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.

Encouraging results from exon skipping gene-based therapy study for DMD

Scientists at Royal Holloway, University of London have reported encouraging results in a new gene-based therapy for Duchenne Muscular dystrophy (DMD) which at present has no known cure and affects one in 3,000 young boys.

4 Jan 2011

Neuroscientists say that general anesthesia is a reversible coma

The brain under general anesthesia isn't "asleep" as surgery patients are often told -- it is placed into a state that is a reversible coma, according to three neuroscientists who have published an extensive review of general anesthesia, sleep and coma, in the Dec. 30 issue of The New England Journal of Medicine.

30 Dec 2010

Researchers receive $100,000 NFL grant to study tissue engineering for spinal injuries

Researchers from Cornell University and Weill Cornell Medical College have been awarded $100,000 by NFL Charities, the charitable foundation of the National Football League owners, to research tissue engineering for spinal injuries.

22 Dec 2010

UH scientist evaluates microRNAs as potential treatment strategy for ovarian cancer

The day when an ovarian cancer patient can treat her tumor with a single, painless pill instead of a toxic drug cocktail is the ultimate goal of the pioneering research of a University of Houston scientist.

22 Dec 2010

First comprehensive guide to hospital emergency preparedness exercise development

With the publication of the nation's first comprehensive, federally funded guide to hospital emergency preparedness exercise development, Dr. Nathaniel Hupert at Weill Cornell Medical College and his collaborators have provided a new toolset for strengthening hospitals' ability to protect communities nationwide against public health disasters, such as creating care centers during an influenza pandemic or treating casualties in the wake of a bioterrorism attack.

17 Dec 2010

Novel adenovirus-based gene therapy for renal cell carcinoma

Researchers at Virginia Commonwealth University Massey Cancer Center and the VCU Institute of Molecular Medicine (VIMM) have developed a novel virus-based gene therapy for renal cell carcinoma that has been shown to kill cancer cells not only at the primary tumor site but also in distant tumors not directly infected by the virus. Renal cell carcinoma is the most common form of kidney cancer in adults and currently there is no effective treatment for the disease once it has spread outside of the kidney.

16 Dec 2010

Charité begins implementation of MGN1601 clinical phase I/II study in renal cancer

The Charité - Medical University of Berlin has today begun the implementation of the clinical study phase I/II with the renal cancer medication MGN1601 (ASET Study). With this MOLOGEN AG product, an innovative cell-based gene therapy for the treatment of advanced renal cancer is entering the first important test phase.

15 Dec 2010

Clinical trial investigates pomalidomide in only U.S. study for multiple myeloma

Multiple myeloma, a type of blood cancer in which malignant plasma cells are overproduced in the bone marrow, has led to an estimated 20,000 new diagnoses and 11,000 deaths in 2010. Patients with the disease, who have already failed previous treatments, may be considered for a currently enrolling Phase II study, examining a unique drug combination.

14 Dec 2010

Research programs embody the promise of anti-aging medicine.

Sometimes the value of research lies as much in exposing false remedies as in discovering true breakthroughs. Scientists at the country's first school of gerontology and in laboratories around the University of Southern California do both, mindful of their responsibility to a society focused on healthful aging.

13 Dec 2010

Clinical, preclinical results of ISF35 in CLL patients presented at ASH 2010

Memgen, LLC announced the presentation of clinical and preclinical results for its lead product, ISF35, during the 52nd Annual Meeting of the American Society of Hematology in Orlando, Florida. The research and clinical development of ISF35 announced today was led by Januario Castro, MD, and Thomas Kipps, MD, PhD, both at the University of California, San Diego.

10 Dec 2010

MU researcher hopes to protect cardiac muscles for children with DMD

Children with Duchenne muscular dystrophy face a future of rapidly weakening muscles, which usually leads to death by respiratory or cardiac failure before their 30th birthday. While researchers are hopeful that gene therapy could eventually evolve into an effective treatment, few have targeted the heart of the problem as much as Dongsheng Duan, PhD.

7 Dec 2010

No evidence of toxicity in hemophilia B patients enrolled in gene therapy trial

Investigators report no evidence of toxicity in the four hemophilia B patients enrolled to date in a gene therapy trial using a vector under development at St. Jude Children's Research Hospital and UCL (University College London) to correct the inherited bleeding disorder.

7 Dec 2010

Research findings could provide breakthrough for development of effective therapies for NSCLC

Research at Virginia Commonwealth University Massey Cancer Center led by Charles E. Chalfant, Ph.D., associate professor of biochemistry and molecular Biology, discovered a previously unknown mechanism in non-small cell lung cancer cells that contributes to their ability to maintain and grow tumors. Narrowing in on this mechanism could provide a breakthrough for the development of effective therapies for NSCLC and other cancers.

4 Dec 2010

AMT, Amgen amend GDNF gene license agreement

Amsterdam Molecular Therapeutics Holding N.V., a leader in the development of gene-based therapies, today announced that it has amended and restated its licensing agreement with Amgen for gene therapy applications incorporating the GDNF (glial cell derived neurotrophic factor) gene, to which Amgen holds rights. Financial terms were not disclosed.

2 Dec 2010

Wake Forest University to begin clinical trial for low-grade gliomas

For patients with low-grade gliomas, or slow growing brain tumors, a shot in the arm might soon lead to a new treatment therapy. A groundbreaking, first in humans vaccine will be tested in an early phase clinical trial that will soon begin at Wake Forest University Baptist Medical Center. Eighteen patients will be the first in the world to receive it.

2 Dec 2010

Physician-scientists enroll patients with NSCLC cancer in MAGE-A3 Phase III trial

An experimental immunotherapy may someday become the newest weapon against lung cancer. Physician-scientists from Weill Cornell Medical College and Columbia University Medical Center are enrolling patients with non-small cell lung cancer (NSCLC) at NewYork-Presbyterian Hospital as part of an ongoing Phase III trial.

1 Dec 2010

Ageing reversed in mice: Study

A scientific team at Harvard-affiliated medical centers genetically manipulated mice to age faster. Then they used gene therapy to lengthen telomeres. Telomeres are compounds found at the ends of strands of DNA that are related to age. This lengthening of telomeres reversed age-related problems such as decreased brain function and infertility.

29 Nov 2010

QIAGEN to supply point-of-need molecular veterinary diagnostics for disease detection in emerging countries

QIAGEN has been selected to contribute to the fight against infectious diseases in animals in emerging countries by supplying an international pilot project associated with the United Nations with portable devices to perform ultra-fast molecular testing in the field.

From QIAGEN Manchester Limited 29 Nov 2010

New strategy to prevent memory deficits in mouse model with AD

Scientists at the Gladstone Institute of Neurological Disease (GIND) in San Francisco have discovered a new strategy to prevent memory deficits in a mouse model of Alzheimer's disease (AD).

28 Nov 2010

Hepcidin hormone may be potential therapeutic tool for anemic blood disorders

A hormone made by the body may be a potential therapeutic tool for the treatment of two anemic blood disorders beta-thalassemia and hemochromatosis. The new research was led by scientists at Weill Cornell Medical College and published in the Journal of Clinical Investigation and the journal Blood.

24 Nov 2010