Gene and Cell Therapy – News and Features

Researchers have discovered that stimulating cells’ DNA repair mechanisms may correct the inherited genetic defect that defines fragile X syndrome, a leading cause of autism spectrum disorders.

The glymphatic system in the brain works to clear metabolic waste and distribute nutrients. Researchers have found that ultrasound can be used to improve the system, which could be used to study brain diseases.

MIT engineers have developed “smart” sutures with the potential to benefit Crohn’s disease patients undergoing surgery, and could also have applications for sensing inflammation, drug delivery and cell therapies.

Results of a Phase 1/2 clinical trial suggest that oncolytic viral therapy and immunotherapy were safe and improved survival in a subset of patients with recurrent glioblastoma.

Mice with a defective human gene responsible for congenital adrenal hyperplasia have been developed for the first time.

University of California (UC) Santa Barbara researchers have added to the ever-growing genome-editing toolbox. In Nature Biotechnology, they publish a method that improves CRISPR/Cas9 editing efficiency without using viral material.

An intracellular toolkit that maps organelle geography could be used to identify cells that will make effective cell therapies.

Study in mice shows scarring of collagen "highway" prevents stem cells from healing damaged tissue in Duchenne muscular dystrophy.

A newly identified biomarker can indicate which patients with non-small cell lung cancer will respond well to chemoimmunotherapy

Researchers show that smelling menthol modulates the immune system and improves cognition in animals with this neurodegenerative disease. This study opens the door to developing new therapies to prevent or alleviate the effects of Alzheimer's and other diseases of the central nervous system.