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Gene and Cell Therapy – News and Features

DNA double helix.

Potential Method for Treating Fragile X Syndrome Identified

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.
A vessel in the glymphatic system lit in red, green and yellow, with smaller branched vessels shown in blue.

Ultrasound Improves the Brain's Waste Clearance System

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.
An operating room.

"Smart" Sutures Could Aid Drug Delivery and Surgical Healing

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.
Computer-generated 3D render of some cancer cells.

Small Trial of Cancer-Killing Virus With Immunotherapy Shows Positive Results

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.
A small brown mouse.

First Humanised Mouse Model Created for Congenital Adrenal Hyperplasia

Mice with a defective human gene responsible for congenital adrenal hyperplasia have been developed for the first time.
A picture of a DNA helix.

Interstrand Cross Links Improve Non-Viral Gene Editing

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.
Six 3D render images showing how different organelles are distributed throughout a cell, and how this is different in young and old cells.

Intracellular Toolkit Identifies Cells Most Likely To Create Effective Cell Therapies

An intracellular toolkit that maps organelle geography could be used to identify cells that will make effective cell therapies.
The muscular back of a woman.

Scarring of Collagen “Highway” Halts Healing Power of Stem Cells

Study in mice shows scarring of collagen "highway" prevents stem cells from healing damaged tissue in Duchenne muscular dystrophy.
A doctor looks at an X-Ray of a torso.

Blood Test May Predict Responses to Chemoimmunotherapy

A newly identified biomarker can indicate which patients with non-small cell lung cancer will respond well to chemoimmunotherapy
Inhaling Menthol Improves Cognition in Alzheimer's Model Mice content piece image

Inhaling Menthol Improves Cognition in Alzheimer's Model Mice

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.