Perinatal systemic gene delivery using adeno-associated viral vectors

• Main Authors: Karda, R, Buckley, S.M.K, Mattar, C.N, Ng, J, Massaro, G, Hughes, M.P, Kurian, M.A, Baruteau, J, Gissen, P, Chan, J.K.Y, Bacchelli, C, Waddington, S.N, Rahim, A.A
• Other Authors: OBSTETRICS & GYNAECOLOGY
• Format: Review
• Published: 2020
• Subjects: parvovirus vector; survival motor neuron protein; blood brain barrier; central nervous system; degenerative disease; disease model; Down syndrome; gene; gene delivery system; gene expression; gene targeting; genetic transduction; human; immune response; monogenic disorder; next generation sequencing; Niemann Pick disease; nonhuman; perinatal period; phase 1 clinical trial (topic); prenatal screening; primate; Review; SMN gene; spinal muscular atrophy; systemic therapy
• Online Access: https://scholarbank.nus.edu.sg/handle/10635/181479
• Institution: National University of Singapore

10.3389/fnmol.2014.00089