A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain disorders known as SYNGAP1-related disorders, or SRDs. Researchers were able ...
In a major step toward treating rare neurological diseases, scientists have used gene editing to correct mutations inside the brain of living mice with a single injection. This new approach, described ...
Corresponding author Akshay Sharma, MBBS, MSc, St. Jude Department of Bone Marrow Transplantation and Cellular Therapy talks to St. Jude patient enrolled on the reported clinical trial, Corey Luellen.
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 ...
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