Fetal gene therapy for neurodegenerative lysosomal storage diseases-Reference-Cited by-同舟云学术

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Fetal gene therapy for neurodegenerative lysosomal storage diseases

Published:2019-02-04 Issue:3 Volume:42 Page:391-393
ISSN:0141-8955
Container-title:Journal of Inherited Metabolic Disease
language:en
Short-container-title:J Inherit Metab Dis

Author:

Baruteau Julien¹²³^ORCID,Waddington Simon N.¹⁴

Affiliation:

1. Gene Transfer Technology Group, Institute for Women's Health; University College London; London UK

2. Metabolic Medicine Department; Great Ormond Street Hospital for Children NHS Foundation Trust; London UK

3. Genetics and Genomic Medicine Programme, Great Ormond Street Institute of Child Health; University College London; London UK

4. Wits/SAMRC Antiviral Gene Therapy Research Unit, Faculty of Health Sciences; University of the Witwatersrand; Johannesburg South Africa

Funder

Medical Research Council Canada

National Institute for Health Research

Publisher

Wiley

Subject

Genetics (clinical),Genetics

Link

http://onlinelibrary.wiley.com/wol1/doi/10.1002/jimd.12018/fullpdf

Reference30 articles.

1. Miglustat therapy in the French cohort of paediatric patients with Niemann-pick disease type C;Heron;Orphanet J Rare Dis,2012

2. Intrathecal 2-hydroxypropyl-beta-cyclodextrin decreases neurological disease progression in Niemann-pick disease, type C1: a non-randomised, open-label, phase 1-2 trial;Ory;Lancet,2017

3. Study of intraventricular Cerliponase alfa for CLN2 disease;Schulz;N Engl J Med,2018

4. Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trial;Tardieu;Hum Gene Ther,2014

5. In utero transfer of adeno-associated viral vectors produces long-term factor IX levels in a Cynomolgus macaque model;Mattar;Mol Ther,2017

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