Safety and long-term efficacy of AAV1.SERCA2a using nebulizer delivery in a pig model of pulmonary hypertension.

Watanabe, Shin; Ishikawa, Kiyotake; Plataki, Maria; Bikou, Olympia; Kohlbrenner, Erik; Aguero, Jaume; Hadri, Lahouaria; Zarragoikoetxea, Iratxe; Fish, Kenneth; Leopold, Jane A; Hajjar, Roger J.

Afiliação

Watanabe S; 1 Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Ishikawa K; 1 Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Plataki M; 1 Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Bikou O; 2 Weill Cornell Medicine, Pulmonary & Critical Care Medicine, New York, NY, USA.
Kohlbrenner E; 1 Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Aguero J; 1 Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Hadri L; 1 Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Zarragoikoetxea I; 3 Hospital Universitari i Politecnic La Fe, Valencia, Spain.
Fish K; 1 Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Leopold JA; 1 Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Hajjar RJ; 3 Hospital Universitari i Politecnic La Fe, Valencia, Spain.

Pulm Circ ; 8(4): 2045894018799738, 2018.

Article em En | MEDLINE | ID: mdl-30129881

ABSTRACT

Nebulization delivery of adeno-associated virus serotype 1 encoding sarcoplasmic reticulum Ca2+-ATPase2a (AAV1.SERCA2a) gene was examined in a Yukatan miniature swine model of chronic pulmonary hypertension (n = 13). Nebulization of AAV1.SERCA2a resulted in homogenous distribution of vectors, lower pulmonary vascular resistance, and a trend towards better long-term survival compared to control animals.

Palavras-chave

airway delivery; gene therapy; nebulization; post-capillary pulmonary hypertension

Texto completo: 1 Base de dados: MEDLINE Idioma: En Ano de publicação: 2018 Tipo de documento: Article