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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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