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.

Multipronged gene therapy restores walking in mice after complete spinal cord injury

When the spinal cords of mice and humans are partially damaged, the initial paralysis is followed by the extensive, spontaneous recovery of motor function.

21 Sep 2023

Scientists uncover a crucial component for restoring functional activity after spinal cord injury

In a new study in mice, a team of researchers from UCLA, the Swiss Federal Institute of Technology, and Harvard University have uncovered a crucial component for restoring functional activity after spinal cord injury.

21 Sep 2023

Ongoing gene therapy trials have driven increased use of ALS genetic testing, finds study

Researchers at The Ohio State University Wexner Medical Center and College of Medicine led the creation of evidence-based consensus guidelines for genetic testing and counseling for patients with amyotrophic lateral sclerosis (ALS), a neurodegenerative disease that affects the cells in the brain and spine.

19 Sep 2023

New research offers insight about the reason mitochondrial DNA is maternally inherited

New research provides insight about the bedrock scientific principle that mitochondrial DNA -; the distinct genetic code embedded in the organelle that serves as the powerplant of every cell in the body -; is exclusively passed down by the mother.

19 Sep 2023

Researchers identify key mechanisms to address future treatments for hereditary blindness

There are still many enigmas about the mechanism of action of the CERKL gene, which causes retinitis pigmentosa and other hereditary vision diseases.

18 Sep 2023

Broken String Biosciences closes $15M series A funding round

Broken String Biosciences today announced that it has closed a $15 million Series A investment round.

From Broken String Biosciences 18 Sep 2023

AACR Cancer Progress Report provides latest statistics on cancer research and treatment

Today, the American Association for Cancer Research (AACR) released the 13th edition of its annual Cancer Progress Report, which chronicles how basic, translational, and clinical cancer research and cancer-related population sciences-;primarily supported by federal investments in the National Institutes of Health (NIH) and the National Cancer Institute (NCI)-;remain vitally important to improving health and saving lives.

13 Sep 2023

Dormant HIV reservoirs can spontaneously produce RNA and proteins, study finds

HIV anti-retroviral therapy is considered a treatment and not a cure because patients usually carry a reservoir of HIV-infected cells that can re-emerge if treatment stops.

13 Sep 2023

Is DNA methylation the missing link in diagnosing and treating psoriasis and psoriatic arthritis?

Researchers across Europe have delved into the DNA methylation patterns in CD4+ T-cells of psoriasis and psoriatic arthritis (PsA) patients, unveiling potential new biomarkers for these conditions. Their study suggests that these unique profiles could serve as both diagnostic and prognostic tools, although further validation is needed for real-world clinical application.

10 Sep 2023

Ohio State University Wexner Medical Center administers first targeted gene therapy for ALS

The Ohio State University Wexner Medical Center is one of the first nationwide to administer a targeted gene therapy for patients with a specific form of amyotrophic lateral sclerosis (ALS), a neurodegenerative disease that affects the cells in the brain and spine.

8 Sep 2023

Newly discovered nanobody may lead to a treatment for Retinitis Pigmentosa

A team of scientists from the University of California, Irvine, believe they have discovered a special antibody which may lead to a treatment for Retinitis Pigmentosa, a condition that causes loss of central vision, as well as night and color vision.

7 Sep 2023

Study identifies potential target for reversing drug resistance in ovarian cancer

For the 314,000 people diagnosed with ovarian cancer each year, hope often comes in the form of platinum-based drugs such as cisplatin.

5 Sep 2023

Big Tuna and automated buffer exchange: An interview with Unchained Labs

In this interview, AZoNetwork speaks with Joe Barco, the Senior Director of Workflow Marketing at Unchained Labs, about the benefits of automated buffer exchange with Big Tuna.

From Unchained Labs 4 Sep 2023

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