Human embryonic stem cells and gene therapy

Yael Strulovici, Philip L. Leopold, Timothy P. O'Connor, Robert G. Pergolizzi, Ronald Crystal

Research output: Contribution to journalReview article

42 Citations (Scopus)


Human embryonic stem cells (hESCs) theoretically represent an unlimited supply of normal differentiated cells to engineer diseased tissues to regain normal function. However, before hESCs can be useful as human therapeutics, technologies must be developed to provide them with the specific signals required to differentiate in a controlled fashion, to regulate and/or shut down the growth of hESCs and their progeny once they have been transferred to the recipient, and to circumvent the recognition of non-autologous hESC-derived cells as foreign. In the context that gene therapy technologies represent strategies to deliver biological signals to address all of these challenges, this review sets out a framework for combined gene transfer/hESC therapies. We discuss how hESCs are derived, characterized, and differentiated into specific cell lineages, and we summarize the characteristics of the 500 hESC lines reported to date. The successes and failures of gene transfer to hESCs are reviewed for both non-viral and viral vectors, as are the challenges to successful use of gene transfer in developing hESC therapy. We also consider gene transfer as a means of facilitating growth and isolation of genetically modified hESCs and as a mechanism for mitigating adverse effects associated with administration of hESCs or their derivatives. Finally, we evaluate the challenges that are likely to be encountered in translating the promise of hESCs to the clinic.

Original languageEnglish
Pages (from-to)850-866
Number of pages17
JournalMolecular Therapy
Issue number5
Publication statusPublished - 1 May 2007
Externally publishedYes


ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology
  • Genetics
  • Pharmacology
  • Drug Discovery

Cite this

Strulovici, Y., Leopold, P. L., O'Connor, T. P., Pergolizzi, R. G., & Crystal, R. (2007). Human embryonic stem cells and gene therapy. Molecular Therapy, 15(5), 850-866.