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.

Takeda brings innovative pipeline portfolio to patients for sustained revenue growth

As part of its Wave 1 Pipeline Market Opportunity Call, Takeda Pharmaceutical Company Limited provided an update on its pipeline portfolio, which has the potential to contribute significantly to revenue growth for the company over the next decade.

From Takeda 10 Dec 2020

GlycoNet scientists secure up to $4 million US investment to advance regenerative medicine

GlycoNet scientists Drs. Robert Ben and Jason Acker, who are revolutionizing the cryopreservation of cells, tissues and organs for human and veterinary therapies, have secured a $2-million USD equity investment for PanTHERA CryoSolutions (PanTHERA), a Canadian biotechnology start-up that they co-founded.

8 Dec 2020

Study finds surprising link between ALS, FTD and Huntington's disease-associated mutation

A study led by researchers at the National Institutes of Health has made a surprising connection between frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS), two disorders of the nervous system, and the genetic mutation normally understood to cause Huntington's disease.

5 Dec 2020

Using gene therapy to achieve the positive health effects of physical exercise

Gene therapy is the most effective method to be able to provide health benefits you normally gain through physical exercise.

5 Dec 2020

Researchers discover why older adults are more susceptible to heart disease

A team of researchers at the University of South Florida Department of Surgery has made a key discovery as to why we become more susceptible to heart disease as we age.

3 Dec 2020

Genethon to start international clinical trial for treatment of Duchenne muscular dystrophy

Genethon, dedicated to designing and developing gene therapy products for rare diseases, received this Monday 30th of November the authorization from the ANSM, the French National Agency for Medicines and Health Products Safety, to start in France a multicentre international clinical trial for the treatment of Duchenne muscular dystrophy with product GNT 004.

2 Dec 2020

Researchers develop telomerase gene therapy for treating different pathologies

After several years of research and many joint scientific publications, molecular biologists Maria Blasco (National Cancer Research Centre, CNIO) and Fàtima Bosch (Universitat Autònoma de Barcelona, UAB) have developed a telomerase gene therapy for the treatment of different pathologies related to shortening telomeres and, therefore, ageing.

2 Dec 2020

Study provides new insights into an untreatable form of childhood-onset dementia

Is the eye a window to the brain in Sanfilippo syndrome, an untreatable form of childhood-onset dementia, Australian researchers ask in a new publication.

2 Dec 2020

Study uncovers a promising therapeutic target for retinal dystrophies

A team of LSU Health New Orleans researchers reports for the first time that deleting one of the inhibitors of the RPE65 gene in a mouse model that carries a human disease mutation prevents degeneration of cone photoreceptors that are used for daytime high-resolution color vision.

1 Dec 2020

Experimental stem cell gene therapy may give a new lease of life for patient with sickle cell disease

"But it's a marathon whereas you keep going, the trail gets rockier and then you lose your shoes," the 27-year-old said. "It gets harder as you get older. Things start to fail and all you can think about is how much worse it's going to get down the road."

1 Dec 2020

Adding exercise to genetic treatment reverses fatigue in mice with common myotonic dystrophy

Adding exercise to a genetic treatment for myotonic dystrophy type 1 (DM1) was more effective at reversing fatigue than administering the treatment alone in a study using a mouse model of the disease.

30 Nov 2020

New gene therapy approach offers promise for treating eye disease

Scientists from Trinity College Dublin have developed a new gene therapy approach that offers promise for one day treating an eye disease that leads to a progressive loss of vision and affects thousands of people across the globe.

26 Nov 2020

Basic concepts lay the foundation for personalized immunotherapy

Personalized Immunotherapy for Tumor Diseases and Beyond introduces personalized immunotherapy with multi-dimensional models of analysis to determine the best plan for the immunotherapy of patients.

23 Nov 2020

New findings could pave the way for more effective, safer and cheaper gene therapy

In a promising advance for affordable, personalized medicine, researchers have used metal-organic frameworks to successfully deliver the genetic snipping tool CRISPR/Cas9 into human cancer cells.

20 Nov 2020

New method of repairing damage to optic nerve offers hope for people with glaucoma-related sight loss

Fight for Sight funded researchers have successfully demonstrated a new method of repairing damage to the optic nerve in the lab for the first time, in new research that hopes to restore vision to those who’ve lost it as a result of glaucoma and other conditions.

20 Nov 2020

Texas Heart Institute to participate in a study to treat ARDS in hospitalized COVID-19 patients

Texas Heart Institute has been selected as one of up to 60 sites in the world to participate in an international investigative study seeking to aid COVID-19 patients who develop acute respiratory distress syndrome (ARDS).

19 Nov 2020

NIH project aims to maximize efficiency of AAV platform for gene therapy

Maximizing the efficiency of the adeno-associated virus (AAV) platform for gene therapy is the aim of a new pilot project of the National Institutes of Health (NIH).

19 Nov 2020

Somatic mutation may act as a natural gene therapy in patients with rare autosomal disease

Researchers affiliated with the Center for Cell-Based Therapy in Ribeirão Preto, Brazil, have identified for the first time a non-hereditary mutation in blood cells from a patient with GATA2 deficiency, a rare autosomal disease caused by inherited mutations in the gene that encodes GATA-binding protein 2 (GATA2).

17 Nov 2020

Researchers use aerosolized molecules to shut down proteins generated by coronavirus

Researchers at Georgetown University Medical Center have successfully used molecules comprised of small strands of RNA to shut down the production of destructive proteins generated by the COVID-19 virus.

16 Nov 2020

New data highlights safety concerns on rAAV vectors used in gene therapy

New data highlight safety concerns for the replication of recombinant adeno-associated viral (rAAV) vectors commonly used in gene therapy.

16 Nov 2020

Gene Therapy News and Research

Further Reading

Takeda brings innovative pipeline portfolio to patients for sustained revenue growth

GlycoNet scientists secure up to $4 million US investment to advance regenerative medicine

Study finds surprising link between ALS, FTD and Huntington's disease-associated mutation

Using gene therapy to achieve the positive health effects of physical exercise

Researchers discover why older adults are more susceptible to heart disease

Genethon to start international clinical trial for treatment of Duchenne muscular dystrophy

Researchers develop telomerase gene therapy for treating different pathologies

Study provides new insights into an untreatable form of childhood-onset dementia

Study uncovers a promising therapeutic target for retinal dystrophies

Experimental stem cell gene therapy may give a new lease of life for patient with sickle cell disease

Adding exercise to genetic treatment reverses fatigue in mice with common myotonic dystrophy

New gene therapy approach offers promise for treating eye disease

Basic concepts lay the foundation for personalized immunotherapy

New findings could pave the way for more effective, safer and cheaper gene therapy

New method of repairing damage to optic nerve offers hope for people with glaucoma-related sight loss

Texas Heart Institute to participate in a study to treat ARDS in hospitalized COVID-19 patients

NIH project aims to maximize efficiency of AAV platform for gene therapy

Somatic mutation may act as a natural gene therapy in patients with rare autosomal disease

Researchers use aerosolized molecules to shut down proteins generated by coronavirus

New data highlights safety concerns on rAAV vectors used in gene therapy

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