“…The more recent demonstration of rAAV9 trans-BBB neurotropism has offered an effective solution for CNS gene delivery,21, 22 leading to successes in developing approaches for the treatment of neurological diseases, including MPS II, in animal models via systemic23, 24, 25, 26, 27, 28, 29, 30 or IT delivery 27, 31, 32, 33, 34, 35, 36, 37, 38. These studies have led to ongoing clinical trials of systemic rAAV9 gene delivery in patients with spinal muscular atrophy (SMA) (ClinicalTrials.gov: NCT02122952), MPS IIIA (ClinicalTrials.gov: NCT02716246), and MPS IIIB (ClinicalTrials.gov: NCT03315182), and IT gene delivery for giant axonal neuropathy (NCT02362438), MPS I (RGX-111, RegenxBio), and MPS II (RGX121, RegenxBio).…”