Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of <i>Ex Vivo</i> Lentiviral Vector-Transduced Hematopoietic Stem and Progenitor Cells-Reference-Cited by-同舟云学术

Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of Ex Vivo Lentiviral Vector-Transduced Hematopoietic Stem and Progenitor Cells

Published:2022-12-01 Issue:23-24 Volume:33 Page:1279-1292
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Smith Miles C.¹^ORCID,Belur Lalitha R.¹^ORCID,Karlen Andrea D.¹,Erlanson Olivia¹,Podetz-Pedersen Kelly M.¹,McKenzie Jessica²,Detellis Jenn²,Gagnidze Khatuna²,Parsons Geoffrey²,Robinson Nicholas²,Labarre Shelby²,Shah Saumil²,Furcich Justin³,Lund Troy C.³,Tsai Hsing-Chen²,McIvor R. Scott¹,Bonner Melissa²

Affiliation:

1. Center for Genome Engineering, Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, Minnesota, USA

2. bluebird bio, Inc., Cambridge, Massachusetts, USA

3. Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2022.141

Reference43 articles.

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3. 3. Neufeld EF, Muenzer J. The Mucopolysaccharidoses. In: The Online Metabolic and Molecular Bases of Inherited Disease. (Valle DL, Antonarakis S, Ballabio A, et al. eds.) McGraw-Hill Education: New York, NY, 2019; pp. 3421–3452.

4. Comparative study of idursulfase beta and idursulfase in vitro and in vivo

5. Phase I/II clinical trial of enzyme replacement therapy with idursulfase beta in patients with mucopolysaccharidosis II (Hunter Syndrome)

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