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.

Novel gene therapy can improve symptoms of Bubble Boy disease in young adults

Adolescents and young adults with a severe inherited immunodeficiency disorder improved following treatment with novel gene therapy developed at St. Jude Children's Research Hospital and at the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health. The results of this study appear today in the journal Science Translational Medicine.

21 Apr 2016

Study shows children with rare eye disease have greatest benefit from gene therapy

Scientists at Oregon Health & Science University's Casey Eye Institute and Baylor College of Medicine's Cullen Eye Institute published findings from a two-year Phase I clinical trial in the journal Ophthalmology, which showed that children had the greatest benefit from gene therapy for treatment of Leber congenital amaurosis (LCA) or severe early childhood onset retinal degeneration (SECORD).

18 Apr 2016

University’s blindness therapy comes a step closer following landmark licensing deal with US firm

Hundreds of thousands of people worldwide, who have a disease that can lead to blindness, could have their sight restored after The University of Manchester entered into a technology license with Seattle-based company Acucela Inc.

13 Apr 2016

University's blindness therapy comes a step closer following landmark licensing deal with US firm

13 Apr 2016

New study shows link between FZD7 protein and breast cancer development

A new study shows that Frizzled7 (FZD7), a protein present on human breast epithelial cells and a component of the Wnt signaling pathway is uniquely controlled by the Notch signaling pathway, both of which play key roles in mammary gland formation and breast cancer development.

6 Apr 2016

Liu Laboratory at Augusta University studies exosomes using Particle Metrix’s ZetaView

Particle Metrix, developers of versatile particle characterization solutions for the life sciences, report on the work in the Liu Laboratory at Augusta University which is studying exosomes where size and concentration are critical parameters.

4 Apr 2016

UC San Diego Health participates in nationwide clinical study on hemophilia B gene therapy

The Hemophilia and Thrombosis Treatment Center at UC San Diego Health has joined a nationwide clinical trial testing a potential gene therapy that may one day provide a better and long-lasting treatment for people with hemophilia B.

23 Mar 2016

Delivering microRNAs in cancer treatment: an interview with Dr Conde and Prof Artzi

microRNAs (miRs) are small endogenous noncoding RNA molecules (20–23 nucleotides) derived from imperfectly paired hairpin RNA structures naturally encoded in the genome that act specifically as triggering molecules to control translational repression or mRNA degradation.

11 Mar 2016

ACGT awards new research grants to take researchers to next level in cancer treatment

Alliance for Cancer Gene Therapy, the nation's only nonprofit dedicated exclusively to cell and gene therapies for cancer, is delighted to announce the recipients of its 2016 ACGT Young Investigator Grants.

8 Mar 2016

U-M researchers develop novel mouse model of children's brain cancer

Children with brain cancer may soon get some help from mice with the same disease, thanks to new research from University of Michigan Medical School scientists and their colleagues.

3 Mar 2016

Preceyes, Nightstar partner to develop drug delivery technology for ocular surgery

Preceyes B.V. (NL) and Nightstar (UK) have entered into a collaboration for the development of a high-precision drug delivery technology in the eye. Nightstar will use the Preceyes robotic device to further refine the delivery of gene therapy to the subretinal space for a range of inherited retinal diseases.

1 Mar 2016

Baxalta seeks FDA approval for ADYNOVATE to treat children with hemophilia A and for use in surgical settings

Baxalta Incorporated, a global biopharmaceutical leader dedicated to delivering transformative therapies to patients with orphan diseases and underserved conditions, announced today that it has submitted supplemental Biologics License Applications (sBLAs) to the U.S. Food and Drug Administration seeking approval for the use of ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated] to treat children under the age of 12 with hemophilia A and for use in surgical settings.

26 Feb 2016

New anti-ageing therapy may be effective against aplastic anaemia

Aplastic anaemia is a rare, and potentially fatal, disease of the blood, by which the bone marrow is unable to generate blood cells at the appropriate pace. Many forms of aplastic anaemia share an important link with the ageing process: the shortening of telomeres, structures that protect the ends of chromosomes.

25 Feb 2016

Ohio State researchers develop new approach to enhance heart function, cardiovascular performance

For the first time, scientists at The Ohio State University have engineered new calcium receptors for the heart to tune the strength of the heartbeat in an animal model. The research is published online by the journal Nature Communications.

24 Feb 2016

Pitt experts lead gene therapy clinical trial in Parkinson's disease patients

Experts at the University of Pittsburgh School of Medicine are leading the second arm of a clinical trial using gene therapy to relieve the symptoms of tremor and mobility impairment in patients with Parkinson's disease.

23 Feb 2016

Berlin researchers use T cells to target mutations in cancer cells

The efficacy of the immune system to combat disease makes the body's own healing powers a promising approach in the fight against cancer. A Berlin research group led by Wolfgang Uckert, Thomas Blankenstein and Matthias Leisegang has been collaborating with U.S. researcher Hans Schreiber in a project to use immune components called T cells to target mutant proteins produced by cancer cells.

22 Feb 2016

Neuroscientists identify specific enzyme that plays critical role in spinal muscular atrophy

Neuroscientists have discovered a specific enzyme that plays a critical role in spinal muscular atrophy, and that suppressing this enzyme's activity, could markedly reduce the disease's severity and improve patients' lifestyles.

19 Feb 2016

Study reveals another clue to workings of trachoma

Another clue to the workings of trachoma - the world's leading infectious cause of blindness - has been revealed in a new study published in BMC Infectious Diseases. Researchers identified markers of genetic regulation present in the early stages of infection that could predispose children to developing the condition in its long-term, severe form.

17 Feb 2016

New approach could be used to treat Duchenne muscular dystrophy

Scientists at the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research and Center for Duchenne Muscular Dystrophy at UCLA have developed a new approach that could eventually be used to treat Duchenne muscular dystrophy.

14 Feb 2016

Kumamoto University researchers identify protein that limits severity of Alport syndrome

Researchers from Kumamoto University, Japan have identified a protein that limits the severity of Alport syndrome, a type of genetic kidney disease. The finding can provide clues that point toward new therapeutic approaches for Alport syndrome.

11 Feb 2016