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Transduction of hematopoietic stem and progenitor cells by an MECP2 lentiviral vector improves Rett syndrome phenotypes

IntroductionRett Syndrome is a genetic neurodevelopmental disorder caused by decreased levels of MeCP2. Due to mutations in the MECP2 gene, insufficient MeCP2 protein levels lead to clinical phenotypes including the loss of normal movement, decreased communication, seizures, sleep disorders, and bre...

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Main Authors: Joseph S. Anderson, Jill L. Silverman, Alyse L. Lodigiani, Camilla M. Barbaduomo, Julie R. Beegle
Format: Artigo
Language:Inglês
Published: Frontiers Media S.A. 2025-02-01
Series:Frontiers in Drug Discovery
Subjects:
MECP2 lentiviral vector
Rett syndrome
hematopoietic stem and progenitor cells
gene therapy
neurodevelopmental disorders
Online Access:https://www.frontiersin.org/articles/10.3389/fddsv.2025.1545391/full
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