Mesenchymal stem cells at the intersection of cell and gene therapy

Timothy J. Myers, Froilan Granero-Molto, Lara Longobardi, Tieshi Li, Yun Yan, Anna Spagnoli

Research output: Contribution to journalReview articlepeer-review

84 Scopus citations


Importance of the field : Mesenchymal stem cells have the ability to differentiate into osteoblasts, chondrocytes and adipocytes. Along with differentiation, MSCs can modulate inflammation, home to damaged tissues and secrete bioactive molecules. These properties can be enhanced through genetic-modification that would combine the best of both cell and gene therapy fields to treat monogenic and multigenic diseases. Areas covered in this review: Findings demonstrating the immunomodulation, homing and paracrine activities of MSCs followed by a summary of the current research utilizing MSCs as a vector for gene therapy, focusing on skeletal disorders, but also cardiovascular disease, ischemic damage and cancer. What the reader will gain: MSCs are a possible therapy for many diseases, especially those related to the musculoskeletal system, as a standalone treatment, or in combination with factors that enhance the abilities of these cells to migrate, survive or promote healing through anti-inflammatory and immunomodulatory effects, differentiation, angiogenesis or delivery of cytolytic or anabolic agents. Take home message: Genetically-modified MSCs are a promising area of research that would be improved by focusing on the biology of MSCs that could lead to identification of the natural and engrafting MSC-niche and a consensus on how to isolate and expand MSCs for therapeutic purposes.

Original languageEnglish (US)
Pages (from-to)1663-1679
Number of pages17
JournalExpert Opinion on Biological Therapy
Issue number12
StatePublished - Dec 2010


  • gene therapy
  • genetic engineering
  • genetic modifications
  • immunomodulatory
  • mesenchymal stem cell
  • paracrine effects
  • skeletal disorders

ASJC Scopus subject areas

  • Pharmacology
  • Drug Discovery
  • Clinical Biochemistry

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