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.

Study provides data to support gene supplementation as a treatment for autosomal dominant retinitis pigmentosa

A new study shows that total RPE65 protein levels in mice with autosomal dominant retinitis pigmentosa were doubled following subretinal delivery of adeno-associated virus (AAV)-RPE65 gene supplementation.

23 May 2023

MGH researchers identify a potential method for treating fragile X syndrome

New research has identified a potential method for treating fragile X syndrome, a leading cause of autism spectrum disorders that is characterized by an inherited repeat of certain nucleotides within the DNA sequence of the FMR1 gene.

19 May 2023

New technique could mechanically enhance glymphatic transport in the mouse brain

Like the lymphatic system in the body, the glymphatic system in the brain clears metabolic waste and distributes nutrients and other important compounds.

16 May 2023

Nemours Children's Health selected to conduct first gene therapy clinical trial for Morquio A syndrome

As part of the Foundation for the National Institutes of Health Accelerating Medicines Partnership Bespoke Gene Therapy Consortium, Nemours Children's Health has been chosen to conduct a first-of-its-kind gene therapy clinical trial for Morquio A syndrome.

16 May 2023

Phase 1 clinical trial of CAR-NKT cell therapy shows promising results in neuroblastoma patients

Researchers at Baylor College of Medicine/Texas Children's Cancer Center and collaborating institutions report interim results from a first-in-human phase 1 clinical trial evaluating the safety, antitumor activity and immunological characteristics of a genetically engineered natural killer T (NKT) cell immunotherapy for neuroblastoma, a childhood tumor that most commonly arises in the adrenal gland.

15 May 2023

Patient-derived head and neck cancer organoids could aid the discovery of novel therapies

Researchers from the Organoid group (Hubrecht Institute) and UMC Utrecht have developed a biobank with organoids derived from patients with head and neck cancer (HNC).

12 May 2023

Cellular Origins partners with ScaleReady to simplify, standardise, and automate cell therapy manufacturing

Cellular Origins a TTP Company focused on enabling scalable, cost-effective, and efficient manufacture of cell and gene therapies (CGTs).

From Cellular Origins 11 May 2023

Menthol inhalation improves cognitive ability in animal models of Alzheimer's disease

Researchers from Cima University of Navarra (Spain) have shown in animal models of Alzheimer's disease that inhaling menthol improves cognitive ability.

10 May 2023

Researchers evaluate the dual-therapeutic effect of gene therapy in mouse model of osteosarcoma

Investigators at Brigham and Women’s Hospital and collaborating institutions evaluated the dual-therapeutic effect of gene therapy in a clinically relevant model for common form of bone cancer.

8 May 2023

First CRISPR-armed phage therapeutic can specifically target and remove E. coli in the human gut

SNIPR Biome ApS ("SNIPR"), the company pioneering CRISPR-based microbial gene therapy, announces today that Nature Biotechnology has published research findings from its preclinical work on SNIPR001, the first CRISPR-armed phage therapeutic developed to specifically target and remove E. coli, including antibiotic-resistant strains, in the human gastrointestinal tract.

8 May 2023

Finnish mitochondrial disease of newborns shows cancer-like changes in proliferating cells

A study found that a mitochondrial disease of newborns shows cancer-like changes in proliferating cells, causing tissues to age prematurely.

3 May 2023

New technology can deliver gene therapy directly to the lung

Scientists at UMass Chan Medical School have developed a technology to deliver gene therapy directly to lung tissue through intranasal administration, a development that could potentially create a new class of treatments for lung disease.

2 May 2023

New approach for efficient engineering of cell and gene therapies

A new approach to the genetic engineering of cells promises significant improvements in speed, efficiency, and reduction in cellular toxicity compared to current methods.

28 Apr 2023

Research develops unique delivery system to treat osteosarcoma

New research has identified a potential therapeutic target and developed a unique delivery system to treat osteosarcoma, a bone cancer that primarily affects children and adolescents.

27 Apr 2023

Researchers take key step toward developing a gene therapy for Usher syndrome type 1F

Usher Syndrome type 1F is a rare but severe genetic disease that causes deafness, lack of balance, and progressive blindness.

26 Apr 2023

The Wonder of DNA: Reflections from Dr. Francis Collins on the 20th Anniversary of the Human Genome Project

To highlight how far the field of genetics has come, we spoke with Dr. Francis Collins, the leader of the Human Genome Project about his incredible career in genetics.

25 Apr 2023

Using engineered stem cells to treat ALS and retinitis pigmentosa

Cedars-Sinai investigators are developing a novel way to treat amyotrophic lateral sclerosis (ALS) and retinitis pigmentosa using engineered stem cells that may eventually lead to personalized treatments.

20 Apr 2023

Gene therapy approach can decrease intraocular pressure in pre-clinical models of glaucoma

Scientists at Trinity College Dublin today announced a significant development towards a new therapeutic treatment of glaucoma.

19 Apr 2023

Researchers develop novel molecular nanocages for siRNA delivery

Small interfering RNAs (siRNAs) are novel therapeutics that can be used to treat a wide range of diseases.