Limb Perfusion Delivery of a rAAV1 Alpha-1 Antitrypsin Vector in Non-Human Primates Is Safe but Insufficient for Therapy.

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Title: Limb Perfusion Delivery of a rAAV1 Alpha-1 Antitrypsin Vector in Non-Human Primates Is Safe but Insufficient for Therapy.
Authors: Pires-Ferreira D; Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA., Reil D; Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA., Tang Q; Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA., Blackwood M; Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA., Gallagher T; Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA., Keeler AM; Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA.; Department of Pediatrics, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA.; NeuroNexus Institute, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA., Chichester JA; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Vyhnal KK; Lovelace Biomedical Research Institute, Albuquerque, NM 87108, USA., Lindborg JA; Lovelace Biomedical Research Institute, Albuquerque, NM 87108, USA., Benson J; Lovelace Biomedical Research Institute, Albuquerque, NM 87108, USA., Fu D; Department of Pathology, Immunology, and Laboratory Medicine, University of Florida, Gainesville, FL 32610, USA., Flotte TR; Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA.; Department of Pediatrics, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA., Gruntman AM; Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA.; Department of Pediatrics, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA.
Source: Genes [Genes (Basel)] 2024 Sep 10; Vol. 15 (9). Date of Electronic Publication: 2024 Sep 10.
Publication Type: Journal Article
Journal Info: Publisher: MDPI Country of Publication: Switzerland NLM ID: 101551097 Publication Model: Electronic Cited Medium: Internet ISSN: 2073-4425 (Electronic) Linking ISSN: 20734425 NLM ISO Abbreviation: Genes (Basel) Subsets: MEDLINE
Database: MEDLINE Ultimate
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ISSN:2073-4425
DOI:10.3390/genes15091188