Mesenchymal stem cells at the intersection of cell and gene therapy.
BACKGROUND
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.


METHODS
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.


RESULTS
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.


CONCLUSIONS
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.
University of North Carolina at Chapel Hill, Department of Pediatrics, Chapel Hill, NC 27599-7239, USA.
Timothy J
TJ
Myers
Froilan
F
Granero-Molto
Lara
L
Longobardi
Tieshi
T
Li
Yun
Y
Yan
Anna
A
Spagnoli
D000818:Animals; D001847:Bone Diseases / Q000235:genetics / Q000473:pathology / Q000628:therapy*; D002318:Cardiovascular Diseases / Q000235:genetics / Q000473:pathology / Q000628:therapy*; D002454:Cell Differentiation; D002465:Cell Movement; D049109:Cell Proliferation; D015316:Genetic Therapy; D006801:Humans; D045164:Mesenchymal Stem Cell Transplantation; D059630:Mesenchymal Stem Cells / Q000276:immunology / Q000378:metabolism; D009369:Neoplasms / Q000235:genetics / Q000473:pathology / Q000628:therapy*; D012038:Regeneration; D055153:Stem Cell Niche; D016896:Treatment Outcome