de copyright

Copyright © 2022 Elsevier B.V. All rights reserved.

Déclaration de conflit d'intérêts

Declaration of Competing Interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.

Auteurs

Flávia Nathiely Silveira Fachel (FNS)

Laboratório de Células, Tecidos e Genes - Centro de Pesquisa Experimental, Hospital de Clínicas de Porto Alegre, Porto Alegre, RS, Brazil; Programa de Pós-Graduação em Ciências Farmacêuticas, Faculdade de Farmácia, UFRGS, Porto Alegre, RS, Brazil.

Lariane Frâncio (L)

Laboratório de Células, Tecidos e Genes - Centro de Pesquisa Experimental, Hospital de Clínicas de Porto Alegre, Porto Alegre, RS, Brazil; Programa de Pós-Graduação em Genética e Biologia Molecular, UFRGS, Porto Alegre, RS, Brazil.

Édina Poletto (É)

Laboratório de Células, Tecidos e Genes - Centro de Pesquisa Experimental, Hospital de Clínicas de Porto Alegre, Porto Alegre, RS, Brazil.

Roselena Silvestri Schuh (RS)

Laboratório de Células, Tecidos e Genes - Centro de Pesquisa Experimental, Hospital de Clínicas de Porto Alegre, Porto Alegre, RS, Brazil; Programa de Pós-Graduação em Ciências Farmacêuticas, Faculdade de Farmácia, UFRGS, Porto Alegre, RS, Brazil.

Helder Ferreira Teixeira (HF)

Programa de Pós-Graduação em Ciências Farmacêuticas, Faculdade de Farmácia, UFRGS, Porto Alegre, RS, Brazil.

Roberto Giugliani (R)

Programa de Pós-Graduação em Genética e Biologia Molecular, UFRGS, Porto Alegre, RS, Brazil; Serviço de Genética Médica, Hospital de Clínicas de Porto Alegre, Porto Alegre, RS, Brazil; Departamento de Genética, UFRGS, Porto Alegre, RS, Brazil.

Guilherme Baldo (G)

Laboratório de Células, Tecidos e Genes - Centro de Pesquisa Experimental, Hospital de Clínicas de Porto Alegre, Porto Alegre, RS, Brazil; Programa de Pós-Graduação em Genética e Biologia Molecular, UFRGS, Porto Alegre, RS, Brazil; Departamento de Fisiologia, UFRGS, Porto Alegre, RS, Brazil.

Ursula Matte (U)

Laboratório de Células, Tecidos e Genes - Centro de Pesquisa Experimental, Hospital de Clínicas de Porto Alegre, Porto Alegre, RS, Brazil; Programa de Pós-Graduação em Genética e Biologia Molecular, UFRGS, Porto Alegre, RS, Brazil; Departamento de Genética, UFRGS, Porto Alegre, RS, Brazil. Electronic address: umatte@hcpa.edu.br.

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Classifications MeSH

questionsmedicales.fr › Eucaryotes › Animaux › Gene editing strategies to treat lysosomal...
questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Génie génétique › Édition de gène › Gene editing strategies to treat lysosomal...
questionsmedicales.fr › Maladies métaboliques et nutritionnelles › Maladies métaboliques › Erreurs innées du métabolisme › Maladies lysosomiales › Mucopolysaccharidoses › Gene editing strategies to treat lysosomal...
questionsmedicales.fr › Maladies métaboliques et nutritionnelles › Maladies métaboliques › Erreurs innées du métabolisme › Maladies lysosomiales › Gene editing strategies to treat lysosomal...
questionsmedicales.fr › Thérapeutique › Traitement médicamenteux › Thérapie enzymatique › Thérapie enzymatique substitutive › Gene editing strategies to treat lysosomal...
questionsmedicales.fr › Cellules › Structures cellulaires › Espace intracellulaire › Cytoplasme › Structures cytoplasmiques › Organites › Vésicules cytoplasmiques › Lysosomes › Gene editing strategies to treat lysosomal...