Ocular Tolerability and Immune Response to Corneal Intrastromal AAV-IDUA Gene Therapy in New Zealand White Rabbits

Ocular Tolerability and Immune Response to Corneal Intrastromal AAV-IDUA Gene Therapy in New Zealand White Rabbits

The chronic ocular toxicity, tolerability, and inflammation following corneal intrastromal injection of saline or escalating doses of an adeno-associated virus (AAV) containing a codon-optimized α-l-iduronidase (AAV-opt-IDUA) expression cassette were evaluated in New Zealand White rabbits. Corneal o...

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Bibliographic Details
Main Authors: Liujiang Song, Jacquelyn J. Bower, Telmo Llanga, Jacklyn H. Salmon, Matthew L. Hirsch, Brian C. Gilger
Format: Article
Language:English
Published: Elsevier 2020-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
Adeno-Associated Virus (AAV)
Mucopolysaccharidosis (MPS)
Gene therapy
Corneal disease
Intrastromal injection
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050120301017

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http://www.sciencedirect.com/science/article/pii/S2329050120301017

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