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.

Scientists discover promising approaches for treatment of pain

Scientists are discovering promising approaches to treating pain, one of the most common and debilitating neurological complaints, according to research released today at Neuroscience 2011, the annual meeting of the Society for Neuroscience and the world's largest source of emerging news about brain science and health.

15 Nov 2011

Damaged mitochondria equivalent to an environmental disaster in the cell

Current thinking about Parkinson's disease is that it's a disorder of mitochondria, the energy-producing organelles inside cells, causing neurons in the brain's substantia nigra to die or become impaired.

11 Nov 2011

Clinical diagnoses of autism may vary widely across centers

To diagnose autism spectrum disorders, clinicians typically administer a variety of tests or scales and use information from observations and parent interviews to classify individuals into subcategories listed in standard psychiatric diagnostic manuals.

10 Nov 2011

New genetic approach to treat fibrodysplasia ossificans progressiva

Scientists at Penn's Perelman School of Medicine Center for Research in FOP and Related Disorders have developed a new genetic approach to specifically block the damaged copy of the gene for a rare bone disease, while leaving the normal copy untouched.

9 Nov 2011

Gene therapy and stem cell transplantation successfully reverse hemophilia A

For the first time, researchers have combined gene therapy and stem cell transplantation to successfully reverse the severe, crippling bleeding disorder hemophilia A in large animals, opening the door to the development of new therapies for human patients.

3 Nov 2011

Clinical-grade viral vector can cure choroideremia

Researchers at Ohio State University Medical Center and Nationwide Children's Hospital have developed a viral vector designed to deliver a gene into the eyes of people born with an inherited, progressive form of blindness that affects mainly males.

3 Nov 2011

Fierce Vaccines highlights Inovio's VGX-3100 as one of 10 Promising Therapeutic Vaccines

Inovio Pharmaceuticals, Inc., which is advancing synthetic vaccines to fight cancers and infectious diseases, announced today that VGX-3100, its investigational SynCon vaccine for treating cervical dysplasia and cancer caused by human papillomavirus, was highlighted by Fierce Vaccines as one of 10 Promising Therapeutic Vaccines.

31 Oct 2011

Mayo Clinic, Oxford BioMedica collaborate to develop gene therapy for glaucoma

Oxford BioMedica plc, the leading gene-based biopharmaceutical company, today announces that it has entered into a research and development collaboration with Mayo Clinic, Rochester (USA) to develop a novel gene therapy for the treatment of chronic glaucoma.

31 Oct 2011

Researchers uncover biochemical signals that trigger generation of new lung alveoli

Researchers at Weill Cornell Medical College say they have taken an important step forward in their quest to "turn on" lung regeneration -- an advance that could effectively treat millions of people suffering from respiratory disorders.

29 Oct 2011

Study provides details on genetics behind many metabolic diseases

In what is so far the largest investigation of its kind, researchers uncovered a wide range of new insights about common diseases and how they are affected by differences between two persons' genes. The results from this study could lead to highly targeted, individualized therapies.

27 Oct 2011

HMS, EPFL to collaborate on 6 pioneering neuroengineering projects

Two of the world's leading universities are joining forces to combine neuroscience and engineering in order to alleviate human suffering caused by such neurological disabilities as paralysis and deafness. Scientists, engineers, and clinicians at Harvard Medical School (HMS) and Ecole Polytechnique F-d-rale de Lausanne (EPFL) will collaborate on six pioneering neuroengineering projects made possible thanks to a $3.6 million grant from the Bertarelli Foundation.

24 Oct 2011

Weill Cornell researcher to study link between obesity, inflammation and breast cancer

Funded through Bloomingdale's annual Pink Campaign, The Breast Cancer Research Foundation (BCRF) is awarding a grant to Dr. Andrew J. Dannenberg of Weill Cornell Medical College on Oct. 26 to fund research into the link between obesity, inflammation and breast cancer. Dr. Dannenberg has been a BCRF grantee since 2004.

21 Oct 2011

RetroSense, WSU sign license agreement for channelrhodopsin-based approaches

RetroSense Therapeutics, LLC, a Michigan-based company, announced that it has executed its exclusive, worldwide option and signed a license agreement for novel gene-therapy approaches for treating blindness developed at Wayne State University's School of Medicine.

19 Oct 2011

FDA approves Oxford BioMedica's UshStat IND to treat Usher syndrome type 1B

Oxford BioMedica plc, the leading gene-based biopharmaceutical company, today announces that the US Food and Drug Administration has approved its Investigational New Drug application for the Phase I/IIa clinical development of UshStat, a novel gene-based treatment for Usher syndrome type 1B.

18 Oct 2011

NEP successfully inserts specific doses of anti-cancer gene into leukemia cells

For the first time, researchers have found a way to inject a precise dose of a gene therapy agent directly into a single living cell without a needle.

18 Oct 2011

Researchers demonstrate that iPS stem cells may be used for gene therapy

This research, published on Oct. 12 on the Nature review website, provides evidence of a major concept could pave the way for the future use of these stem cells to treat humans, through perspective gene therapies.

14 Oct 2011

ESCs express genes linked with early germ cell specification

Ordinarily, embryonic stem cells exist only a day or two as they begin the formation of the embryo itself. Then they are gone. In the laboratory dish, however, they act more like perpetual stem cells - renewing themselves and exhibiting the ability to form cells of almost any type, a status called totipotency.

14 Oct 2011

New insights into how adenovirus triggers an immune response

A Loyola University Chicago Stritch School of Medicine study could lead to improved gene therapies for conditions such as heart disease and cancer as well as more effective vaccines for tuberculosis, malaria and other diseases.

13 Oct 2011

Incoming dean of Weill Cornell receives 2011 ICS Honorary Lifetime Membership

Dr. Laurie Glimcher, the incoming dean of Weill Cornell Medical College, has received the 2011 International Cytokine Society (ICS) Honorary Lifetime Membership, the Society's highest honor, for her pivotal contributions to cytokine research, particularly as it relates to immune response.

12 Oct 2011

Tips on how to prevent unnecessary medication/supplement mishaps

Navigating the supplement and medication maze can get confusing especially when you have multiple prescriptions. It is important to organize yourself with your medications and supplements to prevent unnecessary mishaps.

7 Oct 2011