Promoter considerations in the design of lentiviral vectors for use in treating lysosomal storage diseases-Reference-Cited by-同舟云学术

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Promoter considerations in the design of lentiviral vectors for use in treating lysosomal storage diseases

Published:2022-03 Issue: Volume:24 Page:71-87
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Rintz Estera^ORCID,Higuchi Takashi,Kobayashi Hiroshi,Galileo Deni S.^ORCID,Wegrzyn Grzegorz^ORCID,Tomatsu Shunji^ORCID

Funder

National Institutes of Health

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference129 articles.

1. Lysosomal storage diseases;Platt;Nat. Rev. Dis. Primers,2018

2. Treatment strategies for lysosomal storage disorders;Beck;Dev. Med. Child Neurol.,2018

3. The uptake of enzymes into lysosomes: an overview;Neufeld;Birth Defects Orig. Artic. Ser.,1980

4. Retrovirus vector-mediated correction and cross-correction of lysosomal alpha-mannosidase deficiency in human and feline fibroblasts;Sun;Hum. Gene Ther.,1999

5. Clinical development of cell therapies to halt lysosomal storage diseases: results and lessons learned;Graceffa;Curr Gene Ther.,2020

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