Safety of Same-Eye Subretinal Sequential Readministration of

AAV OCT RPE65 adeno-associated virus gene therapy monkey non-human primate readministration retina

Journal

Molecular therapy. Methods & clinical development
ISSN: 2329-0501
Titre abrégé: Mol Ther Methods Clin Dev
Pays: United States
ID NLM: 101624857

Informations de publication

Date de publication:
13 Dec 2019
Historique:
received: 07 07 2019
accepted: 26 08 2019
entrez: 30 10 2019
pubmed: 30 10 2019
medline: 30 10 2019
Statut: epublish

Résumé

We have demonstrated safe and effective subretinal readministration of recombinant adeno-associated virus serotype (rAAV) to the contralateral eye in large animals and humans even in the setting of preexisting neutralizing antibodies (NAbs). Readministration of AAV to the same retina may be desirable in order to treat additional areas of the retina not targeted initially or to boost transgene expression levels at a later time point. To better understand the immune and structural consequences of subretinal rAAV readministration to the same eye, we administered bilateral subretinal injections of rAAV2-

Identifiants

pubmed: 31660416
doi: 10.1016/j.omtm.2019.08.011
pii: S2329-0501(19)30091-9
pmc: PMC6807311
doi:

Types de publication

Journal Article

Langues

eng

Pagination

133-148

Subventions

Organisme : NEI NIH HHS
ID : P30 EY001583
Pays : United States

Informations de copyright

© 2019 The Authors.

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Auteurs

Lindsey Weed (L)

Center for Advanced Retinal and Ocular Therapeutics (CAROT), Department of Ophthalmology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.

Michael J Ammar (MJ)

Center for Advanced Retinal and Ocular Therapeutics (CAROT), Department of Ophthalmology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.

Shangzhen Zhou (S)

Center for Advanced Retinal and Ocular Therapeutics (CAROT), Department of Ophthalmology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.

Zhangyong Wei (Z)

Center for Advanced Retinal and Ocular Therapeutics (CAROT), Department of Ophthalmology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.

Leona W Serrano (LW)

Center for Advanced Retinal and Ocular Therapeutics (CAROT), Department of Ophthalmology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.

Junwei Sun (J)

Center for Advanced Retinal and Ocular Therapeutics (CAROT), Department of Ophthalmology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.

Vivian Lee (V)

Center for Advanced Retinal and Ocular Therapeutics (CAROT), Department of Ophthalmology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.
Department of Dermatology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.

Albert M Maguire (AM)

Center for Advanced Retinal and Ocular Therapeutics (CAROT), Department of Ophthalmology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.
Department of Dermatology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.

Jean Bennett (J)

Center for Advanced Retinal and Ocular Therapeutics (CAROT), Department of Ophthalmology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.
The Children's Hospital of Philadelphia (CHOP), Philadelphia, PA, USA.

Tomas S Aleman (TS)

Center for Advanced Retinal and Ocular Therapeutics (CAROT), Department of Ophthalmology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.
The Children's Hospital of Philadelphia (CHOP), Philadelphia, PA, USA.

Classifications MeSH