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 virus-based gene therapy holds promise for Canavan disease

Research led by Paola Leone, PhD, of the University of Medicine and Dentistry of New Jersey-School of Osteopathic Medicine (UMDNJ-SOM), demonstrates the long term safety and benefit of a virus-based gene therapy that has been applied for the first time in a clinical setting.

21 Dec 2012

Two recent experimental treatments improve visual function in mouse models of retinitis pigmentosa

Two recent experimental treatments - one involving skin-derived induced pluripotent stem (iPS) cell grafts, the other gene therapy - have been shown to produce long-term improvement in visual function in mouse models of retinitis pigmentosa (RP), according to the Columbia University Medical Center (CUMC) scientists who led the studies. At present, there is no cure for RP, the most common form of inherited blindness.

20 Dec 2012

Scientists uncover TDP-43 protein as possible target for treatment of ALS, FTD

Neuroscientists at Georgetown University Medical Center say they have new evidence that challenges scientific dogma involving two fatal neurodegenerative diseases - amyotrophic lateral sclerosis (ALS), and frontotemporal dementia (FTD) - and, in the process, have uncovered a possible therapeutic target as a novel strategy to treat both disorders.

20 Dec 2012

AAV-based gene therapy cocktail may help extend lives of children with Canavan disease

Results of a clinical trial that began in 2001 show that a gene therapy cocktail conveyed into the brain by a molecular special delivery vehicle may help extend the lives of children with Canavan disease, a rare and fatal neurodegenerative disorder.

20 Dec 2012

FDA grants Orphan Drug designation to Milo Biotechnology's AAV1-FS344 inhibitor

Milo Biotechnology today announced its AAV1-FS344 has been granted Orphan Drug designation from the FDA's Office of Orphan Products Development for treatment of Becker and Duchenne muscular dystrophy. AAV1-FS344 is a gene therapy-delivered myostatin inhibitor that increases muscle strength.

12 Dec 2012

Activated astrocytes could play a harmful role in Alzheimer's disease

A research team composed of University of Kentucky researchers has published a paper which provides the first direct evidence that activated astrocytes could play a harmful role in Alzheimer's disease.

12 Dec 2012

Weill Cornell research team develops new experimental drug therapy to target DLBCL

Diffuse large B-cell lymphoma (DLBCL) is the most common subtype of non-Hodgkin's lymphoma and the seventh most frequently diagnosed cancer. The most chemotherapy resistant form of DLBCL, called activated B-cell - DLBCL (ABC-DLBCL), remains a major therapeutic challenge.

11 Dec 2012

uniQure commences acute intermittent porphyria Phase I clinical trial

uniQure B.V., a leader in the field of human gene therapy, today announced the start of its Phase I clinical trial in acute intermittent porphyria (AIP) with the treatment of the first patient.

11 Dec 2012

New hope for leukemia patients

Nine of twelve leukemia patients who received infusions of their own T cells after the cells had been genetically engineered to attack the patients' tumors responded to the therapy, which was pioneered by scientists in the Perelman School of Medicine at the University of Pennsylvania

10 Dec 2012

Gene therapy could represent new treatment option for severe orthopaedic injuries

Gene therapy involving modified stem cells obtained from fatty tissue and bone marrow could represent a new option for the treatment of severe orthopaedic injuries to the extremities.

7 Dec 2012

New method can generate stable human mesenchymal stem cells for bone engineering

Human mesenchymal stem cells (hMSCs) can develop into bone cells and are useful for tissue engineering and regeneration. However, when grown in the laboratory they quickly lose their ability to continue dividing and they die.

5 Dec 2012

New Weill Cornell campaign to improve hypertension prevention, detection and control

The Weill Cornell Physician Organization at Weill Cornell Medical College has joined more than 120 medical groups and health systems nationwide to launch the new health campaign Measure Up, Pressure Down aimed to improve high blood pressure prevention, detection and control.

1 Dec 2012

Modified vitamin D receptor may reduce risk of osteoporosis

Osteoporosis, or reduced bone mineral density that can increase the risk of fractures, may affect as many as 30% of women and 12% of men worldwide. One risk factor for osteoporosis is vitamin D deficiency.

30 Nov 2012

Tadalafil may offer therapeutic strategy in patients with Becker muscular dystrophy

Cedars-Sinai Heart Institute researchers have found in an initial clinical trial that a drug typically prescribed for erectile dysfunction or pulmonary hypertension restores blood flow to oxygen-starved muscles in patients with a type of muscular dystrophy that affects males, typically starting in childhood or adolescence.

29 Nov 2012

Amarantus demonstrates excellent diffusion profile of MANF in the striatum of rat brains

Amarantus BioSciences, Inc., a biotechnology company developing new disease-modifying treatments and diagnostics for Parkinson's disease and Traumatic Brain Injury centered on its proprietary anti-apoptosis therapeutic protein MANF, today announced that the Company has successfully completed experiments demonstrating that MANF has an excellent diffusion profile in the striatum of rat brains.

27 Nov 2012

Weill Cornell Medical College dean wins Argentina's Dr. Luis Federico Leloir Prize

Dr. Laurie H. Glimcher, the Stephen and Suzanne Weiss Dean of Weill Cornell Medical College and provost for medical affairs of Cornell University, is a winner of the Dr. Luis Federico Leloir Prize of International Cooperation in Science, Technology and Innovation from Argentina's Ministry of Science, Technology and Productive Innovation.

27 Nov 2012

UPC teams conduct research in biomedical engineering to improve people's health

Systems to improve patient rehabilitation, methods that help detect diseases, and smart biomaterials for optimising treatments—scientific advances in the field of biomedical engineering are unstoppable. A number of leading UPC teams are carrying out research aimed at harnessing technology to improve people's health.

23 Nov 2012

Crucial link between protein synthesis and autism spectrum disorders

Researchers from McGill University and the University of Montreal have identified a crucial link between protein synthesis and autism spectrum disorders (ASD), which can bolster new therapeutic avenues.

22 Nov 2012

Concord Medical third quarter 2012 total net revenues increase 65.2% to RMB206.1 million

Concord Medical Services Holdings Limited, a leading specialty hospital management solution provider and operator of the largest network of radiotherapy and diagnostic imaging centers in China and the parent of Chang'an Hospital, today reported its unaudited consolidated financial results for the third quarter ended September 30, 2012.

21 Nov 2012

Synthetic membrane channels can be constructed through DNA nanotechnology

As reported in the journal Science, physicists at the Technische Universitaet Muenchen (TUM) and the University of Michigan have shown that synthetic membrane channels can be constructed through "DNA nanotechnology."

21 Nov 2012

Gene Therapy News and Research

Further Reading

Novel virus-based gene therapy holds promise for Canavan disease

Two recent experimental treatments improve visual function in mouse models of retinitis pigmentosa

Scientists uncover TDP-43 protein as possible target for treatment of ALS, FTD

AAV-based gene therapy cocktail may help extend lives of children with Canavan disease

FDA grants Orphan Drug designation to Milo Biotechnology's AAV1-FS344 inhibitor

Activated astrocytes could play a harmful role in Alzheimer's disease

Weill Cornell research team develops new experimental drug therapy to target DLBCL

uniQure commences acute intermittent porphyria Phase I clinical trial

New hope for leukemia patients

Gene therapy could represent new treatment option for severe orthopaedic injuries

New method can generate stable human mesenchymal stem cells for bone engineering

New Weill Cornell campaign to improve hypertension prevention, detection and control

Modified vitamin D receptor may reduce risk of osteoporosis

Tadalafil may offer therapeutic strategy in patients with Becker muscular dystrophy

Amarantus demonstrates excellent diffusion profile of MANF in the striatum of rat brains

Weill Cornell Medical College dean wins Argentina's Dr. Luis Federico Leloir Prize

UPC teams conduct research in biomedical engineering to improve people's health

Crucial link between protein synthesis and autism spectrum disorders

Concord Medical third quarter 2012 total net revenues increase 65.2% to RMB206.1 million

Synthetic membrane channels can be constructed through DNA nanotechnology

Industry Focus eBook - Cell & Gene Therapy (1st Edition) eBook

Evaluating new approaches for rare disease genomics

Expanding access to NMR with the Fourier 80 Duo

Advancing Intracellular Delivery with LumiPore®

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