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.

1. Differences in MPS I and MPS II Disease Manifestations

2. Gene Therapy for Mucopolysaccharidosis Type II—A Review of the Current Possibilities

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)

Cited by 9 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Engineering Memory T Cells as a platform for Long-Term Enzyme Replacement Therapy in Lysosomal Storage Disorders;2024-04-28

2. Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice;Molecular Therapy - Methods & Clinical Development;2024-03

3. Lentiviral Gene Therapy for Mucopolysaccharidosis II with Tagged Iduronate 2-Sulfatase Prevents Life-Threatening Pathology in Peripheral Tissues But Fails to Correct Cartilage;Human Gene Therapy;2024-02-02

4. Tagged IDS causes efficient and engraftment-independent prevention of brain pathology during lentiviral gene therapy for Mucopolysaccharidosis type II;Molecular Therapy - Methods & Clinical Development;2023-12

5. Sustained long-term disease correction in a murine model of MPSII following stem cell gene therapy;Molecular Therapy - Methods & Clinical Development;2023-12