Promoter considerations in the design of lentiviral vectors for use in treating lysosomal storage diseases.
gene promoter
gene therapy
lentivirus
lysosomal storage disease
stem cell therapy
Journal
Molecular therapy. Methods & clinical development
ISSN: 2329-0501
Titre abrégé: Mol Ther Methods Clin Dev
Pays: United States
ID NLM: 101624857
Informations de publication
Date de publication:
10 Mar 2022
10 Mar 2022
Historique:
entrez:
3
1
2022
pubmed:
4
1
2022
medline:
4
1
2022
Statut:
epublish
Résumé
More than 50 lysosomal storage diseases (LSDs) are associated with lysosomal dysfunctions with the frequency of 1:5,000 live births. As a result of missing enzyme activity, the lysosome dysfunction accumulates undegraded or partially degraded molecules, affecting the entire body. Most of them are life-threatening diseases where patients could die within the first or second decade of life. Approximately 20 LSDs have the approved treatments, which do not provide the cure for the disorder. Therefore, the delivery of missing genes through gene therapy is a promising approach for LSDs. Over the years,
Identifiants
pubmed: 34977274
doi: 10.1016/j.omtm.2021.11.007
pii: S2329-0501(21)00179-0
pmc: PMC8688940
doi:
Types de publication
Journal Article
Review
Langues
eng
Pagination
71-87Subventions
Organisme : NICHD NIH HHS
ID : R01 HD102545
Pays : United States
Informations de copyright
© 2021 The Author(s).
Déclaration de conflit d'intérêts
The authors declare no conflict of interest.
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