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.

Researchers use advanced photodynamic therapy to combat ovarian cancer in laboratory animals

Researchers at Oregon State University have made a significant advance in the use of photodynamic therapy to combat ovarian cancer in laboratory animals, using a combination of techniques that achieved complete cancer cell elimination with no regrowth of tumors.

14 Aug 2015

Charlotte charity Taylor's Tale inspires new law to create treatments for rare diseases, spur growth in NC

Charlotte charity Taylor's Tale helped inspire a law designed to stimulate the creation of new treatments for rare diseases and spur economic development in North Carolina.

13 Aug 2015

NFIX protein drives NSC differentiation toward oligodendrocytes

An international team of researchers has shown that NFIX, a protein that regulates neuronal stem cell activity (NSC), also has a role in driving NSC differentiation toward oligodendrocytes, a type of glial cell. These cells produce the myelin that surrounds and protects neurons.

7 Aug 2015

University of Zurich professor recognized with 2015 Vallee Young Investigator Award

Martin Jinek, a professor at the University of Zurich's Department of Biochemistry, was presented with the 2015 Vallee Young Investigator Award. The international prize is awarded to young researchers for outstanding achievements in biomedicine and carries USD 250,000 in prize money.

6 Aug 2015

Penn Medicine devises new approach to develop vaccines against lethal diseases

Researchers at the Perelman School of Medicine at the University of Pennsylvania have devised an entirely new approach to vaccines - creating immunity without vaccination.

4 Aug 2015

Researchers reconstruct ancient virus to improve gene therapy

Researchers at Massachusetts Eye and Ear/Schepens Eye Research Institute have reconstructed an ancient virus that is highly effective at delivering gene therapies to the liver, muscle, and retina. This discovery, published July 30 in Cell Reports, could potentially be used to design gene therapies that are not only safer and more potent than therapies currently available, but may also help a greater number of patients.

31 Jul 2015

Novel 3D human skin tissue model could help detect presence of known skin sensitizers in medical device extracts

New research shows that exposing a 3D human skin tissue model to extracts of medical device materials can detect the presence of sensitizers known to cause an allergic response on contact in some individuals. Conventional skin sensitization testing of medical devices relies on animal testing, whereas human skin models could replace animal methods, according to an article in the new journal Applied In Vitro Toxicology, a peer-reviewed publication from Mary Ann Liebert, Inc., publishers.

30 Jul 2015

Innovative approach to treating AAT deficiency

Researchers have demonstrated the feasibility of delivering an RNA that encodes for the protein alpha-1-antitrypsin (AAT)--which is missing or nonfunctional in the genetic disorder AAT deficiency--into cells in the laboratory, enabling the cells to produce highly functional AAT.

28 Jul 2015

Infants born with mutation in PLVAP gene develop severe protein losing enteropathy

Newborn children born with a mutation in the Plasmalemma Vesicle Associated Protein (PLVAP) gene develop severe protein losing enteropathy, according to a case study1 published in Cellular and Molecular Gastroenterology and Hepatology, the basic science journal of the American Gastroenterological Association.

23 Jul 2015

New TCR therapy demonstrates encouraging clinical responses in patients with multiple myeloma

Results from a clinical trial investigating a new T cell receptor (TCR) therapy that uses a person's own immune system to recognize and destroy cancer cells demonstrated a clinical response in 80 percent of multiple myeloma patients with advanced disease after undergoing autologous stem cell transplants (ASCT).

22 Jul 2015

Adaptimmune’s NY-ESO-1 TCR-engineered T-cell therapy mediates sustained antitumor effects in multiple myeloma patients

Adaptimmune Therapeutics plc, a clinical stage biopharmaceutical company focused on the use of T-cell therapy to treat cancer, today announced that data from its Phase I/II study of its affinity enhanced T-cell receptor therapeutic targeting the NY-ESO-1 cancer antigen in patients with multiple myeloma has been published in Nature Medicine.

22 Jul 2015

Rowan University, Bamboo Therapeutics partner to combat Canavan disease

Rowan University and Bamboo Therapeutics, Inc. have entered into an asset transfer agreement to commercialize a novel gene therapy for the treatment of Canavan disease. A rare but devastating neurological disease that tragically takes a child's life by age 10, Canavan disease is one of the most common and complex degenerative cerebral diseases in infants.

17 Jul 2015

Gene therapy used for sight restoration also strengthens visual pathways in the brain

Since 2007, clinical trials using gene therapy have resulted in often-dramatic sight restoration for dozens of children and adults who were otherwise doomed to blindness. Now, researchers from the Perelman School of Medicine at the University of Pennsylvania and The Children's Hospital of Philadelphia, have found evidence that this sight restoration leads to strengthening of visual pathways in the brain, published this week in Science Translational Medicine.

16 Jul 2015

Research breakthrough opens door to a world of regenerative medicine for treating mitochondrial disease

A study led by Shoukhrat Mitalipov, Ph.D., and Hong Ma, M.D., Ph.D., at the Center for Embryonic Cell and Gene Therapy at Oregon Health & Science University and the Oregon National Primate Research Center has revealed the first critical step in developing novel gene and stem cell therapy treatments for patients with mitochondrial disease.

16 Jul 2015

Gene therapy provides life-long protection to photoreceptor cells in animal model of retinitis pigmentosa

A collaboration between scientists in the UK and the USA has shown that gene therapy can give life-long protection to the light-sensitive photoreceptor cells responsible for colour vision in a mouse model of the most common inherited eye disorder.

15 Jul 2015

New study reveals highly promising approach to coating tissue engineered constructs

A new study showing the ability to apply a thin coating of viable respiratory epithelial cells to tissue engineered constructs using a commercially available spray device is especially promising for therapeutic approaches in development to repair or replace challenging structures such as trachea or bronchi.

15 Jul 2015

Hiroshima University researchers reveal molecular mechanisms involved in neuropathic pain

A research group from Hiroshima University demonstrated that the downregulation of spinal astrocyte connexin43 (Cx43) expression causes sustained neuropathic pain following peripheral nerve injury. Controlling the Cx43 expression using pharmacological approaches or gene therapy might serve as novel therapeutic strategies ameliorate neurological disorders in general and neuropathic pain in particular.

14 Jul 2015

Novel gene therapy control system regulates expression of therapeutic transgenes

Korean researchers have described a novel control system to regulate the expression of a therapeutic transgene by targeting the passenger strand of a microRNA (miR-122) linked to the transgene.

14 Jul 2015

Researchers reveal how certain lipids help adenoviruses to enter the cell

Adenoviruses cause numerous diseases, such as eye or respiratory infections, and they are widely used in gene therapy. Researchers from the University of Zurich have now discovered how these viruses penetrate the cells, a key step for infection and gene delivery. The cell unwillingly supports virus entry and infection by providing lipids that are normally used to repair damaged membranes.

10 Jul 2015

TSRI study reveals alternative approach to current anti-HIV strategies

HIV-infected patients remain on antiretroviral therapy for life because the virus survives over the long-term in infected dormant cells. Interruption of current types of antiretroviral therapy results in a rebound of the virus and clinical progression to AIDS.

9 Jul 2015