(Press-News.org) New Rochelle, NY, July 25, 2012—A novel approach to gene therapy that instructs a person's own cells to produce more of a natural disease-fighting protein could offer a solution to treating many genetic disorders. The method was used to achieve a 2- to 3-fold increase in production of a protein deficient in patients with Friedreich's ataxia, as described in an article published Instant Online in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc. (http://www.liebertpub.com) The article is available free online at the Human Gene Therapy website (http://www.liebertpub.com/hum).
The innovative gene therapy method described by Jacques Tremblay, Pierre Chapdelaine, Zoé Coulombe, and Joel Rousseau, Laval University, Quebec, and University of Quebec, Canada, takes advantage of the ability of a family of proteins called Tal effector (TALE) proteins to target specific DNA sequences. As a model of how this method could be used to treat genetic disease, the authors engineered TALE proteins to target the gene that codes for the frataxin protein, which is deficient in Friedreich's ataxia. The ability to induce cells to produce more frataxin could reduce symptoms of the disease and provide an effective, long-term therapeutic strategy, conclude the authors in the article "TALE Proteins Induce the Expression of the Frataxin Gene. (http://online.liebertpub.com/doi/full/10.1089/hum.2012.034)
"This is a very clever approach to treat a recessive disease caused by decreased quantity of an otherwise normal protein," says James M. Wilson, MD, PhD, Editor-in-Chief, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia.
INFORMATION:
About the Journal
Human Gene Therapy (http://www.liebertpub.com/hum), the Official Journal of the European Society of Gene and Cell Therapy, British Society for Gene and Cell Therapy, French Society of Cell and Gene Therapy, German Society of Gene Therapy, and five other gene therapy societies is an authoritative peer-reviewed journal published monthly in print and online that presents reports on the transfer and expression of genes in mammals, including humans. Related topics include improvements in vector development, delivery systems, and animal models, particularly in the areas of cancer, heart disease, viral disease, genetic disease, and neurological disease, as well as ethical, legal, and regulatory issues related to the gene transfer in humans. Tables of content and a free sample issue may be viewed online at the Human Gene Therapy website (http://www.liebertpub.com/hum).
About the Publisher
Mary Ann Liebert, Inc., publishers (http://www.liebertpub.com) is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including Tissue Engineering, Stem Cells and Development, and Cellular Reprogramming. Its biotechnology trade magazine, Genetic Engineering & Biotechnology News (GEN), was the first in its field and is today the industry's most widely read publication worldwide. A complete list of the firm's 70 journals, books, and newsmagazines is available at Mary Ann Liebert, Inc. (http://www.liebertpub.com)
Mary Ann Liebert, Inc.
140 Huguenot St., New Rochelle, NY 10801-5215
http://www.liebertpub.com
Phone: (914) 740-2100
(800) M-LIEBERT
Fax: (914) 740-2101
New gene therapy strategy boosts levels of deficient protein in Friedreich's ataxia
2012-07-26
[Attachments] See images for this press release:
ELSE PRESS RELEASES FROM THIS DATE:
ONR-funded research takes flight in Popular Science article
2012-07-26
ARLINGTON, Va.—The Office of Naval Research (ONR) is looking at birds' perceptual and maneuvering abilities as inspiration for small unmanned aerial vehicle (UAV) autonomy, and Popular Science is featuring this effort in its August issue, posted online July 25.
An ONR-funded, five-year Multidisciplinary University Research Initiative (MURI) program is examining the control and behavioral processes of birds and other small animals when flying at high speeds through complex environments, such as forests or urban settings. Researchers are trying to understand why birds make ...
Study links alcohol/energy drink mixes with casual, risky sex
2012-07-26
BUFFALO, N.Y. -- A new study from the University at Buffalo's Research Institute on Addictions (RIA) has found a link between the consumption of caffeinated energy drinks mixed with alcohol and casual -- often risky -- sex among college-age adults.
According to the study's findings, college students who consumed alcohol mixed with energy drinks (AmEDs) were more likely to report having a casual partner and/or being intoxicated during their most recent sexual encounter.
The results seem to indicate that AmEDs may play a role in the "hook-up culture" that exists on ...
Mind vs. body? Dualist beliefs linked with less concern for healthy behaviors
2012-07-26
Many people, whether they know it or not, are philosophical dualists. That is, they believe that the brain and the mind are two separate entities. Despite the fact dualist beliefs are found in virtually all human cultures, surprisingly little is known about the impact of these beliefs on how we think and behave in everyday life.
But a new research article forthcoming in Psychological Science, a journal of the Association for Psychological Science, suggests that espousing a dualist philosophy can have important real-life consequences.
Across five related studies, researchers ...
Public strongly supports programs helping farmers adapt to climate change
2012-07-26
EAST LANSING, Mich. — A survey conducted by Michigan State University reveals strong public support for government programs to assist farmers to adapt to climate change.
According to NASA research, global temperatures have been rising for decades, and it's affecting all aspects of agriculture. Regardless of what those surveyed believe causes climate change, more than 65 percent of them support government assistance for farmers, said Scott Loveridge, MSU professor of agricultural, food and resource economics.
This year has been a particularly harsh example. This summer's ...
NASA and university researchers find a clue to how life turned left
2012-07-26
GREENBELT, Md. -- Researchers analyzing meteorite fragments that fell on a frozen lake in Canada have developed an explanation for the origin of life's handedness – why living things only use molecules with specific orientations. The work also gave the strongest evidence to date that liquid water inside an asteroid leads to a strong preference of left-handed over right-handed forms of some common protein amino acids in meteorites. The result makes the search for extraterrestrial life more challenging.
"Our analysis of the amino acids in meteorite fragments from Tagish ...
Women with diabetes more likely to experience sexual dissatisfaction
2012-07-26
Women with diabetes are just as likely to be interested in, and engage in, sexual activity as non-diabetic women, but they are much more likely to report low overall sexual satisfaction, according to a UCSF study.
The researchers also found that diabetic women receiving insulin treatment were at higher risk for the specific complications of lubrication and orgasm.
"Diabetes is a recognized risk factor for erectile dysfunction in men, but there have been almost no data to indicate whether it also affects sexual function in women," said senior author Alison J. Huang, ...
How the fluid between cells affects tumors
2012-07-26
July 25, 2012
There are many factors that affect tumor invasion, the process where a tumor grows beyond the tissue where it first developed. While factors like genetics, tissue type and environmental exposure affect tumor metastasis and invasion, physical forces like fluid flow remain a poorly understood component of tumor invasion. A new video article in JoVE, the Journal of Visualized Experiments, describes a novel procedure that allows researchers to study and test the microenvironment of a growing tumor. The technique is valuable because it allows scientists to assay ...
Piglets in mazes provide insights into human cognitive development
2012-07-26
URBANA -- Events that take place early in life almost certainly have consequences for later cognitive development. Establishing the connections is difficult, however, because human infants cannot be used as laboratory subjects.
Rodney Johnson and his collaborators have developed an alternative model for studying infant brain development. "Assistant professor Ryan Dilger and I became interested in establishing the neonatal piglet as a model of human brain and cognitive development 3 or 4 years ago," he said.
The idea came to Johnson when a former student, who was working ...
Texas A&M biologists prove ZOLOFT packs potential to fight fungal meningitis
2012-07-26
New research conducted by biologists at Texas A&M University suggests that ZOLOFT®, one of the most widely prescribed antidepressants in the world, also packs a potential preventative bonus — potent mechanisms capable of inhibiting deadly fungal infections.
The findings are the result of a two-year investigation by Xiaorong Lin, assistant professor of biology, and Matthew S. Sachs, professor of biology, involving sertraline hydrocholoride (ZOLOFT) and its effects on Cryptococcus neoformans, the major causative agent of fungal meningitis — specifically, cryptococcal meningitis, ...
Increasing dopamine in brain's frontal cortex decreases impulsive tendency, UCSF-Gallo study finds
2012-07-26
Raising levels of the neurotransmitter dopamine in the frontal cortex of the brain significantly decreased impulsivity in healthy adults, in a study conducted by researchers at the Ernest Gallo Clinic and Research Center at the University of California, San Francisco.
"Impulsivity is a risk factor for addiction to many substances, and it has been suggested that people with lower dopamine levels in the frontal cortex tend to be more impulsive," said lead author Andrew Kayser, PhD, an investigator at Gallo and an assistant professor of neurology at UCSF. "We wanted to ...