Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN-Reference-Cited by-同舟云学术

Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN

Published:2018-03 Issue:3 Volume:29 Page:285-298
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Hinderer Christian¹,Katz Nathan¹,Buza Elizabeth L.¹,Dyer Cecilia¹,Goode Tamara¹,Bell Peter¹,Richman Laura K.¹,Wilson James M.¹

Affiliation:

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

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2018.015

Reference34 articles.

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3. Retrograde Viral Delivery of IGF-1 Prolongs Survival in a Mouse ALS Model

4. Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1G93A Mice and Nonhuman Primates

5. Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates

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