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.

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

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.