AI-Powered Gene Vectors Offer Cell-by-Cell Access to Brain and Spinal Cord

New gene delivery vehicle shows promise for human brain gene therapy

In an important step toward more effective gene therapies for brain diseases, researchers have engineered a gene-delivery vehicle that uses a human protein to efficiently cross the blood-brain barrier and deliver a disease-relevant gene to the brain in mice expressing the human protein. Because the vehicle binds to a well-studied protein in the blood-brain barrier, the scientists say it has a good chance at working in patients.

New gene delivery systems can reach cells in the brain and spinal cord with exceptional accuracy

Research teams funded by the National Institutes of Health (NIH) have created a versatile set of gene delivery systems that can reach different neural cell types in the human brain and spinal cord with exceptional accuracy.

The Armamentarium: Scientists create next generation of tools in battle against brain disease

In a scientific first, researchers from around 29 universities and institutions across North America have teamed up to create a large, versatile, and effective arsenal of new biological tools that will play a critical role in the battle against brain disease.

A Suite of Enhancer AAVs Support Targeted Gene Therapy for Brain Disease

The mammalian cortex, the region of the brain responsible for a wide range of higher-level cognitive functions, including sensory perception, motor control, and complex thought, is composed of a diverse array of cell types. Defining the contribution of each cell type to cortex function is an ongoing challenge for understanding brain health and disease.  In a new study published in Cell titled “A suite of enhancer AAVs and transgenic mouse lines …

AI-Guided Gene Vectors Precisely Target Brain and Spinal Cells

Scientists have engineered dozens of adeno-associated virus (AAV) systems that ferry genes to specific neuron and glial subtypes in the brain and spinal cord with unprecedented accuracy. Powered by AI-selected DNA “light switches,” the vectors can switch on therapeutic or research genes only in targeted cells—eliminating the need for transgenic animals and enabling fine-grained circuit mapping, activation, or silencing.

Scientists create new toolkit to fight brain diseases

Scientists say they’ve put together a new kind of molecular toolkit that could eventually be used to treat a variety of brain diseases, possibly including epilepsy, sleep disorders and Huntington’s disease. The kit currently contains more than 1,000 tools of a type known as enhancer AAV vectors, with AAV standing for “adeno-associated virus.” A consortium that included researchers from Seattle’s Allen Institute for Brain Science and the Universi…