Idioma:

Lack of Immunotoxicity After Regional Intravenous (RI) Delivery of rAAV to Nonhuman Primate Skeletal Muscle

Toromanoff, Alice ; Adjali, Oumeya ; Larcher, Thibaut ; Hill, Marcelo ; Guigand, Lydie ; Chenuaud, Pierre ; Deschamps, Jack-Yves ; Gauthier, Olivier ; Blancho, Gilles ; Vanhove, Bernard ; Rolling, Fabienne ; Chérel, Yan ; Moullier, Philippe ; Anegon, Ignacio ; Le Guiner, Caroline

Molecular therapy, 2010-01, Vol.18 (1), p.151-160 [Periódico revisado por pares]

United States: Elsevier Inc

Texto completo disponível

Citações Citado por

Enviar para

Título:
Lack of Immunotoxicity After Regional Intravenous (RI) Delivery of rAAV to Nonhuman Primate Skeletal Muscle
Autor: Toromanoff, Alice ; Adjali, Oumeya ; Larcher, Thibaut ; Hill, Marcelo ; Guigand, Lydie ; Chenuaud, Pierre ; Deschamps, Jack-Yves ; Gauthier, Olivier ; Blancho, Gilles ; Vanhove, Bernard ; Rolling, Fabienne ; Chérel, Yan ; Moullier, Philippe ; Anegon, Ignacio ; Le Guiner, Caroline
Assuntos: Animals ; Antibodies ; Biopsy ; Cytotoxicity ; Dependovirus - genetics ; Dependovirus - immunology ; Enzyme-Linked Immunosorbent Assay ; Genetic Vectors - administration & dosage ; Genetic Vectors - genetics ; Genetic Vectors - immunology ; Immunohistochemistry ; In Situ Hybridization ; Injections, Intramuscular - adverse effects ; Injections, Intravenous - adverse effects ; Life Sciences ; Macaca ; Male ; Monkeys & apes ; Muscle Fibers, Skeletal - drug effects ; Muscle Fibers, Skeletal - pathology ; Muscle, Skeletal - drug effects ; Muscle, Skeletal - metabolism ; Musculoskeletal system ; Original ; Reverse Transcriptase Polymerase Chain Reaction ; Transduction, Genetic - methods
É parte de: Molecular therapy, 2010-01, Vol.18 (1), p.151-160
Notas: PMCID: PMC2839209
The first two authors contributed equally to this work.
Descrição: In the absence of an immune response from the host, intramuscular (IM) injection of recombinant adeno-associated virus (rAAV) results in the permanent expression of the transgene from mouse to primate models. However, recent gene transfer studies into animal models and humans indicate that the risk of transgene and/or capsid-specific immune responses occurs and depends on multiple factors. Among these factors, the route of delivery is important, although poorly addressed in large animal models. Here, we compare the IM and the drug-free regional intravenous (RI) deliveries of rAAV in nonhuman primate (NHP) skeletal muscle monitoring the host immune response toward the transgene. We show that IM is consistently associated with immunotoxicity and the destruction of the genetically modified myofibers, whereas RI allows the stable expression of the transgene. This has important implications for the design of clinical trials for gene transfer in skeletal muscle.
Editor: United States: Elsevier Inc
Idioma: Inglês

Links

Voltar para lista de resultados

Anterior Resultado 4 Avançar Ir para próxima página

Realização: Logos de Redes Sociais:

Lack of Immunotoxicity After Regional Intravenous (RI) Delivery of rAAV to Nonhuman Primate Skeletal Muscle

Toromanoff, Alice ; Adjali, Oumeya ; Larcher, Thibaut ; Hill, Marcelo ; Guigand, Lydie ; Chenuaud, Pierre ; Deschamps, Jack-Yves ; Gauthier, Olivier ; Blancho, Gilles ; Vanhove, Bernard ; Rolling, Fabienne ; Chérel, Yan ; Moullier, Philippe ; Anegon, Ignacio ; Le Guiner, Caroline

United States: Elsevier Inc

Buscando em bases de dados remotas. Favor aguardar.