Scientists design gene delivery systems for cells in the brain and spinal cord

Researchers contribute to new toolkit for battling brain disorders

New studies stemming from the Armamentarium consortium outline findings that advance tools based on Adeno-associated virus (AAV) vectors. An announcement about the work explains how an AAV "acts like a shuttle capable of transporting specially designed DNA into the cell."

Scientists design gene delivery systems for cells in the brain and spinal cord

Research teams 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. These delivery systems are a significant step toward future precise gene therapy for the brain that could safely control errant brain activity with high precision. In contrast, current therapies for brain disorders mostly treat only symptoms.

Researchers Develop a New Way to Deliver Gene Therapies to the Brain

Scientists working at the NIH’s BRAIN Initiative have created new viral vectors that one day could lead to gene therapies for ALS, seizure disorders, Parkinson’s and Alzheimer’s disease, Huntington’s disease and other diseases of the brain.

Parallel Reporter and Transgenic Assays Reveal Neuronal Enhancers

In an era where understanding the genomic regulation of the brain’s intricate circuitry is paramount, researchers are pushing the boundaries of technology to unravel the mysteries of neuronal enhancer elements—key regulatory DNA sequences that dictate when and where genes are expressed in neurons. A landmark study recently published in Nature Communications by Kosicki, Laboy Cintrón, Keukeleire, and colleagues has pioneered a multifaceted approa…