Review

Gene Therapy (2006) 13, 478–486. doi:10.1038/sj.gt.3302723; published online 16 February 2006

RNA interference in embryonic stem cells and the prospects for future therapies

A Heidersbach1, A Gaspar-Maia1,2, M T McManus1 and M Ramalho-Santos1,2

  1. 1Diabetes Center, University of California, San Francisco, CA, USA
  2. 2Program in Developmental and Stem Cell Biology, University of California, San Francisco, CA, USA

Correspondence: Dr M Ramalho-Santos, Program in Developmental and Stem Cell Biology, UCSF, 513 Parnassus Avenue, HSW1201G, San Francisco, CA 94143-0525, USA. E-mail: mrsantos@diabetes.ucsf.edu

Received 17 August 2005; Revised 21 December 2005; Accepted 3 January 2006; Published online 16 February 2006.

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Abstract

In 1998, two distinct and exciting scientific fields emerged which have profoundly shaped the current direction of biomedical research. The discovery of RNA interference (RNAi) and the derivation of human embryonic stem (ES) cells have yielded exciting new possibilities for researchers and clinicians alike. While fundamentally different, aspects from these two fields may be combined to yield extraordinary scientific and medical benefits. Here, we review the prospects of combining RNAi and ES cell manipulation for both basic research and future therapies, as well as current limitations and obstacles that need to be overcome.

Keywords:

miRNA, ES cells, RNAi, siRNA, differentiation, cell-replacement therapy

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