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.

Albumedix enters into collaboration agreement with Cell and Gene Therapy Catapult

Albumedix Ltd. (‘Albumedix’), an enabler of advanced therapies and the world leader in recombinant human albumin (rAlb), announced today that they have entered into a collaboration agreement with Cell and Gene Therapy Catapult (CGT Catapult) to investigate the use of Albumedix´ proprietary albumin-based solutions for advanced therapy applications, including viral vectors manufacturing.

From Albumedix Ltd. 2 Feb 2021

Life-threatening genetic immunodeficiency reversed by a rare DNA change

Researchers have revealed how a rare DNA change rebalanced the immune system of patients with a life-threatening genetic immunodeficiency.

1 Feb 2021

New gene-based vaccine strategy receives grant from the Bill & Melinda Gates Foundation

The AAVCOVID vaccine program, a novel gene-based vaccine strategy that utilizes an adeno-associated virus (AAV) vector, was granted an award for up to $2.1 million from the Bill & Melinda Gates Foundation.

29 Jan 2021

NEI scientists develop and test a promising gene therapy strategy for rare eye disease

Scientists at the National Eye Institute have developed a promising gene therapy strategy for a rare disease that causes severe vision loss in childhood.

28 Jan 2021

Scientist wins 2021 Pioneer in Basic Science Award for breakthrough research on the inner ear

When Andrew Griffith, MD, PhD, looks at the inner ear, he sees what he describes as "an engineering marvel."

28 Jan 2021

Insight into cancer cells' own first aid could provide the basis for new immunotherapy

Insight into cancer cells' own first aid could help the development of a new type of treatment. Johanna Olweus and her team at the UiO (University of Oslo) and the OUS (Oslo University Hospital) are important contributors to this study, which has been published in Nature.

27 Jan 2021

Using gene therapy to mimic the effects of physical exercise

It sounds too good to be true - and it is. But Jose Bianco Moreira and the CERG research group at the Norwegian University of Science and Technology are convinced that some of the positive health effects of physical exercise can be achieved using gene therapy and medication.

21 Jan 2021

Gene editing shows potential to correct mutations causing inherited retinal degeneration

Gene editing therapies, including CRISPR-Cas systems, offer the potential to correct mutations causing inherited retinal degenerations, a leading cause of blindness.

20 Jan 2021

New therapeutic approach enables functional recovery after spinal cord injury in mice

Using gene therapy, a research team has succeeded for the first time in getting mice to walk again after a complete cross-sectional injury. The nerve cells produced the curative protein themselves.

16 Jan 2021

Myelin‐specific T cells implicated in central nervous system disease in monkeys

Scientists have uncovered new clues implicating a type of herpes virus as the cause of a central nervous system disease in monkeys that's similar to multiple sclerosis in people.

15 Jan 2021

OXGENE introduces SLIM platform for discovery of antibodies against membrane proteins

OXGENE™ announces the introduction of its self-labelling integral membrane (SLIM™) protein discovery system.

12 Jan 2021

Study finds association between severity of COVID-19 and shorter telomeres

Patients with severe COVID-19 disease have significantly shorter telomeres, according to a study conducted by researchers at the Spanish National Cancer Research Centre in collaboration with the COVID-IFEMA Field Hospital, published in the journal Aging.

11 Jan 2021

Gene therapy can effectively treat mice with tuberous sclerosis complex, shows study

Patients with tuberous sclerosis complex, a genetic disorder characterized by the growth of noncancerous tumors in multiple organs of the body, have limited treatment options.

8 Jan 2021

Huntington’s disease mutant gene affects brain and body growth in childhood

There is growing evidence to support the hypothesis that there is a neurodevelopmental component to the late-onset neurodegeneration occurring in the brain of huntingtin gene (HTT gene) mutation carriers, and that this increased susceptibility to brain cell death begins during childhood.

6 Jan 2021

Study presents novel gene therapy for deafness

A new study from Tel Aviv University presents an innovative treatment for deafness, based on the delivery of genetic material into the cells of the inner ear.

23 Dec 2020

Researchers create new polymer to deliver DNA and RNA-based therapies for diseases

University of Minnesota Twin Cities researchers in the Department of Chemistry have created a new polymer to deliver DNA and RNA-based therapies for diseases.

21 Dec 2020

Thermo Fisher announces construction of new cGMP plasmid DNA manufacturing facility

Thermo Fisher Scientific, the world leader in serving science, today announced the construction of a new cGMP plasmid DNA manufacturing facility in Carlsbad, Calif.

From Thermo Fisher Scientific Inc. 18 Dec 2020

Study reveals innovative gene therapy approach to regenerate functional new neurons

Spinal cord injury (SCI) often causes disability and seriously compromises quality of life. While decades of research have made significant progress in axonal regeneration after SCI, most of the interventions have not been translated into clinical therapies.

18 Dec 2020

New data shows encouraging results for patients with neuro-degenerative blinding eye disease

New data published in the Journal Science Translational Medicine shows encouraging results in a worldwide clinical trial for patients diagnosed with the neuro-degenerative blinding eye disease; Leber Hereditary Optic Neuropathy (LHON.)

16 Dec 2020

Researchers identify adenovirus gene therapy for placental insufficiency

A new study identified an adenovirus gene therapy vector carrying a VEGF isoform. It can improve uterine blood flow in placental insufficiency, as reported in the peer-reviewed journal Human Gene Therapy.

15 Dec 2020