Protease-Activatable Adeno-Associated Virus Vector for Gene Delivery to Damaged Heart Tissue

Caitlin M. Guenther, Mitchell J. Brun, Antonette D. Bennett, Michelle L. Ho, Weitong Chen, Banghe Zhu, Michael Lam, Momona Yamagami, Sunkuk Kwon, Nilakshee Bhattacharya, Duncan Sousa, Annicka C. Evans, Julie Voss, Eva M. Sevick-Muraca, Mavis Agbandje-McKenna, Junghae Suh

Research output: Contribution to journalArticlepeer-review

Abstract

Guenther et al. have developed a protease-activatable AAV provector to target diseased tissue microenvironments exhibiting elevated extracellular protease activity. The provector delivers transgenes to high-MMP-activity regions of the damaged heart following a myocardial infarction, with concomitant decreased delivery to many off-target organs, including the liver.

Original languageEnglish (US)
Pages (from-to)611-622
Number of pages12
JournalMolecular Therapy
Volume27
Issue number3
DOIs
StatePublished - Mar 6 2019
Externally publishedYes

Keywords

  • AAV
  • activatable
  • adeno-associated virus
  • cardiac gene therapy
  • gene delivery
  • gene therapy
  • inflammation targeting
  • matrix metalloproteinase
  • myocardial infarction
  • provector
  • stimulus responsive
  • viral vector

ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology
  • Genetics
  • Pharmacology
  • Drug Discovery

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