Cas9-AAV6-engineered human mesenchymal stromal cells improved cutaneous wound healing in diabetic mice

Nat Commun. 2020 May 18;11(1):2470. doi: 10.1038/s41467-020-16065-3.

Abstract

Human mesenchymal stromal cells (hMSCs) are a promising source for engineered cell-based therapies in which genetic engineering could enhance therapeutic efficacy and install novel cellular functions. Here, we describe an optimized Cas9-AAV6-based genome editing tool platform for site-specific mutagenesis and integration of up to more than 3 kilobases of exogenous DNA in the genome of hMSCs derived from the bone marrow, adipose tissue, and umbilical cord blood without altering their ex vivo characteristics. We generate safe harbor-integrated lines of engineered hMSCs and show that engineered luciferase-expressing hMSCs are transiently active in vivo in wound beds of db/db mice. Moreover, we generate PDGF-BB- and VEGFA-hypersecreting hMSC lines as short-term, local wound healing agents with superior therapeutic efficacy over wildtype hMSCs in the diabetic mouse model without replacing resident cells long-term. This study establishes a precise genetic engineering platform for genetic studies of hMSCs and development of engineered hMSC-based therapies.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • CRISPR-Associated Protein 9 / metabolism*
  • Cell Proliferation
  • Cell Survival
  • Cross-Linking Reagents / chemistry
  • Dependovirus
  • Diabetes Mellitus, Experimental / pathology*
  • Diabetes Mellitus, Experimental / therapy*
  • Gene Editing
  • Green Fluorescent Proteins / metabolism
  • Humans
  • Hydrogels / chemistry
  • Kinetics
  • Mesenchymal Stem Cell Transplantation*
  • Mesenchymal Stem Cells / metabolism*
  • Mice
  • Proto-Oncogene Proteins c-sis
  • Skin / pathology*
  • Vascular Endothelial Growth Factor A / metabolism
  • Wound Healing*
  • Xenograft Model Antitumor Assays

Substances

  • Cross-Linking Reagents
  • Hydrogels
  • Proto-Oncogene Proteins c-sis
  • Vascular Endothelial Growth Factor A
  • Green Fluorescent Proteins
  • CRISPR-Associated Protein 9