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.

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.

19 Nov 2014

TapImmune signs new collaborative research agreement with VGTI Florida

TapImmune, Inc., is pleased to announce a new collaborative research agreement with The Vaccine & Gene Therapy Institute of Florida, a leading, non-profit biomedical research institute, forming a partnership to advance TapImmune's proprietary, cancer vaccines into Phase II human clinical trials for the treatment of breast and ovarian cancers.

18 Nov 2014

Leading causes of blindness can be detected by looking at how eyes respond to watching TV

One of the leading causes of blindness worldwide could be detected by how our eyes respond to watching TV according to a new study from researchers at City University London.

11 Nov 2014

Scientists may have discovered new way to repair damaged tissue

By transforming human scar cells into blood vessel cells, scientists at Houston Methodist may have discovered a new way to repair damaged tissue. The method, described in an upcoming issue of Circulation (early online), appeared to improve blood flow, oxygenation, and nutrition to areas in need.

9 Nov 2014

Regenerative medicine may offer new standard of advanced treatment for foot and leg ulcers

These are exciting times for regenerative medicine. Unlike conventional medicines, the regenerative approach can potentially work to restore the lost functionality of tissues or organs—the major reason for intensive focus on research and development in the field.

7 Nov 2014

New consensus guidelines for toxicity testing of oligonucleotide-based therapeutics

Oligonucleotide-based therapeutics present unique challenges when it comes to testing their potential to cause reproductive and developmental harm. New consensus guidelines for toxicity testing that take into consideration the combined chemical and biological characteristics of these novel biopharmaceuticals are presented in Nucleic Acid Therapeutics, a peer-reviewed journal from Mary Ann Liebert, Inc. publishers.

31 Oct 2014

UAlberta researchers discover link between pulmonary hypertension, diabetes and cancer

A UAlberta team has discovered that a protein that plays a critical role in metabolism, the process by which the cell generates energy from foods, is important for the development of pulmonary hypertension, a deadly disease.

30 Oct 2014