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J Orthop Res. 2019 Jun;37(6):1287-1293. doi: 10.1002/jor.24304. Epub 2019 May 2.

Designer Stem Cells: Genome Engineering and the Next Generation of Cell-Based Therapies.

Guilak F1,2,3, Pferdehirt L1,2,3, Ross AK1,2,3, Choi YR1,2,4, Collins K1,2, Nims RJ1,2, Katz DB1,2,3, Klimak M1,2,3, Tabbaa S5, Pham CTN6.

Author information

1
Department of Orthopaedic Surgery, Washington University, St. Louis, Missouri.
2
Shriners Hospitals for Children-St. Louis, St. Louis, Missouri.
3
Department of Biomedical Engineering, Washington University, St. Louis, Missouri.
4
Department of Orthopaedic Surgery, Yonsei University College of Medicine, Seoul, South Korea.
5
SymplexBio Consulting LLC, Flagstaff, Arizona.
6
Department of Medicine, Division of Rheumatology, Washington University, St. Louis, Missouri.

Abstract

Stem cells provide tremendous promise for the development of new therapeutic approaches for musculoskeletal conditions. In addition to their multipotency, certain types of stem cells exhibit immunomodulatory effects that can mitigate inflammation and enhance tissue repair. However, the translation of stem cell therapies to clinical practice has proven difficult due to challenges in intradonor and interdonor variability, engraftment, variability in recipient microenvironment and patient indications, and limited therapeutic biological activity. In this regard, the success of stem cell-based therapies may benefit from cellular engineering approaches to enhance factors such as purification, homing and cell survival, trophic effects, or immunomodulatory signaling. By combining recent advances in gene editing, synthetic biology, and tissue engineering, the potential exists to create new classes of "designer" cells that have prescribed cell-surface molecules and receptors as well as synthetic gene circuits that provide for autoregulated drug delivery or enhanced tissue repair. Published by Wiley Periodicals, Inc. J Orthop Res 37:1287-1293, 2019.

KEYWORDS:

CRISPR-Cas9; MSC; iPSC; regenerative medicine; synthetic biology

PMID:
30977548
PMCID:
PMC6546536
[Available on 2020-06-01]
DOI:
10.1002/jor.24304
[Indexed for MEDLINE]

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