(Press-News.org) BOSTON — 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 Boston is to find a way to harvest the potential of glial cell line-derived neurotrophic factor (GDNF) as a treatment for PD. GDNF is a protein known to nourish dopamine neurons by activating survival and growth-promoting pathways inside the cells. Not surprisingly, GDNF is able to protect dopamine neurons from injury and restore the function of damaged and dying neurons in many animal models of PD. However, the action of GDNF is limited by its inability to cross the blood-brain barrier (BBB), thus requiring direct surgical injection into the brain. To circumvent this problem, Waszczak's lab is investigating intranasal delivery as a way to bypass the BBB. Their previous work showed that intranasal delivery of GDNF protects dopamine neurons from damage by the neurotoxin, 6-hydroxydopamine (6-OHDA), a standard rat model of PD.
Taking this work a step further, Brendan Harmon, working in Waszczak's lab, has adapted the intranasal approach so that cells in the brain can continuously produce GDNF. His work utilized nanoparticles, developed by Copernicus Therapeutics, Inc., which are able to transfect brain cells with an expression plasmid carrying the 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.
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To request an interview with Dr. Waszczak, please contact Jim Bernstein at the contact information listed above.
About Experimental Biology 2013
Experimental Biology's mission is to share the newest scientific concepts and research findings shaping future and current clinical advances – and to give scientists and clinicians an unparalleled opportunity to hear from colleagues working on similar biomedical problems using different disciplines. With six sponsoring societies and another 20 U.S. and international guest societies, the annual meeting brings together scientists from throughout the United States and the world, representing dozens of scientific areas, from laboratory to translational to clinical research. The meeting also offers a wide spectrum of professional development sessions.
About the American Society for Pharmacology and Experimental Therapeutics
ASPET is a 5,100 member scientific society whose members conduct basic and clinical pharmacological research within the academic, industrial and government sectors. Our members discover and develop new medicines and therapeutic agents that fight existing and emerging diseases, as well as increase our knowledge regarding how therapeutics affects humans.
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BOSTON — Heart disease and approximately half of all strokes are the results of advanced atherosclerosis with damaged endothelium, the inner lining of blood vessels. In 2009, the direct and indirect annual cost of heart disease and stroke was approximately $312.6 billion. Projections are for the total cost of heart disease to increase from $523 to $1.126 billion from 2013 to 2030. And by 2030, it is expected that there will be more than 148 million of the US population would have heart disease. Development of new technologies for assessing and treating endothelium damage ...
BOSTON — Stress triggered neuropsychiatric disorders take an enormous personal, social and economic toll on society. In the US more than half of adults are exposed to at least one traumatic event throughout their lives. Post-traumatic stress disorder (PTSD) is a debilitating anxiety disorder associated with exposure to a traumatic event outside the range of normal human experience. PTSD typically follows a chronic, often lifelong, course. Patients have diminished quality of life, are more likely to manifest other psychiatric disorders such as depression and six times more ...
BOSTON — More than 1 in 270 people in the US are living with HIV and every 9.5 minutes someone is else is infected. The economic cost estimates associated with HIV/AIDS exceed 36 billion dollars a year. The development of effective drug treatments have allowed people with HIV to live longer with federal health officials now predicting that by 2015 one-half of the population with HIV in the US will be older than 50.
Efavirenz (tradenames: Sustiva®, Stocrin®) is an antiretroviral (ARV) drug commonly used to treat HIV. Its popularity as a medication, alone or more commonly ...
BOSTON — Depression is the leading cause of disability with more than 350 million people globally affected by this disease. In addition to debilitating consequences on mental health, depression predisposes an individual to physiological disease such as heart disease, and conversely heart disease increases the risk of depression. According to the World Health Organization by the year 2020 heart disease and depression will be the number one and number two leading causes of disability in developed countries. While the co-occurrence of these disorders is well recognized, an ...
Geneva, Switzerland: Unavoidable damage caused to the heart and lungs by radiotherapy treatment of tumours in the chest region can be limited by the administration of an ACE inhibitor, a drug commonly used in the treatment of cardiovascular disease, a group of Dutch researchers have found. [1]
Common cancers such as breast, oesophagus, lung, and Hodgkin's lymphoma are frequently treated with radiotherapy, but the radiation dose that can be given safely is limited by the sensitivity of the health lung tissue which is also irradiated.
The lung is a particularly complex ...
Middle school students from rural Maine will present a poster at the Experimental Biology 2013 conference detailing how their involvement with the Aspirnaut science-outreach program led to them becoming active scientific researchers.
Students from the Dedham School in Dedham, Maine, were participants in the Aspirnaut program (http://www.aspirnaut.org), a science-outreach program started at Vanderbilt University, in which scientists engage with middle- and high-school students through videoconferencing. Showing a great interest in the research that was being demoed by ...
Geneva, Switzerland: Intensity modulated radiotherapy (IMRT) gives better results than standard radiotherapy in patients with early breast cancer, according to results from a randomised trial presented today (Sunday) to the 2nd Forum of the European Society for Radiotherapy and Oncology (ESTRO). IMRT is an advanced, high-precision form of radiotherapy that can deliver an even dose of radiation, thus reducing the cosmetic problems that can often occur after breast radiotherapy.
"We believe that this study, the largest prospective trial in the world to test breast IMRT ...
Lisa B. Overton is opening up and telling the world about her life as the child of a forbidden love affair. This moving story details her experience growing up as the child of a Roman Catholic priest and an ex-nun who entered into a 30+ year clandestine love affair.
She tells the poignant story of her childhood and how she survived loneliness, shame and hypocrisy in her parents' painful and secretive shadow and how she eventually forgave them both. She never knew who her father was until finally, on her mother's death bed Lisa asked, "Was Uncle my father?" ...
Brene Brown will be the featured speaker at the University of Houston Graduate College of Social Work (GCSW) inaugural scholarship luncheon, "Daring to Soar," at 11:30 a.m., Wednesday, May 8 at the Houston Country Club.
For more than a decade, Brown has been researching and writing about shame, vulnerability, courage and compassion in her role as a research professor at the UH GCSW. Her latest milestone was a two-episode appearance with celebrated talk show host Oprah Winfrey to discuss Brown's new book, "Daring Greatly," which made its way onto ...
On Monday, April 29th 2013 the 14th annual Newport Beach Film Festival will present its Australian Spotlight, an evening celebration of Australian cinema and culture. The event will feature the West Coast Premiere of the highly anticipated film, Drift, followed by a festive post screening gala.
Drift is an action-packed ride that reflects the dynamic surf culture and spirit that is shared by Austrialia and southern Califoria. The film, based on a true story, follows two brothers (Absolut Vodka and Stella Artois Beer, 10 top Orange County restaurants serving signature ...
