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Gene therapy offers new hope for children with metachromatic leukodystrophy.

Authors :
Kurtzberg J
Source :
Lancet (London, England) [Lancet] 2022 Jan 22; Vol. 399 (10322), pp. 338-339.
Publication Year :
2022

Abstract

Competing Interests: JK is a consultant for Neurogene, a company developing gene therapy for Krabbe disease. She is also developing a cell therapy using a microglial/macrophage cell manufactured from cord blood and under study funded by the Marcus Foundation. This technology was licensed by her institution to CryoCell International.

Subjects

Subjects :
Child
Genetic Therapy
Humans
Hematopoietic Stem Cell Transplantation
Leukodystrophy, Metachromatic genetics
Leukodystrophy, Metachromatic therapy

Details

Language :
English
ISSN :
1474-547X
Volume :
399
Issue :
10322
Database :
MEDLINE
Journal :
Lancet (London, England)
Publication Type :
Academic Journal
Accession number :
35065771
Full Text :
https://doi.org/10.1016/S0140-6736(22)00057-5
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