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.

Scientists come together to better understand patented innovations available for gene therapy

The global gene therapy market is expected to reach $13 billion by 2024 as new treatment options target cancers and other diseases.

9 Feb 2020

New study sheds light on human in vitro organ model research trends

Two distinct approaches are predominantly used to recapitulate physiologically relevant in vitro human organ models.

9 Feb 2020

St. Jude's researchers receive ACGT grant to tackle challenges of pediatric sarcomas

An innovative approach to tackling the challenges of pediatric sarcomas is being pursued by researchers at St. Jude Children's Research Hospital (Memphis, Tennessee) with a new grant from Alliance for Cancer Gene Therapy.

9 Feb 2020

New gene therapy improves vision in blind mice

Mice born blind have shown significant improvement in vision after undergoing a new gene therapy developed by a team of Japanese scientists.

7 Feb 2020

Gene therapy approach could pave the way for treatment of Stargardt disease

A leading Professor of Ophthalmology at the University of Oxford has shared the details of his research which could pave the way for a new treatment for sight loss condition Stargardt disease.

7 Feb 2020

Rejuvenate Bio secures license to commercialize gene therapy for treating age-related diseases in dogs

Rejuvenate Bio has secured a license to commercialize a gene therapy technology developed at the Wyss Institute to treat multiple age-related diseases in dogs, which could open the door to similar treatments for humans in the future.

6 Feb 2020

Gene-editing taken to advanced levels in human stem cells

A new study published in the journal Stem Cell Reports in January 2020 reports the use of an adaptation to a commonly used gene editing technology to achieve a more than 900% increase in efficiency.

30 Jan 2020

New way to treat stroke using an already FDA-approved drug shows promise

Stroke is the third leading cause of death and disability in the United States. More than 87 percent are ischemic strokes, caused by obstruction of one or more cerebral arteries. With limited progress in developing treatments, there is a critical need for neuroprotective agents to effectively treat stroke.

29 Jan 2020

Successful gene therapy trial in patients with X-linked Chronic Septic Granulomatosis

American and British teams led by Drs Kohn (University of California, Los Angeles), Malech (NIH), Williams (Boston Children's) and Trasher (Great Ormond Street Institute of Child Health) published yesterday in Nature Medicine the conclusive results of a gene therapy trial conducted in the United States and Great Britain in 9 patients with X-linked Chronic Septic Granulomatosis (X-CGD), a rare and severe immune dysfunction. Six of them are free of treatment for complications generated by the disease.

28 Jan 2020

New gene correction therapy for hereditary muscular disease among children

Duchenne type muscular dystrophy is the most common hereditary muscular disease among children, leaving them wheelchair-bound before the age of twelve and reducing life expectancy.

27 Jan 2020

Researchers identify novel cellular entry factor for AAV vector types

Researchers led by a team at Massachusetts Eye and Ear have identified a novel cellular entry factor for adeno-associated virus vector (AAV) types--the most commonly used viral vectors for in vivo gene therapy. AAVs are vectors-or vehicles--that are created from a virus that is made harmless by molecular engineering, and have shown promise transporting genetic therapy treatments to affected tissues.

23 Jan 2020

Synthego adopts GMP standards for producing sgRNA

Synthego has adopted Good Manufacturing Practice standards for producing customized GMP-grade synthetic guide RNA at its facilities, an initiative the company said will benefit its academic and industry customers as they develop advanced cell and gene therapies.

From Synthego Corporation 23 Jan 2020

Researchers identify potential way to halt age-related macular degeneration

Researchers have successfully treated age-related macular degeneration in mice after finding an unexpected link between the two main forms of the blinding eye disease, the leading cause of vision loss in people 60 and older.

22 Jan 2020

Potential Parkinson's treatment effectively targets a specific group of brain cells

Scientists have discovered that a non-invasive technique which could one day be used to treat Parkinson's disease, can successfully target a highly specific group of brain cells which play a key role in development of the condition.

22 Jan 2020

Study shows how gene therapy may help protect against vision loss in multiple sclerosis

New research by Dorothy P. Schafer, PhD, at the University of Massachusetts Medical School, reveals the molecular process in which synaptic connections in the brain are damaged in multiple sclerosis and how this contributes to neurodegenerative symptoms.

21 Jan 2020

Gene Therapy News and Research

Further Reading

Scientists come together to better understand patented innovations available for gene therapy

New study sheds light on human in vitro organ model research trends

St. Jude's researchers receive ACGT grant to tackle challenges of pediatric sarcomas

New gene therapy improves vision in blind mice

Gene therapy approach could pave the way for treatment of Stargardt disease

Rejuvenate Bio secures license to commercialize gene therapy for treating age-related diseases in dogs

Gene-editing taken to advanced levels in human stem cells

New way to treat stroke using an already FDA-approved drug shows promise

Successful gene therapy trial in patients with X-linked Chronic Septic Granulomatosis

New gene correction therapy for hereditary muscular disease among children

Researchers identify novel cellular entry factor for AAV vector types

Synthego adopts GMP standards for producing sgRNA

Researchers identify potential way to halt age-related macular degeneration

Potential Parkinson's treatment effectively targets a specific group of brain cells

Study shows how gene therapy may help protect against vision loss in multiple sclerosis

Discovery of new killer T-cell offers hope of a 'one-size-fits-all' cancer therapy

SITESEEKER to be used for Otsuka’s therapeutics discovery programs

Horizon Discovery to offer novel base editing technology for clinical research

Scientists discover a new way to combine coding languages into single "bilingual" molecule

Scientists find way to combine two main coding languages into single 'bilingual' molecule

Leveraging the power of automation to boost research

Automating research - solutions and best practices

How can microdialysis benefit drug development

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