Due to its feasibility and reduced invasiveness, IT delivery was chosen as the route of administration in a phase I/II safety study where an AAV9-based vector expressing the CLN6 gene was administered via lumbar puncture to patients with CLN6 type late infantile NCL (NCT02725580), following positive results in NHPs experimentation [250]. Here, CLN6 is linked to neuronal ceroid lipofuscinosis.