U.S., March 4 -- ClinicalTrials.gov registry received information related to the study (NCT07445490) titled 'Translational Potential of ex Vivo Gene Therapy in GM2 Gangliosidosis' on Feb. 25.
Brief Summary: The project aims to optimize and validate this new therapeutic strategy using cells from GM2 patients to evaluate the cross-correction of neurons in vitro by the culture medium of genetically modified myeloid cell lines. The ultimate goal is to demonstrate the potential of CHS-TGEX as an effective treatment in humans for GM2 gangliosidosis.
Study Start Date: May, 2026
Study Type: OBSERVATIONAL
Condition:
Tay-Sachs Disease Ganglioside
Sandhoff Disease Ganglioside
Intervention:
BIOLOGICAL: blood sample
collecting blood sample for v...