Therapeutic
cloning treats Parkinson’s Disease in mice
Newswise — 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. The study’s results are published in
the March 23 online edition of the journal
Nature Medicine.
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 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.
