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Research Shows Therapeutic Cloning Can Cure Parkinson's-like Disease in Mice

NEW YORK, Sept. 21 (AScribe Newswire) -- New research from Memorial Sloan-Kettering Cancer Center (MSKCC), Cornell
University, and The University of Connecticut describes a novel way of producing therapeutic nerve cells that can cure
mice with Parkinson's-like disease. The work, which will be published in the October issue of Nature Biotechnology
(available online September 21), provides the first evidence that cloned cells can cure disease in an animal model.

In 2001, Lorenz Studer, MD, Head of the Stem Cell and Tumor Biology Laboratory at MSKCC, and his colleagues at
Rockefeller University published research in which they generated unlimited numbers of genetically matched dopamine
nerve cells using cloned stem cells whose genetic material originated from the mouse's own tail. Dopamine neurons are
nerve cells that are lost in patients who have Parkinson's disease.

(See press release http://www.mskcc.org/mskcc/html/3122.cfm for more information.)

Because the initial method worked for cells derived from some mice but not others, Dr. Studer and his colleagues
developed a better, more efficient way of selectively generating dopamine neurons that eliminates that variability in
order for therapeutic cloning to work consistently for every animal. While they did not yet develop a new cell line for
each of the mice treated, their results prove in principle that the method can work for all cloned cell lines tested.

Using the new technique, the team differentiated stem cells into genetically matched neural cells in vitro. They were
able to selectively develop nerve cells specific to the forebrain, midbrain, hindbrain, and spinal cord, as well as
supporting neural cell types called glial cells. The research demonstrates how closely the generated nerve cells in the
culture dish mimic normal brain cell development, including how long the process takes, the appearance of the cells,
and their function.

"The new technique is a model system that will provide scientists with the opportunity to see how the brain develops in
vitro, and conduct experiments such as observing in a culture dish the developmental consequences of disrupting single
or multiple genes," said Dr. Studer, senior author of the study.

The next step is to develop unique cell lines for a number of Parkinsonian mice and show that these cloned cells can
cure each individual mouse.

The work was supported by the Michael J. Fox Foundation for Parkinson's Research, the National Institute of
Neurological Disorders and Stroke at the National Institutes of Health, and The Parkinson's Disease Foundation.

Memorial Sloan-Kettering Cancer Center is the world's oldest and largest private institution devoted to prevention,
patient care, research, and education in cancer. Our scientists and clinicians generate innovative approaches to better
understand, diagnose and treat cancer. Our specialists are leaders in biomedical research and in translating the latest
research to advance the standard of cancer care worldwide.

SOURCE: AScribe Newswire
http://tinyurl.com/o6y7

Reference:

Nature Biotechnology
http://www.nature.com/nbt/

Advance Online Publication
http://tinyurl.com/o724

ARTICLE:
Neural subtype specification of fertilization and nuclear transfer embryonic stem cells and application in parkinsonian
mice

Tiziano Barberi, Peter Klivenyi, Noel Y Calingasan, Hyojin Lee, Hibiki Kawamata, Kathleen Loonam, Anselme L Perrier,
Juan Bruses, Maria E Rubio, Norbert Topf, Viviane Tabar, Neil L Harrison, M Flint Beal, Malcolm A S Moore & Lorenz
Studer

Published online: 21 September 2003

Abstract:
http://tinyurl.com/o720

Full Text:
http://tinyurl.com/o71z

PDF
http://www.nature.com/cgi-taf/DynaPage.taf?file=/nbt/journal/vaop/ncurrent/full/nbt870.html&filetype=pdf

Supplementary Instructions
http://www.nature.com/nbt/journal/vaop/ncurrent/suppinfo/nbt870_S1.html

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