Brain and visceral gene editing of mucopolysaccharidosis I mice by nasal delivery of the CRISPR/Cas9 system-Reference-Cited by-同舟云学术

Brain and visceral gene editing of mucopolysaccharidosis I mice by nasal delivery of the CRISPR/Cas9 system

Published:2022-01-31 Issue:4 Volume:24 Page:
ISSN:1099-498X
Container-title:The Journal of Gene Medicine
language:en
Short-container-title:The Journal of Gene Medicine

Author:

Vera Luisa Natalia Pimentel¹²^ORCID,Schuh Roselena Silvestri¹³,Fachel Flavia Nathielly Silveira³,Poletto Edina¹²,Piovesan Eduarda¹³,Kubaski Francyne⁴,Couto Eduarda³,Brum Bruna³,Rodrigues Graziella¹²,Souza Hallana¹²,Giugliani Roberto¹²⁴,Matte Ursula¹²,Baldo Guilherme¹²⁵,Teixeira Helder F.³

Affiliation:

1. Centro de Terapia Gênica do Hospital de Clinicas de Porto Alegre Porto Alegre RS Brazil

2. Departamento de Genética Programa de Pós‐Graduação em Genética e Biologia Molecular da Universidade Federal do Rio Grande do Sul (UFRGS) Porto Alegre RS Brazil

3. Faculdade de Farmácia Programa de Pós‐Graduação em Ciências Farmacêuticas da Universidade Federal do Rio Grande do Sul (UFRGS) Porto Alegre RS Brazil

4. Serviço de Genética Medica Hospital de Clinicas de Porto Alegre Porto Alegre RS Brazil

5. Programa de Pós‐Graduação em Fisiologia da Universidade Federal do Rio Grande do Sul (UFRGS) Instituto de Ciências Básicas da Saúde Porto Alegre RS Brazil

Funder

Coordenação de Aperfeiçoamento de Pessoal de Nível Superior

Conselho Nacional de Desenvolvimento Científico e Tecnológico

Fundação de Amparo à Pesquisa do Estado do Rio Grande do Sul

Publisher

Wiley

Subject

Genetics (clinical),Drug Discovery,Genetics,Molecular Biology,Molecular Medicine

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/jgm.3410

Reference37 articles.

1. Mucopolysaccharidosis Type I

2. Long-term outcomes of systemic therapies for Hurler syndrome: an international multicenter comparison

3. Hematopoietic cell transplantation for severe MPS I in the first six months of life: The heart of the matter

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