Gene Therapy News and Research

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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.

Fourth patient dosed in Benitec Biopharma's Phase I/IIa hepatitis C trial

Benitec Biopharma, a biopharmaceutical company focused on providing potentially curative therapies with its proprietary gene-silencing technology called ddRNAi or "expressed RNAi," today announced that the fourth patient in the company's Phase I/IIa dose escalation clinical trial of its lead program TT-034 for treating hepatitis C was dosed at the Duke Clinical Research Unit.

11 Mar 2015

New drug shows promise in driving insulin-producing beta cells to multiply

In a screen of more than 100,000 potential drugs, only one, harmine, drove human insulin-producing beta cells to multiply, according to a study led by researchers at the Icahn School of Medicine at Mount Sinai, funded by JDRF and the National Institutes of Health, and published online today in Nature Medicine.

10 Mar 2015

Viagra can have anti-cancer, anti-Alzheimer's disease effects if used with new drugs

Chaperone proteins play an important role in protein folding in human cells and in bacteria and are promising new targets for drugs to treat cancer and Alzheimer's disease and for novel antiviral drugs and antibiotics. How existing drugs such as Viagra or Cialis and a derivative of the drug Celebrex, for example, can reduce the activity of a specific chaperone protein, with the potential for anti-tumor and anti-Alzheimer's disease effects, is described in a Review article in DNA and Cell Biology, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers.

10 Mar 2015

Non-viral gene therapy provides relief for diabetics with constant foot pain

Walking barefoot on sand "felt like walking on glass" for Keith Wenckowski, who has lived with type-one diabetes for more than two decades.

6 Mar 2015

MIT researchers develop new nanodevice that can help overcome drug resistance

Chemotherapy often shrinks tumors at first, but as cancer cells become resistant to drug treatment, tumors can grow back. A new nanodevice developed by MIT researchers can help overcome that by first blocking the gene that confers drug resistance, then launching a new chemotherapy attack against the disarmed tumors.

3 Mar 2015

New strategy may ensure safety of adult epidermal stem cells before performing treatments

A team of European researchers has devised a strategy to ensure that adult epidermal stem cells are safe before they are used as treatments for patients. The approach involves a clonal strategy where stem cells are collected and cultivated, genetically modified and single cells isolated before being rigorously tested to make sure they meet the highest possible safety criteria.

28 Feb 2015

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.

26 Feb 2015

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.

26 Feb 2015

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.

25 Feb 2015

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.

25 Feb 2015

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.

23 Feb 2015

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.

19 Feb 2015

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.

19 Feb 2015

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.

19 Feb 2015

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.

16 Feb 2015

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.

16 Feb 2015

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?

9 Feb 2015

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.

9 Feb 2015

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.

9 Feb 2015

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.

9 Feb 2015

Gene Therapy News and Research

Further Reading

Fourth patient dosed in Benitec Biopharma's Phase I/IIa hepatitis C trial

New drug shows promise in driving insulin-producing beta cells to multiply

Viagra can have anti-cancer, anti-Alzheimer's disease effects if used with new drugs

Non-viral gene therapy provides relief for diabetics with constant foot pain

MIT researchers develop new nanodevice that can help overcome drug resistance

New strategy may ensure safety of adult epidermal stem cells before performing treatments

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

Scientists discover DNA vaccine that alleviates chronic inflammation in the body

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

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

Researchers develop targeted approach that allows muscle to burn more energy

UT Dallas study reports new gene therapy approach

Weill Cornell receives NIH grant to study TB-causing bacteria

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

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

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

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

Investigators develop microbiome map of New York City subway system

Two researchers receive Pioneer Award from Human Gene Therapy

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

Leveraging the power of automation to boost research

Automating research - solutions and best practices

How can microdialysis benefit drug development

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