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.

Improved gene therapy treatment shows promise in mice with cystic fibrosis

An improved gene therapy treatment can cure mice with cystic fibrosis (CF). Cell cultures from CF patients, too, respond well to the treatment. Those are the encouraging results of a study presented by the Laboratory for Molecular Virology and Gene Therapy at KU Leuven, Belgium.

17 Nov 2015

CHOP researchers delay symptoms, extend lifespan in animal model of Batten disease

Researchers have taken a significant step forward in developing gene therapy against a fatal neurodegenerative disease that strikes children. By delivering a working version of a gene to produce a key enzyme that is lacking in Batten disease, the scientists delayed symptoms and extended lifespan in dogs with a comparable disease.

12 Nov 2015

Nightstar announces $35 million Series B financing round

NightstaRx Ltd, a biopharmaceutical company specialising in developing gene therapies for inherited retinal dystrophies, announces a $35 million Series B financing round. The round was led by New Enterprise Associates, one of the world's leading venture capital firms.

9 Nov 2015

Novel genetic therapy treats ‘incurable’ leukaemia

A new treatment that involves editing DNA has been used for the first time at Great Ormond Street Hospital to successfully treat drug-resistant leukaemia.

6 Nov 2015

Researchers successfully repair nerve cell damage in Alzheimer's dementia

In laboratory experiments on the basic mechanisms that cause Alzheimer's dementia, an international research team led by Heidelberg neurobiologist Prof. Dr. Ulrike Müller and a team of French scientists have succeeded in largely "repairing" the nerve cell damage typical in this disease.

5 Nov 2015

Approval to start trial of Videregen's replacement trachea technology

The INSPIRE consortium, led by Videregen Ltd, has received approval from the MHRA1 to start a UK clinical trial with its tissue engineered replacement trachea.

3 Nov 2015

Scalable production of gene therapy vectors: an interview with Frank Ubags

Gene therapy involves introducing genetic material into cells to replace missing or malfunctioning genes or to add a gene to express a beneficial protein in order to treat certain diseases, such as cancer or inherited disorders.

26 Oct 2015

MU researchers successfully treat dogs with DMD, plan for human clinical trials

Muscular dystrophy, which affects approximately 250,000 people in the U.S., occurs when damaged muscle tissue is replaced with fibrous, fatty or bony tissue and loses function. For years, scientists have searched for a way to successfully treat the most common form of the disease, Duchenne Muscular Dystrophy (DMD), which primarily affects boys. Now, a team of University of Missouri researchers have successfully treated dogs with DMD and say that human clinical trials are being planned in the next few years.

23 Oct 2015

Leptin gene therapy aids weight loss without significant side effect of bone loss

Delivering the hormone leptin directly to the brain through gene therapy aids weight loss without the significant side effect of bone loss, according to new collaborative research from Oregon State University and University of Florida.

22 Oct 2015

New study from TSRI and Salk points to cause of debilitating nerve disease

Scientists at The Scripps Research Institute (TSRI) and the Salk Institute for Biological Studies have discovered how a mutant protein triggers nerve damage in a subtype of Charcot-Marie-Tooth (CMT) diseases, a group of currently untreatable conditions that cause loss of function in a person’s hands and feet.

22 Oct 2015

Restoring memory in aging mice

A new study, led by scientists at The Scripps Research Institute (TSRI), the Veterans Affairs San Diego Healthcare System (VA) and University of California (UC) San Diego School of Medicine shows that increasing a crucial cholesterol-binding membrane protein in nerve cells (neurons) within the brain can improve learning and memory in aged mice.

22 Oct 2015

Gene therapy preserves vision in canine model of late-stage retinitis pigmentosa

Gene therapy preserved vision in a study involving dogs with naturally occurring, late-stage retinitis pigmentosa, according to research funded by the National Eye Institute (NEI), part of the National Institutes of Health. The findings contribute to the groundwork needed to move gene therapy forward into clinical trials for people with the blinding eye disorder, for which there is currently no cure.

15 Oct 2015

enGene, Janssen Biotech sign agreement to develop and commercialize new IBD therapies

enGene, Inc. a gene therapy company focused on development of therapeutics using its proprietary "Gene Pill" and enema gene delivery platform, today announced that it has entered into a definitive agreement with Janssen Biotech, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson, to develop and commercialize new therapies for inflammatory bowel disease (IBD).

14 Oct 2015

New hope for patients with Duchenne muscular dystrophy

New hope for Duchenne muscular dystrophy (DMD) patients. A mouse genetic study in PLoS Medicine reports that targeting the P2RX7 gene, a purinoreceptor, may halt the progression of DMD.

14 Oct 2015

Sense oligonucleotide antidote reverses actions of antisense antithrombotic drug, prevents bleeding

Researchers from Isis Pharmaceuticals (Carlsbad, CA) and Prysis Biotechnologies (Pudong, Shanghai, China) have demonstrated proof-of-concept for using a sense oligonucleotide to undo the effects of an antisense drug, an antithrombotic agent in this novel study.

14 Oct 2015

New breakthrough may provide key to understanding how to treat Muscular Dystrophy

New research published today in the journal eLife has demonstrated a new method for observing the behaviour of the protein Dystrophin in a living animal cell, in real-time. This breakthrough may provide a key to understanding how to treat the genetic disease, Muscular Dystrophy.

14 Oct 2015

Penn researchers show that canine X-linked retinitis pigmentosa can be cured over the long term

Three years ago, a team from the University of Pennsylvania announced that they had cured X-linked retinitis pigmentosa, a blinding retinal disease, in dogs. Now they've shown that they can cure the canine disease over the long term, even when the treatment is given after half or more of the affected photoreceptor cells have been destroyed.

13 Oct 2015

Gene therapy prevents neuronal death, disease effects in experimental model of PD

An estimated seven to ten million people worldwide are living with Parkinson's disease (PD), which is an incurable and progressive disease of the nervous system affecting movement and cognitive function. More than half of PD patients develop progressive disease showing signs of dementia similar to Alzheimer's disease.

9 Oct 2015

Cell Therapy Catapult launches Cell History File

The Cell Therapy Catapult, the organisation dedicated to the growth of the UK cell and gene therapy industry by bridging the gap between scientific research and commercialisation, today announces the availability of the Cell History File (CHF) template document. The Cell History File template has been developed in conjunction with the UK’s Medicines and Healthcare products Regulatory Agency (MHRA), the Human Tissue Authority (HTA) and other cell therapy developers and manufacturers from academia and industry.

7 Oct 2015

Lonza, Benitec partner to develop scalable manufacturing process for AAV-based viral gene therapies

Lonza Houston, Inc., a global leader in biological, cell and viral gene therapy manufacturing, and Benitec Biopharma, a clinical stage biotechnology company commercializing a patented gene-silencing technology, DNA-directed RNA interference (ddRNAi), today announced that they have entered into a Manufacturing Services Agreement to develop a scalable manufacturing process for Benitec's ddRNAi-based, Adeno-Associated Virus (AAV)-delivered products intended for therapeutic use in humans.

7 Oct 2015

Gene Therapy News and Research

Further Reading

Improved gene therapy treatment shows promise in mice with cystic fibrosis

CHOP researchers delay symptoms, extend lifespan in animal model of Batten disease

Nightstar announces $35 million Series B financing round

Novel genetic therapy treats ‘incurable’ leukaemia

Researchers successfully repair nerve cell damage in Alzheimer's dementia

Approval to start trial of Videregen's replacement trachea technology

Scalable production of gene therapy vectors: an interview with Frank Ubags

MU researchers successfully treat dogs with DMD, plan for human clinical trials

Leptin gene therapy aids weight loss without significant side effect of bone loss

New study from TSRI and Salk points to cause of debilitating nerve disease

Restoring memory in aging mice

Gene therapy preserves vision in canine model of late-stage retinitis pigmentosa

enGene, Janssen Biotech sign agreement to develop and commercialize new IBD therapies

New hope for patients with Duchenne muscular dystrophy

Sense oligonucleotide antidote reverses actions of antisense antithrombotic drug, prevents bleeding

New breakthrough may provide key to understanding how to treat Muscular Dystrophy

Penn researchers show that canine X-linked retinitis pigmentosa can be cured over the long term

Gene therapy prevents neuronal death, disease effects in experimental model of PD

Cell Therapy Catapult launches Cell History File

Lonza, Benitec partner to develop scalable manufacturing process for AAV-based viral gene therapies

Leveraging the power of automation to boost research

Automating research - solutions and best practices

How can microdialysis benefit drug development

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