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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.
Belgian receives research grant from Jeffrey Modell to help cure IPEX syndrome

Belgian receives research grant from Jeffrey Modell to help cure IPEX syndrome

For the first time the Jeffrey Modell Foundation is giving a research grant to a Belgian laboratory. The team of Adrian Liston from VIB-KU Leuven will use the grant to develop a gene therapy to cure children that suffer from IPEX syndrome, a rare and fatal autoimmune disorder in which the immune system attacks the body's own tissues and organs. [More]
PHI collaborates with Northeastern University for establishing novel cell analysis applications

PHI collaborates with Northeastern University for establishing novel cell analysis applications

Phase Holographic Imaging (PHI) today announces a collaboration agreement with Northeastern University of Boston. [More]
One-two punch designed to knock out most dangerous brain cancer

One-two punch designed to knock out most dangerous brain cancer

University of Michigan Health System doctors have started testing a unique new approach to fighting brain tumors -- one that delivers a one-two punch designed to knock out the most dangerous brain cancer. [More]
White pill may help scientists learn why patients with cystic fibrosis have less exercise capacity

White pill may help scientists learn why patients with cystic fibrosis have less exercise capacity

A little white pill may help scientists learn why patients with cystic fibrosis have less exercise capacity than their peers, even if their lungs are relatively healthy. [More]
Gene therapy may offer significant advantages in prevention of botulism exposure

Gene therapy may offer significant advantages in prevention of botulism exposure

The current method to treat acute toxin poisoning is to inject antibodies, commonly produced in animals, to neutralize the toxin. But this method has challenges ranging from safety to difficulties in developing, producing and maintaining the anti-serums in large quantities. [More]
Research report: Orphan Diseases Partnering Terms and Agreements

Research report: Orphan Diseases Partnering Terms and Agreements

The Orphan Diseases Partnering Terms and Agreements report provides an understanding and access to the orphan diseases partnering deals and agreements entered into by the worlds leading healthcare companies. [More]
Report on Co-development Terms & Agreements in Pharma, Biotech and Diagnostics

Report on Co-development Terms & Agreements in Pharma, Biotech and Diagnostics

The Co-development Terms and Agreements in Pharma, Biotech and Diagnostics report provides comprehensive understanding and unprecedented access to the co-development deals and agreements entered into by the worlds leading life science companies. [More]
Penn Medicine launches CAROT to build novel therapies for retinal and ocular disorders

Penn Medicine launches CAROT to build novel therapies for retinal and ocular disorders

The Perelman School of Medicine at the University of Pennsylvania has launched the Penn Center for Advanced Retinal and Ocular Therapeutics (CAROT) to build on its previous success developing novel therapies for the personalized diagnosis and treatment of retinal and ocular disorders. [More]
New gene therapy protects mice from life-threatening heart condition

New gene therapy protects mice from life-threatening heart condition

A new gene therapy developed by researchers at the University of Missouri School of Medicine has been shown to protect mice from a life-threatening heart condition caused by muscular dystrophy. [More]
Newborn screening for SCID holds promise that affected children can lead healthy lives

Newborn screening for SCID holds promise that affected children can lead healthy lives

Using population-based screening outcomes of approximately 3 million infants, a team of scientists across 14 states, including four researchers at the University of Massachusetts Medical School, have shown that newborn screening for severe combined immunodeficiency (SCID) can be successfully implemented across public health newborn screening programs. [More]
Early intervention hope for childhood achromatopsia

Early intervention hope for childhood achromatopsia

Researchers have found that children with achromatopsia have milder foveal pathology than their older counterparts, highlighting a potential opportunity for early intervention with gene therapy. [More]
Study examines national impact of newborn screening test for SCID

Study examines national impact of newborn screening test for SCID

Severe combined immunodeficiency (SCID), a potentially life-threatening, but treatable, disorder affecting infants, is twice as common as previously believed, according to a new study that is the first to examine the national impact of this newborn screening test. [More]
Newborn screening indicates that incidence of SCID is higher than previously believed

Newborn screening indicates that incidence of SCID is higher than previously believed

Newborn screening performed in numerous states indicates that the incidence of the potentially life-threatening disorder, severe combined immunodeficiency, is higher than previously believed, at 1 in 58,000 births, although there is a high rate of survival, according to a study in the August 20 issue of JAMA. [More]
Complete analysis of bioseparation technologies for global biopharmaceutical markets

Complete analysis of bioseparation technologies for global biopharmaceutical markets

Reportlinker.com announces that a new market research report is available in its catalogue: ​Bioseparation Systems for Global Biopharmaceutical Markets [More]
Researchers use new gene editing method to correct mutation that leads to DMD

Researchers use new gene editing method to correct mutation that leads to DMD

UT Southwestern Medical Center researchers successfully used a new gene editing method to correct the mutation that leads to Duchenne muscular dystrophy (DMD) in a mouse model of the condition. [More]
New x-ray imaging system helps monitor effectiveness of treatment for cystic fibrosis

New x-ray imaging system helps monitor effectiveness of treatment for cystic fibrosis

Scientists have developed an x-ray imaging system that enables researchers to see 'live' how effective treatments are for cystic fibrosis. [More]
Cardium announces review in Journal of Cardiovascular Pharmacology finds gene therapy for subset of heart disease patients ‘highly warranted’

Cardium announces review in Journal of Cardiovascular Pharmacology finds gene therapy for subset of heart disease patients ‘highly warranted’

Cardium Therapeutics, an operating unit of Taxus Cardium Pharmaceuticals Group Inc. (Trading Symbol: CRXM) has announced the publication of a review article in the Journal of Cardiovascular Pharmacology that concludes a gene therapy product promoting the growth of blood vessels is “highly warranted” to treat about 1 million U.S. heart-disease patients and 6 million more worldwide who are either ineligible or poor candidates for traditional angioplasty, stent placement or bypass surgery. [More]
Oncothyreon acquires Alpine Biosciences to enable targeted delivery of multiple therapeutic agents

Oncothyreon acquires Alpine Biosciences to enable targeted delivery of multiple therapeutic agents

Oncothyreon Inc., a biotechnology company specializing in the development of innovative therapeutic products for the treatment of cancer, today announced that it has acquired Alpine Biosciences, Inc., of Seattle, Washington, a privately held biotechnology company developing protocells, a nanoparticle platform technology designed to enable the targeted delivery of multiple therapeutic agents, including nucleic acids, proteins, peptides and small molecules. [More]
Patient with mechanical heart pump receives new gene therapy for heart failure

Patient with mechanical heart pump receives new gene therapy for heart failure

For the first time in the world, a patient with a mechanical heart pump has received a new gene therapy for heart failure at Harefield Hospital, London. [More]
Isoform: New protein offers novel therapeutic approach for patients with DMD

Isoform: New protein offers novel therapeutic approach for patients with DMD

Scientists have discovered a new form of dystrophin, a protein critical to normal muscle function, and identified the genetic mechanism responsible for its production. [More]