Disease-specific induced pluripotent stem (iPS) cells

By In-Hyun Park, Natasha Arora, Hongguang Huo, Nimet Maherali, Tim Ahfeldt, A. Shimamura, M. Lensch, Chad A. Cowan, K. Hochedlinger, George Q. Daley. Cell, 2008

2,162 citations recorded by the source corpus.

Tissue culture of immortal cell strains from diseased patients is an invaluable resource for medical research, but is largely limited to tumor cell lines or transformed derivatives of native tissues. Here we describe the generation of induced pluripotent stem (iPS) cells from patients with a variety of genetic diseases with either Mendelian or complex inheritance that include: adenosine deaminase deficiency-related severe combined immunodeficiency (ADA-SCID), Shwachman-Bodian-Diamond syndrome (SBDS), Gaucher disease (GD) type III, Duchenne (DMD) and Becker muscular dystrophy (BMD), Parkinson disease (PD), Huntington disease (HD), juvenile-onset, type 1 diabetes mellitus (JDM), Down syndrome (DS)/trisomy 21 and the carrier state of Lesch-Nyhan syndrome. Such patient-specific stem cells offer an unprecedented opportunity to recapitulate both normal and pathologic human tissue formation in vitro, thereby enabling disease investigation and drug development.

Subfields: Genetics, Molecular Biology

View Paper: https://www.semanticscholar.org/paper/d1aee6446762435af8cd89e06d371282cc69ba93

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In Genetics

  1. K. Hochedlinger
  2. Kathrin Plath
  3. Gene W. Yeo
  4. Fred H. Gage
  5. Christopher K. Glass
  6. S. Yamanaka
  7. Helen M. Blau
  8. A. Cheng

In Molecular Biology

  1. K. Hochedlinger
  2. S. Yamanaka
  3. R. Jaenisch
  4. Kathrin Plath
  5. G. Daley
  6. Daisy A. Robinton
  7. Helen M. Blau
  8. Fred H. Gage

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