Stem Cells Isolated from Patients with Genetic Diseases

August 11, 2008

Stem cells can be produced from cells from patients with a variety of genetic disorders, allowing investigation into disease pathogenesis and drug development, according to research published online Aug. 7 in Cell.

MONDAY, Aug. 11 (HealthDay News) -- Stem cells can be produced from cells from patients with a variety of genetic disorders, allowing investigation into disease pathogenesis and drug development, according to research published online Aug. 7 in Cell.

In-Hyun Park, Ph.D., from Harvard Medical School in Boston, and colleagues transduced dermal fibroblasts or bone marrow-derived mesenchymal cells from patients with a range of Mendelian and complex genetic disorders with 3-4 reprogramming genes followed by cell culture to produce induced pluripotent stem (iPS) cells.

The researchers were able to produce iPS cell lines from patients with adenosine deaminase deficiency-related severe combined immunodeficiency, Shwachman-Bodian-Diamond syndrome, Gaucher disease type III, Duchenne and Becker muscular dystrophy, Parkinson's disease, Huntington's disease, juvenile-onset type 1 diabetes mellitus, Down's syndrome/trisomy 21, and the carrier state of Lesch-Nyhan syndrome. In all cases, the cells resembled embryonic stem cells based on morphology and gene expression, and could differentiate into the three embryonic germ layers.

"Such disease-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," Park and colleagues conclude.

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