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.

New therapeutic approach shows potential for treating cancer

Case Western Reserve researchers have identified a two-pronged therapeutic approach that shows great potential for weakening and then defeating cancer cells.

31 Dec 2014

Researchers treat myocardial infarction with new telomerase-based gene therapy

The enzyme telomerase repairs cell damage produced by ageing, and has been used successfully in therapies to lengthen the life of mice. Now it has been observed that it could also be used to cure illnesses related to the ageing process.

18 Dec 2014

High-dose flu vaccine better than regular flu shot for frail, older adults of long-term care facilities

The high-dose flu vaccine is significantly better than the regular flu shot at boosting the immune response to the flu virus in frail, older residents of long-term care facilities, according to the results of a University of Pittsburgh School of Medicine study.

18 Dec 2014

Scientists demonstrate technique for editing genome in sperm-producing adult stem cells

Scientists at Indiana University and colleagues at Stanford and the University of Texas have demonstrated a technique for "editing" the genome in sperm-producing adult stem cells, a result with powerful potential for basic research and for gene therapy.

16 Dec 2014

Study finds that K13 gene mutations cause malaria drug resistance in Southeast Asia

Growing resistance to malaria drugs in Southeast Asia is caused by a single mutated gene inside the disease-causing Plasmodium falciparum parasite, according to a study led by David Fidock, PhD, professor of microbiology & immunology and of medical sciences (in medicine) at Columbia University Medical Center.

12 Dec 2014

Spark Therapeutics, Pfizer partner to develop SPK-FIX for potential treatment of hemophilia B

Spark Therapeutics, a late-stage gene therapy company developing treatments for debilitating genetic diseases, announced today that it has entered into a global collaboration with Pfizer Inc. for the development and potential commercialization of SPK-FIX, a development program advancing proprietary, bio-engineered adeno-associated virus (AAV) vectors for the potential treatment of hemophilia B.

8 Dec 2014

Henry Ford Hospital recruits patients to participate in brain cancer clinical trial

Henry Ford Hospital today announced that it is actively recruiting patients to participate in a clinical trial taking place at the Hermelin Brain Tumor Center which is showing promising results in patients with brain cancer.

8 Dec 2014

New study explores reliability of optogenetics as method of intervention of temporal lobe seizures

Optogenetics is one of the hottest tools in biomedical research today, a method that uses gene therapy to deliver light-sensitive proteins into specific cells. This new tool allows researchers to interact with a single cell or a network of cells with exquisite precision. Whereas imaging and other technologies allow researchers to watch the brain in action, optogenetics enables them to influence those actions.

7 Dec 2014

Genetics may play major role in Lou Gehrig's disease, study reveals

Genetics may play a larger role in causing Lou Gehrig's disease than previously believed, potentially accounting for more than one-third of all cases, according to one of the most comprehensive genetic studies to date of patients who suffer from the condition also known as amyotrophic lateral sclerosis, or ALS.

6 Dec 2014

Researchers find way to prevent noise-induced hearing loss

Researchers from Weill Cornell Medical College and the Gladstone Institutes have found a way to prevent noise-induced hearing loss in a mouse using a simple chemical compound that is a precursor to vitamin B3. This discovery has important implications not only for preventing hearing loss, but also potentially for treating some aging-related conditions that are linked to the same protein.

3 Dec 2014

Novartis to highlight advances in blood, breast cancer research at ASH and SABCS 2014

Novartis will highlight more than 250 abstracts demonstrating advances in blood and breast cancer research at the upcoming American Society of Hematology (ASH) annual meeting December 6-9, and CTRC-AACR San Antonio Breast Cancer Symposium (SABCS) December 9-13.

2 Dec 2014

President of Institute of Medicine to deliver presentation at University of Louisville

The president of the Institute of Medicine of the National Academies will present the 2014 Leonard Leight Lecture at the University of Louisville.

2 Dec 2014

UC San Diego Health System opens nation's first angioedema treatment center

UC San Diego Health System in partnership with the U.S. Hereditary Angioedema Association, a non-profit patient advocacy organization, has opened the nation's first dedicated center for diagnosing and treating diverse forms of swelling, known collectively as angioedema.

24 Nov 2014

Authors review current progress in developing transgenic pig models for human diseases

Genetically engineered pigs, minipigs, and microminipigs are valuable tools for biomedical research, as their lifespan, anatomy, physiology, genetic make-up, and disease mechanisms are more similar to humans than the rodent models typically used in drug discovery research.

24 Nov 2014

Researchers identify way to improve memory by manipulating molecule linked to Alzheimer's

In a new study conducted by the Sagol Department of Neurobiology at the University of Haifa and published recently in the Journal of Neuroscience, researchers report that they've found a way to improve memory by manipulating a specific molecule that is known to function poorly in old age and is closely linked to Alzheimer's disease.

23 Nov 2014

TSRI study shows how mutations in Tmie gene can cause deafness from birth

Scientists at The Scripps Research Institute have discovered how one gene is essential to hearing, uncovering a cause of deafness and suggesting new avenues for therapies.

21 Nov 2014

Researchers discover new method to deliver drugs into aggressive tumors

A multi-disciplinary team of Yale Cancer Center researchers has discovered a promising new method for delivering drugs into aggressive tumors by exploiting a unique feature of tumors themselves.

21 Nov 2014

Study shows how stem cells can help regenerate damaged muscle after heart attack

Delivering stem cell factor directly into damaged heart muscle after a heart attack may help repair and regenerate injured tissue, according to a study led by researchers from Icahn School of Medicine at Mount Sinai presented November 18 at the American Heart Association Scientific Sessions 2014 in Chicago, IL.

19 Nov 2014

Gene therapy transforms life for men with severe form of hemophilia B

Gene therapy developed at St. Jude Children's Research Hospital, University College London and the Royal Free Hospital has transformed life for men with a severe form of hemophilia B by providing a safe, reliable source of the blood clotting protein Factor IX that has allowed some to adopt a more active lifestyle, researchers reported.