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

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

Research output: Contribution to journalArticlepeer-review

16 Scopus citations

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.

Original languageEnglish
JournalPulmonary Circulation
Volume8
Issue number4
DOIs
StatePublished - 1 Oct 2018

Keywords

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

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