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Noninvasive Avenue for Parkinson's Disease Gene Therapy

Apr. 21, 2013 — Researchers at Northeastern University in Boston have developed a gene therapy approach that may one day stop Parkinson's disease (PD) in it tracks, preventing disease progression and reversing its symptoms. The novelty of the approach lies in the nasal route of administration and nanoparticles containing a gene capable of rescuing dying neurons in the brain. Parkinson's is a devastating neurodegenerative disorder caused by the death of dopamine neurons in a key motor area of the brain, the substantia nigra (SN). Loss of these neurons leads to the characteristic tremor and slowed movements of PD, which get increasingly worse with time. Currently, more than 1% of the population over age 60 has PD and approximately 60,000 Americans are newly diagnosed every year. The available drugs on the market for PD mimic or replace the lost dopamine but do not get to the heart of the problem, which is the progressive loss of the dopamine neurons.

The focus of Dr. Barbara Waszczak's lab at Northeastern University in

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Boston is to find a way to harvest the potential of glial cell line-derived

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neurotrophic factor (GDNF) as a treatment for PD. GDNF is a protein

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known to nourish dopamine neurons by activating survival and growth-

0

promoting pathways inside the cells. Not surprisingly, GDNF is able to

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protect dopamine neurons from injury and restore the function of damaged and dying neurons in

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many animal models of PD.

Parkinson 'S

However, the action of GDNF is

Brain Injury

limited by its inability to cross the

Stem Cells

blood-brain barrier (BBB), thus

Cancer Research

requiring direct surgical injection into the brain. To circumvent this

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problem, Waszczak's lab is investigating intranasal delivery as a

Health & Medicine

way to bypass the BBB. Their

Parkinson's

previous work showed that

Research

intranasal delivery of GDNF protects

Nervous System

dopamine neurons from damage by

Gene Therapy

the neurotoxin, 6-hydroxydopamine

Mind & Brain

(6-OHDA), a standard rat model of

Disorders and

PD.

Syndromes

Taking this work a step further,

Brain Injury

Brendan Harmon, working in

Parkinson's

Waszczak's lab, has adapted the

Reference

intranasal approach so that cells in

Sensory neuron

the brain can continuously produce

Dementia with Lewy

GDNF. His work utilized

bodies

nanoparticles, developed by

Dopamine

Copernicus Therapeutics, Inc., which

hypothesis of

are able to transfect brain cells with

schizophrenia

an expression plasmid carrying the

Astrocyte

gene for GDNF (pGDNF). When given intranasally to rats, these pGDNF nanoparticles increase GDNF production throughout the brain for long periods, avoiding the need for frequent re-dosing. Now, in new research presented on April 20 at 12:30 pm during Experimental Biology 2013 in Boston, MA, Harmon reports that intranasal administration of Copernicus' pGDNF nanoparticles results in GDNF expression sufficient to protect SN dopamine neurons in the 6-OHDA model of PD.

Waszczak and Harmon believe that intranasal delivery of Copernicus' nanoparticles may provide an effective and non-invasive means of GDNF gene therapy for PD, and an avenue for transporting other gene therapy vectors to the brain. This work, which was funded in part by the Michael J. Fox Foundation for Parkinson's Research and Northeastern University, has the potential to greatly expand treatment options for PD and many other central nervous system disorders. Share this story on Facebook, Twitter, and Google:

4 Replies

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  • thisd is completely beyond em scientifically

    but a nasal spray was devleloped as a potential use in PSP patients but was dropped as it was not thought ot be productive/.

    so any research which points the way si good is it not?

    ;lol JIll

    :-)

  • sounds like good news;;how long to help us in practical terms?

  • If you google this therapy it's pretty exciting! The catch is...."may one day stop Parkinson's......."

  • This sounds promising, every day prayers for all PD, an related dieases.

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