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.

Innovate UK funding to support the development of powdered gene therapy for bone graft augmentation

TherageniX, a University of Nottingham spin out pioneering a dry powder gene therapy formulation for bone graft augmentation, and its collaborator, the University of Nottingham, have been awarded a £995,000 grant from Innovate UK, the UK’s innovation agency.

4 Sep 2023

TherageniX and University of Nottingham awarded £995K Innovate UK grant to develop powdered gene therapy for bone regeneration

Funding from Innovate UK’s Transforming Medicines Manufacturing program supporting innovative technologies for intracellular drug delivery.

1 Sep 2023

New gene therapy offers hope for beta thalassemia patients

UCSF Benioff Children's Hospital Oakland provides new therapy to first patient since FDA approval.

31 Aug 2023

Stem cell gene therapy may offer a promising, curative treatment for sickle cell disease

New research published in the New England Journal of Medicine indicates that stem cell gene therapy may offer a promising, curative treatment for the painful, inherited blood disorder sickle cell disease (SCD).

30 Aug 2023

Gene therapy reduces hearing loss associated with Norrie disease in mice

An international team led by researchers at UCL and the NIHR Great Ormond Street Hospital Biomedical Research Centre have developed in mice a gene therapy that significantly reduces the hearing loss associated with Norrie disease.

29 Aug 2023

Drexel University receives grants to support cell and gene therapy education

Drexel University's School of Biomedical Engineering, Science and Health Systems, in collaboration with Drexel's College of Medicine, has received grants from the pharmaceutical company Bristol Myers Squibb, to support the education and training of diverse and talented students looking to pursue careers in cell and gene therapy.

25 Aug 2023

Engineered adeno-associated virus vector yields high transduction of the brain vasculature

Researchers have developed an engineered adeno-associated virus (AAV) vector that yields high transduction of brain vascular pericytes and smooth muscle cells.

24 Aug 2023

Waters launches XBridge Premier GTx BEH SEC columns for gene therapy applications

Waters Corporation introduced the first in a new line of size exclusion chromatography (SEC) columns aimed at improving the analysis while lowering the cost of gene therapies, specifically adeno-associated viral (AAV) vectors.

From Waters Corporation 24 Aug 2023

Gene therapy repairs neural connections in mice with Hurler syndrome

A new study from the University of Minnesota is the first to demonstrate the ability for gene therapy to repair neural connections for those with the rare genetic brain disorder known as Hurler syndrome.

21 Aug 2023

Oncolytic viruses could be used to 'warm up' cold tumors and improve immunotherapy

Equipping cancer-infecting, or oncolytic, viruses with tumor-inhibiting genetic cargo stimulates the immune system and helps immunotherapy to shrink or completely clear aggressive tumors in mice, according to a new study in the Journal of Experimental Medicine led by University of Pittsburgh and UPMC researchers.

14 Aug 2023

New gene therapy shows promise for treating fusion-driven cancers

A new study presents a promising treatment method for so-called fusion-driven cancers, which are currently often difficult to cure. These fusion-driven cancers are caused by an error in cell division that creates a fusion of different genes.

11 Aug 2023

CRISPR/sgRNA-SAM therapy: a potential breakthrough for Parkinson's disease?

Researchers utilize the CRISPR-Cas9 system to induce the synthesis of dopamine (DA) in the brains of a rat model for Parkinson’s disease.

11 Aug 2023

Research breaks ground on new ways of treating blood disorders with gene therapy

Research from experts at Michigan Medicine, the Children's Hospital of Philadelphia and Penn Medicine is breaking ground on new ways of treating blood disorders, such as sickle cell anemia, through gene therapy.

9 Aug 2023

Nationwide Children's Hospital pioneers new gene therapy for Duchenne muscular dystrophy

In a landmark moment for the Abigail Wexner Research Institute at Nationwide Children's, a 5-year-old from Bellefontaine, Ohio, received the first dose of a recently approved gene therapy for Duchenne muscular dystrophy at Nationwide Children's Hospital, where the therapy was invented and initially tested.

8 Aug 2023

Can genome editing transform ocular disease treatment?

The discovery and development of gene-editing technologies and their applications in managing ocular diseases.

3 Aug 2023

RNF41 protein could be a new therapeutic target against cirrhosis and liver inflammation

The RNF41 protein could be a new therapeutic target in the fight against two chronic liver diseases: cirrhosis and liver inflammation.

28 Jul 2023

New gene therapy shows promise in reversing chronic pain

Researchers at NYU College of Dentistry's Pain Research Center have developed a gene therapy that treats chronic pain by indirectly regulating a specific sodium ion channel, according to a new study published in the Proceedings of the National Academy of Sciences.

27 Jul 2023

Penn Medicine researchers identify new therapeutic target for Alzheimer's disease

A gene encoding a protein linked to tau production-;tripartite motif protein 11 (TRIM11)-;was found to suppress deterioration in small animal models of neurodegenerative diseases similar to Alzheimer's disease (AD), while improving cognitive and motor abilities, according to new research from the Perelman School of Medicine at the University of Pennsylvania.

27 Jul 2023

New gene editing approach could revolutionize the treatment of blood disorders

In a step forward in the development of genetic medicines, researchers at Children's Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification of diseased blood cells directly within the body.

27 Jul 2023

NK:IO raises £1.2M, bringing total Seed funding to £5.1M, to advance pioneering NK cell therapy

New investment of £1.2M brings total Seed funding to £5.1M including Innovate UK grants of £1.9M.

From NK:IO Limited 25 Jul 2023

Gene Therapy News and Research

Further Reading

Innovate UK funding to support the development of powdered gene therapy for bone graft augmentation

TherageniX and University of Nottingham awarded £995K Innovate UK grant to develop powdered gene therapy for bone regeneration

New gene therapy offers hope for beta thalassemia patients

Stem cell gene therapy may offer a promising, curative treatment for sickle cell disease

Gene therapy reduces hearing loss associated with Norrie disease in mice

Drexel University receives grants to support cell and gene therapy education

Engineered adeno-associated virus vector yields high transduction of the brain vasculature

Waters launches XBridge Premier GTx BEH SEC columns for gene therapy applications

Gene therapy repairs neural connections in mice with Hurler syndrome

Oncolytic viruses could be used to 'warm up' cold tumors and improve immunotherapy

New gene therapy shows promise for treating fusion-driven cancers

CRISPR/sgRNA-SAM therapy: a potential breakthrough for Parkinson's disease?

Research breaks ground on new ways of treating blood disorders with gene therapy

Nationwide Children's Hospital pioneers new gene therapy for Duchenne muscular dystrophy

Can genome editing transform ocular disease treatment?

RNF41 protein could be a new therapeutic target against cirrhosis and liver inflammation

New gene therapy shows promise in reversing chronic pain

Penn Medicine researchers identify new therapeutic target for Alzheimer's disease

New gene editing approach could revolutionize the treatment of blood disorders

NK:IO raises £1.2M, bringing total Seed funding to £5.1M, to advance pioneering NK cell therapy

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

From Puberty to Menopause: Clue’s CEO, Audrey Tsang on the Power of Femtech

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