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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.
RowanSOM researcher awarded NINDS grant to develop stem cell-based therapy for Canavan disease

RowanSOM researcher awarded NINDS grant to develop stem cell-based therapy for Canavan disease

Paola Leone, PhD, the director of the Cell and Gene Therapy Center and a professor of Cell Biology at the Rowan University School of Osteopathic Medicine, has been awarded a three-year, $477,000 grant from the National Institute of Neurological Disorders and Stroke to develop a stem cell-based therapy for Canavan disease, a rare but devastating neurological disorder in children that typically takes a child's life by age 10. [More]
Scientists discover DNA vaccine that alleviates chronic inflammation in the body

Scientists discover DNA vaccine that alleviates chronic inflammation in the body

An international team of scientists including CureLab Oncology, Inc. (Boston), University of Camerino (Italy), and Boston University have serendipitously discovered a DNA vaccine, which systemically alleviates chronic inflammation in the body. Since osteoporosis is an inflammatory disease, preventive and therapeutic effects of the new vaccine were demonstrated on mouse models with osteoporosis. [More]
New TAU study may offer hope to people diagnosed with Glioblastoma multiforme

New TAU study may offer hope to people diagnosed with Glioblastoma multiforme

There are no effective available treatments for sufferers of Glioblastoma multiforme (GBM), the most aggressive and devastating form of brain tumor. The disease, always fatal, has a survival rate of only 6-18 months. [More]
PCF announces Challenge Awards to support research on new treatment strategies for prostate cancer

PCF announces Challenge Awards to support research on new treatment strategies for prostate cancer

The Prostate Cancer Foundation (PCF) announces 3 new Challenge Awards to support discoveries for the treatment of lethal prostate cancer. [More]
Researchers develop targeted approach that allows muscle to burn more energy

Researchers develop targeted approach that allows muscle to burn more energy

What started as an evolutionary protection against starvation has become a biological "bad joke" for people who need to lose weight. The human body doesn't distinguish between dieting and possible starvation, so when there is a decrease in calories consumed, human metabolism increases its energy efficiency and weight loss is resisted. [More]
UT Dallas study reports new gene therapy approach

UT Dallas study reports new gene therapy approach

Bioengineers at The University of Texas at Dallas have created a novel gene-delivery system that shuttles a gene into a cell, but only for a temporary stay, providing a potential new gene-therapy strategy for treating disease. [More]
Weill Cornell receives NIH grant to study TB-causing bacteria

Weill Cornell receives NIH grant to study TB-causing bacteria

In an effort to stop tuberculosis (TB) from becoming progressively less treatable worldwide, the National Institutes of Health has awarded Weill Cornell Medical College more than $6.2 million in first-year funding to support a research collaboration among six institutions in close alliance with voluntary pharmaceutical partners. [More]
New molecule shows promise in controlling HIV without using daily antiretroviral drugs

New molecule shows promise in controlling HIV without using daily antiretroviral drugs

Scientists have created a new molecule that shows promise for controlling HIV without daily antiretroviral drugs. The molecule foils a wider range of HIV strains in the laboratory than any known broadly neutralizing HIV antibody and is more powerful than some of the most potent of these antibodies. [More]
New study finds promising target for future therapies to treat breast and ovarian cancer patients

New study finds promising target for future therapies to treat breast and ovarian cancer patients

The Food and Drug Administration's recent approval of the drug olaparib for ovarian cancer patients with inherited mutations in the genes BRCA1 or BRCA2 came as welcome news to the thousands of women now eligible to receive it. A new study by Dana-Farber Cancer Institute scientists indicates that the pool of patients who can benefit from the drug is potentially much wider - and offers a ready means of identifying them. [More]

R. Bryan Miller Symposium to spotlight special focus on rare disease research

The University of California, Davis Department of Chemistry will spotlight a special focus on rare disease research with this year's 15th annual R. Bryan Miller Symposium. Experts in rare diseases will gather at the UC Davis Conference Center March 5 for a conference highlighting the opportunities and challenges in applying cutting edge technologies and "precision medicine" to better treat conditions that together affect millions of people, especially children. [More]
Preclinical testing of anti-Ebola antibodies can lead to universal flu vaccine

Preclinical testing of anti-Ebola antibodies can lead to universal flu vaccine

Does the blood of Ebola virus disease survivors contain antibodies and immune cells that could help doctors fight Ebola infections in other people? [More]
Investigators develop microbiome map of New York City subway system

Investigators develop microbiome map of New York City subway system

The microbes that call the New York City subway system home are mostly harmless, but include samples of disease-causing bacteria that are resistant to drugs -- and even DNA fragments associated with anthrax and Bubonic plague -- according to a citywide microbiome map published today by Weill Cornell Medical College investigators. [More]
Two researchers receive Pioneer Award from Human Gene Therapy

Two researchers receive Pioneer Award from Human Gene Therapy

Recognized for their pioneering work in the development of gene transfer technology using retroviral vectors to deliver therapeutic genes into cells, Richard C. Mulligan, PhD, Director of the Harvard Gene Therapy Initiative, Harvard Institutes of Medicine, Boston, MA, and A. Dusty Miller, PhD, Fred Hutchinson Cancer Research Center, Seattle, WA, received the Pioneer Award from Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. [More]
Personalized prevention, treatments of cancer now accessible through Stanford University's new genetics course

Personalized prevention, treatments of cancer now accessible through Stanford University's new genetics course

Instrumental research that aims to enhance how we predict, diagnose and design personalized prevention and treatments of cancer is now accessible through Stanford University's latest professional education course, Understanding Cancer at the Genetic Level. [More]
Johns Hopkins researchers successfully deliver new nanoparticle gene therapy to treat glioma in rats

Johns Hopkins researchers successfully deliver new nanoparticle gene therapy to treat glioma in rats

Despite improvements in the past few decades with surgery, chemotherapy and radiation therapy, a predictably curative treatment for glioma does not yet exist. New insights into specific gene mutations that arise in this often deadly form of brain cancer have pointed to the potential of gene therapy, but it's very difficult to effectively deliver toxic or missing genes to cancer cells in the brain. [More]
Morphogenesis, University of Florida partner to develop novel device to isolate rare cells

Morphogenesis, University of Florida partner to develop novel device to isolate rare cells

Senior engineering students and faculty at the University of Florida in Gainesville are partnering with biotech cell and gene therapy company Morphogenesis, Inc. in Tampa to design an advanced, fully automated cell separation system capable of capturing rare cells – such as stem cells – and circulating tumor cells. [More]
Cell-penetrating nanoparticles can efficiently transport oligonucleotide drugs into cells

Cell-penetrating nanoparticles can efficiently transport oligonucleotide drugs into cells

Therapeutic oligonucleotide analogs represent a new and promising family of drugs that act on nucleic acid targets such as RNA or DNA; however, their effectiveness has been limited due to difficulty crossing the cell membrane. [More]
Using Cultrex BME 2 reduced growth factor to enable long-term culture of human hepatocytes

Using Cultrex BME 2 reduced growth factor to enable long-term culture of human hepatocytes

AMSBIO reports on the recent publication in Cell [1] by Dr Meritxell Huch, Prof Hans Clevers et al. of ground-breaking research using Cultrex BME2 reduced growth factor (organoid growth matrix) to enable long-term (>1 year) culture of genome-stable bipotent stem cells from adult human liver. These results open up new experimental avenues towards the use of human liver material expanded in vitro as an alternative cell source for disease modeling, toxicology studies, drug testing, regenerative medicine and gene therapy. [More]
Using antioxidant to reverse brain inflammation improves obesity, diabetes symptoms

Using antioxidant to reverse brain inflammation improves obesity, diabetes symptoms

Using an antioxidant to reverse inflammation in the brain caused by a high-fat diet greatly improves symptoms related to obesity and type II diabetes, a new study from New Zealand's University of Otago suggests. [More]
UCSD professor wins 2015 Japan Prize

UCSD professor wins 2015 Japan Prize

Theodore Friedmann, MD, professor in the Department of Pediatrics at University of California, San Diego School of Medicine was named today one of three recipients of the 2015 Japan Prize, a prestigious international award honoring laureates whose "original and outstanding achievements in science and technology have advanced the frontiers of knowledge and served the cause of peace and prosperity for mankind. [More]