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In both cases, the human cells worked even better than the mouse stem cells,
the scientists reported, repairing tissue and extending life.
The results show "the first successful use of human embryonic stem cells in
treating a human degenerative disease, significantly preserving and
extending life," Snyder said.
Wise Young, a spinal cord researcher and stem cell expert at Rutgers
University, said he was fascinated by the researchers' achievement of
comparing three types of stem cells in the study.
"Apparently, all three types of cells worked," said Young, reached in China.
"Also, it appears that the (mice) are not rejecting human cells even though
the animals were not immune-suppressed. This is surprising."
The researchers also were able to double the lives of the mice when they
gave them, in addition to the stem cell injections, an oral drug that
boosted the efficiency of the cells' ability to process fat.
"In the future, the most successful therapies -- including those employing
stem cells -- will likely involve the use of multiple strategies in
concert," Snyder said. "Indeed, the stem cell may be the 'glue' that
ultimately holds these therapies together in an effective manner by virtue
of its fundamental biology."
Sandhoff disease, which does not afflict any specific ethnic group, results
from a genetic mutation that reduces the body's supply of an enzyme called
"hex" used by brain cells to process excess fatty material. It usually
strikes infants by the time they are six months old and kills them before
they are 6 years old. Deposits of unprocessed fat produced by the disease
destroy the brain cells instrumental in controlling and coordinating body
movement.
Tay-Sachs also is brought about by deficiencies in the body's supply of the
hex protein. Tay-Sachs and Sandhoff are part of a much more common group of
neurodegenerative diseases such as Alzheimer's, Parkinson's, autism and
cerebral palsy. The work is expected to give insights into these diseases,
as well.
On the Web at http://www.nature.com/nm/journal/vaop/ncurrent/index.html.
Kitta MacPherson may be reached at (973) 392-5836 or at
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