Fusion of Rabies Virus Glycoprotein or gh625 to Iduronate-2-Sulfatase for the Treatment of Mucopolysaccharidosis Type II-Reference-Cited by-同舟云学术

Fusion of Rabies Virus Glycoprotein or gh625 to Iduronate-2-Sulfatase for the Treatment of Mucopolysaccharidosis Type II

Published:2023-07-07 Issue: Volume: Page:
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Wood Shaun R.¹^ORCID,Chaudrhy Ahsan¹,Ellison Stuart¹,Searle Rachel¹,Burgod Constance¹,Tehseen Ghazala¹,Forte Gabriella¹,O'Leary Claire¹,Gleitz Hélène¹²,Liao Aiyin¹³,Cook James¹,Holley Rebecca¹,Bigger Brian W.¹^ORCID

Affiliation:

1. Division of Cell Matrix Biology and Regenerative Medicine, University of Manchester, Manchester, United Kingdom

2. Department of Developmental Biology, Erasmus University, Rotterdam, the Netherlands.

3. Department of Infection, Immunity and Inflammation,University College London, London, United Kingdom.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2023.025

Reference13 articles.

1. Lysosomal disorders

2. Delivering gene therapy for mucopolysaccharide diseases

3. Brain‐targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanisms

4. Busulfan Conditioning Enhances Engraftment of Hematopoietic Donor-derived Cells in the Brain Compared With Irradiation

5. Transvascular delivery of small interfering RNA to the central nervous system

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1. An empowered, clinically viable hematopoietic stem cell gene therapy for the treatment of multisystemic mucopolysaccharidosis type II;Molecular Therapy;2024-03