U.S., March 7 -- ClinicalTrials.gov registry received information related to the study (NCT06860672) titled 'Clinical Trial of the Dual Vector Base Editor for the Treatment of the CHD3-R1025W Mutation' on Feb. 24.
Brief Summary: To evaluate the safety, tolerability and preliminary efficacy study of a single intrathecal injection of the dual vector AAV-CHD3-R1025W base editor for the treatment of developmental disorders caused by the R1025W mutation in the CHD3 gene
Study Start Date: Feb. 19
Study Type: INTERVENTIONAL
Condition:
Developmental Delay Disorder
Intellectual Disability
Rare Diseases
Intervention:
GENETIC: Dual vector DNA base editor
The base editor is delivered using a dual vector adeno-associated virus (AAV) system and i...