U.S., Sept. 16 -- ClinicalTrials.gov registry received information related to the study (NCT07173153) titled 'Gene Therapy for SLC6A1 Neurodevelopmental Disorder' on Sept. 08.
Brief Summary: This is gene therapy study of an AAV9 vector carrying the SLCA1 gene for SLC6A1 neurodevelopmental disorder.
Study Start Date: Aug. 25
Study Type: INTERVENTIONAL
Condition:
SLC6A1
Intervention:
BIOLOGICAL: AAV9.SLC6A1 Gene Therapy
This is an open-label, single injection study of an AAV9 vector carrying the SLCA1 coding sequence delivered one time through an intrathecal injection.
Recruitment Status: ENROLLING_BY_INVITATION
Sponsor: Emily de los Reyes
Information provided by (Responsible Party): Emily de los Reyes, Nationwide Children's Hospit...