Arbor Biotechnologies is exploring an investigational Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) approach to treating Angelman syndrome.
A therapeutic approach with two parts: one that recognizes a specific section of DNA or RNA and one that uses an enzyme (CAS) to cut at a specific section in the DNA or RNA. A CRISPR could be designed to cut the UBE3A-ATS and potentially allow the paternal UBE3A gene to be turned on.
Arbor Biotechnologies to Present Data on CNS Programs and Platform Advances at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting
