<i>Ex vivo</i> gene therapy for lysosomal storage disorders: future perspectives-Reference-Cited by-同舟云学术

Ex vivo gene therapy for lysosomal storage disorders: future perspectives

Published:2023-03-20 Issue:4 Volume:23 Page:353-364
ISSN:1471-2598
Container-title:Expert Opinion on Biological Therapy
language:en
Short-container-title:Expert Opinion on Biological Therapy

Author:

Poletto Edina¹²^ORCID,Silva Andrew Oliveira²,Weinlich Ricardo²³^ORCID,Martin Priscila Keiko Matsumoto³,Torres Davi Coe³,Giugliani Roberto¹²^ORCID,Baldo Guilherme¹²^ORCID

Affiliation:

1. Departamento de Genética, Universidade Federal do Rio Grande do Sul (UFRGS), Porto alegre, Brazil

2. Centro de Pesquisa Experimental (CPE), Hospital de Clínicas de Porto Alegre (HCPA), Porto Alegre, Brazil

3. Centro de Ensino e Pesquisa/Pesquisa Experimental, Hospital Israelita Albert Einstein, São Paulo, Brazil

Funder

FAPERGS

Proadi-SUS

Publisher

Informa UK Limited

Subject

Clinical Biochemistry,Drug Discovery,Pharmacology

Link

https://www.tandfonline.com/doi/pdf/10.1080/14712598.2023.2192348

Reference108 articles.

1. Lysosomal storage diseases

2. Lysosomal storage disorders: The cellular impact of lysosomal dysfunction

3. Lysosomal diseases: Overview on current diagnosis and treatment

4. Lysosomal enzyme replacement therapies: Historical development, clinical outcomes, and future perspectives

5. TPC2 rescues lysosomal storage in mucolipidosis type IV , Niemann–Pick type C1, and Batten disease

Cited by 3 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Design and validation of a GMP stem cell manufacturing protocol for MPSII hematopoietic stem cell gene therapy;Molecular Therapy - Methods & Clinical Development;2024-06

2. Iron oxide-coupled CRISPR-nCas9-based genome editing assessment in mucopolysaccharidosis IVA mice;Molecular Therapy - Methods & Clinical Development;2023-12

3. Molecular Trojan Horses for treating lysosomal storage diseases;Molecular Genetics and Metabolism;2023-11