DNA nanoparticles hold promise in gene therapy for Parkinson's disease

University of Kentucky researcher David Yurek was recently awarded $66,000 by The Michael J. Fox Foundation for Parkinson's Research under the foundation's Rapid Response Innovation Awards program. The goal of this newly launched initiative is to move quickly to support innovative research focused on the cause of and cure for Parkinson's disease (PD). In particular, MJFF seeks to fund high-risk, high-reward projects tackling critical scientific roadblocks that if successful, can open new avenues for PD therapy development.

Yurek's project, titled "Nanoparticle Gene Therapy for Parkinson's Disease," examines a relatively new gene therapy approach for treating neurodegenerative disorders. He is testing the feasibility of using a novel technology to condense DNA plasmids into nanoparticles and deliver them to the brain as a means to halt or prevent the neurodegenerative process.

The technology comes from Copernicus Therapeutics, Inc., a biotechnology company in Cleveland, Ohio. Yurek, whose laboratory is one of the first to apply this technology to central nervous system disorders, said this relatively new gene therapy strategy holds potential to help repair faulty genes. It entails transduction, a technique for expressing a particular gene in a cell by delivering DNA into the cell and making the cell synthesize the protein that corresponds to that DNA.

"We plan to use this technology to transduce brain cells so that they express proteins beneficial to the cell's survival," Yurek said.

The MJFF award will allow Yurek to test the feasibility of delivering condensed DNA nanoparticles that encode for a neurotrophic factor to the brain as a means to halt or prevent the neurodegenerative process in an animal model of PD. Neurotrophic factors are capable of protecting neurons from dying, thereby rescuing essential neurons in the brain. In animal studies, neurotrophic factors have revived dormant brain cells, caused them to produce dopamine, and prompted dramatic improvement of symptoms.

PD is a chronic, progressive disorder of the central nervous system, and is the direct result of the loss of cells in a section of the brain called the substantia nigra. Those cells produce dopamine, a chemical messenger responsible for transmitting signals within the brain. Loss of dopamine causes critical nerve cells in the brain, or neurons, to fire out of control, leaving patients unable to direct or control their movement in a normal manner.

The Michael J. Fox Foundation for Parkinson's Research is dedicated to ensuring the development of a cure for PD within this decade through an aggressively funded research agenda. Enormous progress toward finding a cure has been made on many neurological fronts, and scientists' understanding of the brain and how disease affects it has increased dramatically. The foundation seeks to hasten progress further by awarding grants that help guarantee that new and innovative research avenues are thoroughly funded and explored.

The MJFF Rapid Response Innovation Awards support projects that may have little to no existing preliminary data, but that hold potential to significantly impact understanding or treatment of PD.

"Given the extremely tight budget of federal government research funding, MJFF's work in prioritizing and funding new and innovative projects is extremely valuable," Yurek said.

Source: University of Kentucky

Related stories:

When a light goes on during thought processes
(PhysOrg.com) -- Thought processes made visible: An international team of scientists headed by Mazahir Hasan of the Max Planck Institute for Medical Research in Heidelberg has succeeded in optically detecting individual action potentials in the brains of living animals. The scientists introduced fluorescent indicator proteins into the brain cells of mice via viral gene vectors: the illumination of the fluorescent proteins indicates both when and which neurons are communicating with each other.
Lessons from yeast: A possible cure for Parkinson's disease?
Parkinson disease (PD) is a debilitating and lethal neurodegenerative disease, for which there is currently no cure. It is caused by the progressive loss of nerve cells that produce the chemical dopamine and is characterized by the accumulation of abnormal aggregates of a protein called alpha-syn in these dopaminergic nerve cells.
Carbon Nanotube-Coated Electrodes Improve Brain Readouts
A research group has significantly improved the quality of brain-function measurements by coating metal neural electrodes with carbon nanotubes. Their work could potentially allow scientists to learn more about brain diseases that are based on electrical impulse malfunctions, such as Parkinson's and epilepsy.
Blood-related genetic mechanisms found important in Parkinson's disease
What does the genetics of blood cells have to do with brain cells related to Parkinson's disease? From an unusual collaboration of neurologists and a pharmacologist comes the surprising answer: Genetic mechanisms at play in blood cells also control a gene and protein that cause Parkinson's disease.
Mind over matter: Monkey feeds itself using its brain
A monkey has successfully fed itself with fluid, well-controlled movements of a human-like robotic arm by using only signals from its brain, researchers from the University of Pittsburgh School of Medicine report in the journal Nature. This significant advance could benefit development of prosthetics for people with spinal cord injuries and those with “locked-in” conditions such as Lou Gehrig’s disease, or amyotrophic lateral sclerosis.
Cell-based therapy shows promise in patients with Parkinson's disease
A novel cell therapy using retinal pigment epithelial (RPE) cells attached to tiny gelatin bead microcarriers implanted in the brain can improve the symptoms of patients with moderate to advanced Parkinson’s disease (PD).
Blood urate levels associated with the progression of Parkinson's disease
Higher blood levels of the compound urate, a salt derived from uric acid that is associated with gout, may be associated with a slower progression of Parkinson’s disease, according to an article posted online today that will appear in the June 2008 print issue of Archives of Neurology, one of the JAMA/Archives journals.
Reprogrammed cells reduce Parkinson's symptoms in rats
Neurons derived from reprogrammed adult skin cells successfully integrated into fetal mouse brains and reduced symptoms in a Parkinson’s disease rat model, according to a study published on April 7 in the online Early Edition of PNAS.

News discussion:

Nanotechnology news