Safe and Sustained Expression of Human Iduronidase After Intrathecal Administration of Adeno-Associated Virus Serotype 9 in Infant Rhesus Monkeys-Reference-Cited by-同舟云学术

Safe and Sustained Expression of Human Iduronidase After Intrathecal Administration of Adeno-Associated Virus Serotype 9 in Infant Rhesus Monkeys

Published:2019-08 Issue:8 Volume:30 Page:957-966
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Hordeaux Juliette¹,Hinderer Christian¹,Buza Elizabeth L.¹,Louboutin Jean-Pierre²,Jahan Tahsin¹,Bell Peter¹,Chichester Jessica A.¹,Tarantal Alice F.³,Wilson James M.¹

Affiliation:

1. Gene Therapy Program, Department of Medicine, University of Pennsylvania, Perelman School of Medicine, Philadelphia, Pennsylvania

2. Section of Anatomy, Department of Basic Medical Sciences, University of West Indies, Kingston, Jamaica

3. Center for Fetal Monkey Gene Transfer for Heart, Lung, and Blood Diseases, Departments of Pediatrics and Cell Biology and Human Anatomy, School of Medicine, and California National Primate Research Center, University of California, Davis, California.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2019.012

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4. Long-term Efficacy and Safety of Laronidase in the Treatment of Mucopolysaccharidosis I

5. A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 years

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