Print

Print


The source of this article is Health India: http://tinyurl.com/4nmr2

Scientists identify how gene therapy could help cure Parkinson's disease:

[Health India]: Washington, Dec 5 : A new study, published this week in the
"Proceedings of the National Academy of Sciences," has found that viral
delivery of a gene associated with Parkinson's disease protected neurons
from degeneration.

Researchers from Switzerland, studied a more common, recessive form of
inherited Parkinson's disease that typically strikes before the age of 40
and is linked to a mutation in the parkin gene. The loss of enzyme activity
resulting from this mutation leads to an accumulation of protein substrates,
including alpha- synuclein, in the cell. In these juvenile Parkinson's cases
Lewy bodies are not generally present.

Both these forms of the disease involve buildup of alpha- synuclein in the
cell, and result in cell degeneration and death. All the evidence pointed to
the possibility that parkin might play an important role in protecting
neurons from toxic overaccumulations of alpha-synuclein.

The researchers tested this by injecting rats sub-cranially with lentiviral
vectors containing the genes for parkin as well as a mutated form of the
alpha-synuclein gene. Harmless forms of viruses such as the lentiviral
vector are used by researchers because they can efficiently penetrate cells
and deliver the gene of interest without inducing a major immune response.

The viruses successfully delivered the genetic material to about 30 percent
of the rat neurons, causing overexpression of parkin and alpha-synuclein.
After six weeks, the researchers found that almost all these affected
dopamine neurons in rats that had been injected with alpha-synuclein alone
had died. In contrast, numerous affected dopamine neurons in animals
injected with both parkin and alpha-synuclein were still going strong. The
presence of parkin had protected the neurons from alpha-synuclein toxicity.

In addition, rats that had been injected with parkin showed an increase in
Lewy body-type intracellular deposits. The researchers postulated that
parkin might work to enhance cell survival by somehow transforming soluble
toxic proteins into insoluble aggregates.

The researchers say that the findings constitute a significant step forward
in understanding the molecular mechanisms behind the disease. "We
demonstrated for the first time the feasibility of the gene therapy approach
in a genetic model of Parkinson's disease," they wrote in their study.

According to them, results of this research might lead to new treatments
that could prevent the onset of Parkinson's disease in genetically
predisposed subjects and arrest the disease's progression once it has been
diagnosed.

"These observations could translate into the clinic either through a direct
gene therapy approach or through the identification of small drugs which
enhance the expression of the endogenous parkin," the researchers added. (ANI)

----------------------------------------------------------------------
To sign-off Parkinsn send a message to: mailto:[log in to unmask]
In the body of the message put: signoff parkinsn