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Therapeutic Cloning Treats Parkinson's Disease In Mice

ScienceDaily (Mar. 24, 2008) - Research led by investigators at Memorial 
Sloan-Kettering Cancer Center (MSKCC) has shown that therapeutic cloning, 
also known as somatic-cell nuclear transfer (SCNT), can be used to treat 
Parkinson's disease in mice.

For the first time, researchers showed that therapeutic cloning or SCNT has 
been successfully used to treat disease in the same subjects from whom the 
initial cells were derived. While this current work is in animals, it could 
have future implications as this method may be an effective way to reduce 
transplant rejection and enhance recovery in other diseases and in other 
organ systems.

In therapeutic cloning or SCNT, the nucleus of a somatic cell from a donor 
subject is inserted into an egg from which the nucleus has been removed. 
This cell then develops into a blastocyst from which embryonic stem cells 
can be harvested and differentiated for therapeutic purposes. As the genetic 
information in the resulting stem cells comes from the donor subject, 
therapeutic cloning or SCNT would yield subject-specific cells that are 
spared by the immune system after transplantation.

The new study shows that therapeutic cloning can treat Parkinson's disease 
in a mouse model. The scientists used skin cells from the tail of the animal 
to generate customized or autologous dopamine neurons--the missing neurons 
in Parkinson's disease. The mice that received neurons derived from 
individually matched stem cell lines exhibited neurological improvement. But 
when these neurons were grafted into mice that did not genetically match the 
transplanted cells, the cells did not survive well and the mice did not 
recover.

The study's results are published in the March 23 online edition of the 
journal Nature Medicine.

The work was led by senior author Lorenz Studer, MD, Head of the Stem Cell 
and Tumor Biology Laboratory within the Sloan-Kettering Institute at MSKCC, 
and lead author Viviane Tabar, MD, Neurosurgeon and stem cell scientist at 
MSKCC. The work was performed in collaboration with scientists at the Riken 
Institute in Kobe, Japan.

Other MSKCC researchers who contributed to this study are: Mark Tomishima, 
Georgia Panagiotakos, George Al-Shamy, Bill Chan, and Jayanthi Menon. 
Scientists in Japan include group leader Teruhiko Wakayama and scientists 
Eiji Mizutani, Sayaka Wakayama and Hiroshi Ohta. This research was supported 
by the US National Institute of Neurological Disorders and Stroke, the Starr 
Tri-institutional Stem Cell Initiative, the Michael J. Fox Foundation for 
Parkinson's Research, the Michael W. McCarthy Foundation and an unrestricted 
grant from the Kinetics Foundation.

Adapted from materials provided by Memorial Sloan-Kettering Cancer Center.

Rayilyn Brown
Director AZNPF
Arizona Chapter National Parkinson Foundation
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