Research report: Extra chromosome 21 removed from Down syndrome cell line

University of Washington News, 8th November 2012 (Leila Gray):

University of Washington scientists have succeeded in removing the extra copy of chromosome 21 in cell cultures derived from a person with Down syndrome, a condition in which the body's cells contain three copies of chromosome 21 rather than the usual pair.

In their report  a team led by Dr. Li B. Li of the UW Department of Medicine described how they corrected trisomy 21 in human cell lines they grew in the lab. The senior scientists on the project were gene therapy researchers Dr. David W. Russell, professor of medicine and biochemistry, and Dr. Thalia Papayannopoulou, professor of medicine.

The targeted removal of a human trisomy, they noted, could have both clinical and research applications.

Down syndrome ... can cause many medical problems, including heart defects, impaired intellect, premature aging and dementia, and certain forms of leukemia, a type of blood cancer.

"We are certainly not proposing that the method we describe would lead to a treatment for Down syndrome," Russell said. "What we are looking at is the possibility that medical scientists could create cell therapies for some of the blood-forming disorders that (can) accompany Down syndrome."

Read the whole University of Washington News report here 

Journal reference:
Li B. Li, Kai-Hsin Chang, Pei-Rong Wang, Roli K. Hirata, Thalia Papayannopoulou, David W. Russell. Trisomy Correction in Down Syndrome Induced Pluripotent Stem Cells  Cell Stem Cell, 2012; 11 (5): 615 DOI:10.1016/j.stem.2012.08.004

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