Transpher A, a multicenter, single-dose, phase 1/2 clinical trial of ABO-102, an intravenous AAV9-based gene therapy for Sanfilippo syndrome type A …

KM Flanigan, NJC Smith, ML Couce, KV Truxal… - 2020 - AAN Enterprises
Objective: To develop a gene therapy treatment for Mucopolysaccharidosis IIIA (MPS-IIIA)
based on intravenous administration of ABO-102. Background: MPS-IIIA is a lysosomal …
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Transpher A, a multicenter, single-dose, phase 1/2 clinical trial of ABO-102, an intravenous AAV9-based gene therapy for Sanfilippo Syndrome Type A …

KM Flanigan, NJC Smith, ML Couce, KV Truxal… - Neurology, 2020 - AAN Enterprises
Objective: To develop a gene therapy treatment for Mucopolysaccharidosis IIIA (MPS-IIIA)
based on intravenous administration of ABO-102. Background: MPS-IIIA is a lysosomal …