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.

Cedars-Sinai earns excellence in 2010 adult heart transplants

The Cedars-Sinai Heart Institute and Comprehensive Transplant Center performed the most adult heart transplants of any U.S. medical center in 2010, according to government statistics released today.

10 Mar 2011

Unique gene therapy to combat Parkinson's disease

Physicians at Rush University Medical Center are testing a unique gene therapy product called CERE-120 to evaluate if its use can improve the symptoms of Parkinson's disease. Rush is one of 11 sites in the U.S. and the only site in Illinois enrolling patients into the new, double-blinded trial.

9 Mar 2011

Duchenne Parent Project awards grant to AMT for DMD gene therapy

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today that the Duchenne Parent Project, based in the Netherlands, has awarded AMT a grant of EUR 145,000 to support the development of AMT-080, AMT's gene therapy for Duchenne Muscular Dystrophy.

7 Mar 2011

Novel gene therapy can promote removal of amyloid plaques and prevent Alzheimer's disease

Gene therapy that boosts the ability of brain cells to gobble up toxic proteins prevents development of Alzheimer's disease in mice that are predestined to develop it, report researchers at Georgetown University Medical Center. They say the treatment - which is given just once - could potentially do the same in people at the beginning stages of the disease.

5 Mar 2011

New personalized therapy fools cancer cells into killing themselves

A Wayne State University School of Medicine physician-researcher has developed a personalized therapy to treat a wide range of cancers. The treatment is based on a naturally occurring human enzyme that has been genetically modified to fool cancer cells into killing themselves.

1 Mar 2011

QIAGEN, Epigenomics enter option agreement to commercialize colorectal cancer blood test

Epigenomics AG, the cancer molecular diagnostics company, and QIAGEN today announced that the companies have entered into an option agreement allowing QIAGEN to develop and, should QIAGEN exercise the option, commercialize a colorectal cancer blood test based on Epigenomics' proprietary biomarker mSEPT9 and certain DNA methylation analysis technologies.

From QIAGEN Manchester Limited 28 Feb 2011

Innovative miniscule gene transport system can cause breast cancer cells to self-destruct

Scientists at Queen's University Belfast have shown that they can deliver a gene directly into breast cancer cells causing them to self-destruct, using an innovative, miniscule gene transport system, according to research published today in the International Journal of Pharmaceutics.

28 Feb 2011

fMRI reveals complexity of consciousness

Using a sophisticated imaging test to probe for higher-level cognitive functioning in severely brain-injured patients provides a window into consciousness -- but the view it presents is one that is blurred in fascinating ways, say researchers at Weill Cornell Medical College in the Feb. 25 online edition of the journal Brain.

25 Feb 2011

First evidence of neurotrophic factors improving status of degenerating dopamine neurons in Parkinson's brains

Ceregene, Inc., a biopharmaceutical company, reported today that its scientists and collaborators have published new experimental findings in support of its Parkinson's disease program which appeared as the lead article in the current issue of Movement Disorders. The publication reports the first evidence that gene transfer can provide targeted expression of a neurotrophic factor, i.e., neurturin or NRTN, intended to restore and preserve dying neurons.

17 Feb 2011

The Children's Hospital of Philadelphia establishes Jeffrey Modell Endowed Chair in Pediatric Immunology Research

The Children's Hospital of Philadelphia today announced the establishment of the Jeffrey Modell Endowed Chair in Pediatric Immunology Research.

16 Feb 2011

AMT 2010 total net income increases to EUR 1.4 million

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today reported its results for the year to December 31, 2010.

16 Feb 2011

New clinical trial to test surgery specifically for Type 2 diabetes

A new clinical trial at NewYork-Presbyterian Hospital/Weill Cornell Medical Center is among the first to test surgery specifically for Type 2 diabetes. The aim of the study is to understand whether surgery can control diabetes, as well or even better than the best medical treatment available today. This is the first study of its kind open to patients who are overweight or mildly obese.

14 Feb 2011

New trial tests surgery for Type 2 diabetes

11 Feb 2011

Full genetic blueprint of multiple prostate tumors laid bare

For the first time, researchers have laid bare the full genetic blueprint of multiple prostate tumors, uncovering alterations that have never before been detected and offering a deep view of the genetic missteps that underlie the disease. The study, made possible by key advances in whole genome sequencing and analysis, points to several new prostate cancer genes and a critical category of genomic changes as important drivers of prostate cancer growth.

10 Feb 2011

Indiana University creates Institute for Personalized Medicine

Indiana University has announced a major commitment to research in one of health care's most promising fields with the creation of the Indiana Institute for Personalized Medicine.

10 Feb 2011

Munich research team provides promising alternative to DNA-based procedures

Defects in the genome are the cause of many diseases. Gene therapy - direct replacement of mutant genes by intact DNA copies - offers a means of correcting such defects. Now a research team based at the Medical Center of the University of Munich, and led by Privatdozent Dr. Carsten Rudolph, has taken a new approach that avoids DNA delivery.

8 Feb 2011

Lack of DOT1L enzyme could lead to some types of heart disease: Study

Everyone knows chocolate is critical to a happy Valentine's Day. Now scientists are one step closer to knowing what makes a heart happy the rest of the year.

5 Feb 2011

Digna Biotech, CIMA receive EU grant for development of gene therapy product for Acute Intermittent Porphyria

Digna Biotech and the Centro de Investigacion Medica Aplicada (CIMA) de la Universidad de Navarra announced today that the European Union (EU) has finalized a EUR 3.3 million grant to AIPGENE consortium. Digna Biotech and CIMA will receive, as members of the consortium, EUR 0.5 and EUR 0.8 million respectively for the clinical development of a gene therapy product for Acute Intermittent Porphyria (AIP).

4 Feb 2011

More careful regulation of FDA's Humanitarian Device Exemption in psychiatric patients needed

Warning that patients could be at risk, an interdisciplinary group of multinational investigators is calling on the U.S. Congress and federal regulators to tighten a law that permits use of brain devices to treat rare neuropsychiatric disorders without supporting clinical trials or stringent patient oversight.