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.

Severe combined immunodeficiency treated successfully with gene therapy

Gene therapy can offer a cure for a rare condition that causes children’s immune systems to fail. In this rare condition, known as Severe Combined Immunodeficiency (SCID), inherited genetic mutations mean that babies are unable to fight off infection, severely limiting their chances of surviving more than a few years. Severe combined immunodeficiency (SCID) is a rare genetic disorder estimated to affect between 1 in 200,000 and 1 in 1,000,000 live births

26 Aug 2011

AMT total net loss decreases 7% to € 8.7 million for first half year of 2011

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today reported its results for the first half year of 2011.

25 Aug 2011

KU professor to create patient-friendly lung cancer treatment

A Kansas State University professor is trying to create a patient-friendly treatment to help the more than 220,000 people who are diagnosed with lung cancer each year.

23 Aug 2011

NCRM at Case Western Reserve to host stem cell conference in Cleveland

The National Center for Regenerative Medicine (NCRM) at Case Western Reserve University is proud to present "MSC 2011: Innovations in Cell-Based Regenerative Therapies," a three-day forum to highlight transformative breakthroughs in the area of adult stem cell medicine with a focus on how this research is being applied in patient care settings through innovative clinical trials.

18 Aug 2011

World's top genetic scientists to present latest research findings at ICHG/ASHG 2011 Meeting

The world's top scientists and clinicians in the human genetics field will gather to present their latest research findings at the 12th International Congress of Human Genetics (ICHG) / 61st Annual Meeting of the American Society of Human Genetics (ASHG), which will be held on October 11-15, 2011, in Montreal (Quebec), Canada, at the Montreal Convention Center.

17 Aug 2011

LYSOGENE launches the first gene therapy clinical trials in Sanfilippo disease

LYSOGENE announces the authorization by AFSSAPS (the French Agency for the Safety of Health Products) and by the Ethical Review Board of SAF-301, its leading intra-cerebral gene therapy phase I/II clinical trial aiming at treating Sanfilippo Type A Syndrome.

16 Aug 2011

Gene therapy cocktail can treat inherited diseases with 'misfolded' proteins

In a kind of molecular gymnastics, scientists at the University of North Carolina at Chapel Hill School of Medicine have devised a gene therapy cocktail that has the potential to treat some inherited diseases associated with "misfolded" proteins.

16 Aug 2011

New pathway for type 2 diabetes

Newly diagnosed type 2 diabetics tend to have one thing in common: obesity. Exactly how diet and obesity trigger diabetes has long been the subject of intense scientific research.

16 Aug 2011

Fat interferes with body's sugar sensors to trigger type 2 diabetes

Researchers from the United States have identified how a high-fat diet can trigger type 2 diabetes, in experiments on mice and human tissue. They reveal in their study published in the journal Nature Medicine that fat interferes with the body's sugar sensors. The authors add that deeper understanding of the processes involved could help them develop a cure.

15 Aug 2011

NPL scientists mimick the ways viruses infect human cells and deliver genetic material

Scientists at the National Physical Laboratory (NPL) have mimicked the ways viruses infect human cells and deliver their genetic material. The research hopes to apply the approach to gene therapy - a therapeutic strategy to correct defective genes such as those that cause cancer.

12 Aug 2011

New model for amyotrophic lateral sclerosis

By isolating cells from patients' spinal tissue within a few days after death, researchers funded by the National Institutes of Health have developed a new model of the paralyzing disease amyotrophic lateral sclerosis.

12 Aug 2011

Modified spider-silk proteins for nonviral gene delivery

Genetically engineered spider silk could help overcome a major barrier to the use of gene therapy in everyday medicine, according to a new study that reported development and successful initial laboratory tests of such a material. It appears in ACS' journal Bioconjugate Chemistry.

11 Aug 2011

Gene therapy to combat leukemia

In a medical breakthrough scientists have found that gene therapy could be used to cure leukemia. Researchers at the University of Pennsylvania engineered patients' own pathogen-fighting T-cells to target a molecule found on the surface of leukemia cells. These genetically modified T-cells were grown outside of the body and infused back into patients suffering from late-stage chronic lymphocytic leukemia (CLL), which affects the blood and bone marrow and is the most common form of leukemia.

11 Aug 2011

New genetic cause of retinitis pigmentosa

Combining the expertise of several different labs, University of Iowa researchers have found a new genetic cause of the blinding eye disease retinitis pigmentosa (RP) and, in the process, discovered an entirely new version of the message that codes for the affected protein.

10 Aug 2011

Lentiviral vector can kill HIV-infected cells

In what represents an important step toward curing HIV, a USC scientist has created a virus that hunts down HIV-infected cells.

9 Aug 2011

De novo mutations account for 50% of sporadic cases of schizophrenia

Columbia University Medical Center researchers have shown that new, or "de novo," protein-altering mutations—genetic errors that are present in patients but not in their parents—play a role in more than 50 percent of "sporadic" —i.e., not hereditary—cases of schizophrenia.

8 Aug 2011

U.S. physicians spend more time, money for interaction with health plans than Canadian doctors

Physicians in the United States spend nearly four times as much dealing with health insurers and payers compared with doctors in Canada. Most of the difference stems from the fact that Canadian physicians deal with a single payer, in contrast to the multiple payers in the United States.

5 Aug 2011

Study finds HIV epidemics emerging in the Middle East and North Africa among gays

HIV epidemics are emerging in several countries in the Middle East and North Africa among men who have sex with men, a term that encompasses gay, non-gay identified homosexual men, and transgendered and bisexual men.

3 Aug 2011

Lipid nanocarriers in cancer diagnosis and therapy: New book

This book introduces fundamental principles of lipid nanocarriers science relevant to cancer therapy. The first in this field, it fills a need for an accurate, coherent and authoritative introduction to lipid nanocarriers focusing in cancer therapy; both because of the growing popularity of these modern drug delivery systems and also because of the emergent need of dealing with cancer treatment.

2 Aug 2011

New fluorescent tool can track mysterious workings of cellular RNAs

The ability to tag proteins with a green fluorescent light to watch how they behave inside cells so revolutionized the understanding of protein biology that it earned the scientific teams who developed the technique Nobel Prizes in 2008.

30 Jul 2011