Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mouse model of multiple sulfatase deficiency-Reference-Cited by-同舟云学术

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Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mouse model of multiple sulfatase deficiency

Published:2024-08 Issue: Volume: Page:
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Pham Vi,Tricoli Lucas,Hong Xinying,Wongkittichote Parith,Castruccio Castracani Carlo,Guerra Amaliris,Schlotawa Lars,Adang Laura A.,Kuhs Amanda,Cassidy Margaret M.,Kane Owen,Tsai Emily,Presa Maximiliano,Lutz Cathleen,Rivella Stefano B.,Ahrens-Nicklas Rebecca C.

Publisher

Elsevier BV

Reference60 articles.

1. A systematic cross-sectional survey of multiple sulfatase deficiency;Cappuccio;Mol. Genet. Metab.,2020

2. Multiple sulfatase deficiency is caused by mutations in the gene encoding the human C(alpha)-formylglycine generating enzyme;Dierks;Cell,2003

3. The multiple sulfatase deficiency gene encodes an essential and limiting factor for the activity of sulfatases;Cosma;Cell,2003

4. Molecular and functional analysis of SUMF1 mutations in multiple sulfatase deficiency;Cosma;Hum. Mutat.,2004

5. Molecular basis for multiple sulfatase deficiency and mechanism for formylglycine generation of the human formylglycine-generating enzyme;Dierks;Cell,2005

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