Long-term follow-up after gene therapy for canavan disease P Leone, D Shera, SWJ McPhee, JS Francis, EH Kolodny, LT Bilaniuk, ... Science translational medicine 4 (165), 165ra163-165ra163, 2012 | 293 | 2012 |
Gene Therapy of Canavan Disease: AAV-2 Vector for Neurosurgical Delivery of Aspartoacylase Gene (ASPA) to the Human Brain C Janson, S McPhee, L Bilaniuk, J Haselgrove, M Testaiuti, A Freese, ... Human gene therapy 13 (11), 1391-1412, 2002 | 293 | 2002 |
Immune responses to AAV in a phase I study for Canavan disease SWJ McPhee, CG Janson, C Li, RJ Samulski, AS Camp, J Francis, ... The Journal of Gene Medicine: A cross‐disciplinary journal for research on …, 2006 | 284 | 2006 |
Quantitative comparison of expression with adeno-associated virus (AAV-2) brain-specific gene cassettes R Xu, CG Janson, M Mastakov, P Lawlor, D Young, A Mouravlev, ... Gene therapy 8 (17), 1323-1332, 2001 | 234 | 2001 |
Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease P Leone, CG Janson, L Bilianuk, Z Wang, F Sorgi, L Huang, R Matalon, ... Annals of neurology 48 (1), 27-38, 2000 | 203 | 2000 |
Simultaneous high performance liquid chromatographic separation of purines, pyrimidines, N-acetylated amino acids, and dicarboxylic acids for the chemical diagnosis of inborn … B Tavazzi, G Lazzarino, P Leone, AM Amorini, F Bellia, CG Janson, ... Clinical biochemistry 38 (11), 997-1008, 2005 | 130 | 2005 |
Human intrathecal transplantation of peripheral blood stem cells in amyotrophic lateral sclerosis CG Janson, TM Ramesh, MJ During, P Leone, J Heywood Journal of hematotherapy & stem cell research 10 (6), 913-915, 2001 | 115 | 2001 |
Morpholinos and PNAs compared CG Janson, MJ During, JE Summerton Peptide nucleic acids, morpholinos and related antisense biomolecules, 89-113, 2006 | 93 | 2006 |
Natural History of Canavan Disease Revealed by Proton Magnetic Resonance Spectroscopy (1H‐MRS) and Diffusion-weighted MRI CG Janson, SWJ McPhee, J Francis, D Shera, M Assadi, A Freese, ... Neuropediatrics 37 (04), 209-221, 2006 | 86 | 2006 |
Lithium citrate reduces excessive intra-cerebral N-acetyl aspartate in Canavan disease M Assadi, C Janson, DJ Wang, O Goldfarb, N Suri, L Bilaniuk, P Leone european journal of paediatric neurology 14 (4), 354-359, 2010 | 65 | 2010 |
Viral-based gene transfer to the mammalian CNS for functional genomic studies CG Janson, SWJ McPhee, P Leone, A Freese, MJ During Trends in neurosciences 24 (12), 706-712, 2001 | 61 | 2001 |
Improvement in clinical outcomes following optimal targeting of brain ventricular catheters with intraoperative imaging CG Janson, LG Romanova, KD Rudser, SJ Haines Journal of neurosurgery 120 (3), 684-696, 2014 | 55 | 2014 |
Effects of AAV-2-mediated aspartoacylase gene transfer in the tremor rat model of Canavan disease SWJ McPhee, J Francis, CG Janson, T Serikawa, K Hyland, EO Ong, ... Molecular brain research 135 (1-2), 112-121, 2005 | 54 | 2005 |
Mild‐onset presentation of Canavan's disease associated with novel G212A point mutation in aspartoacylase gene CG Janson, EH Kolodny, BJ Zeng, S Raghavan, G Pastores, P Torres, ... Annals of Neurology: Official Journal of the American Neurological …, 2006 | 53 | 2006 |
Treatment of spinocerebellar ataxia with buspirone M Assadi, JV Campellone, CG Janson, JJ Veloski, RJ Schwartzman, ... Journal of the neurological sciences 260 (1-2), 143-146, 2007 | 50 | 2007 |
Global CNS gene transfer for a childhood neurogenetic enzyme deficiency: Canavan disease. P Leone, CG Janson, SJ McPhee, MJ During | 50 | 1999 |
Lithium citrate for Canavan disease CG Janson, M Assadi, J Francis, L Bilaniuk, D Shera, P Leone Pediatric neurology 33 (4), 235-243, 2005 | 45 | 2005 |
Peptide nucleic acids, morpholinos and related antisense biomolecules CG Janson, MJ During Springer US, 2006 | 39 | 2006 |
N-acetylaspartate supports the energetic demands of developmental myelination via oligodendroglial aspartoacylase JS Francis, I Wojtas, V Markov, SJ Gray, TJ McCown, RJ Samulski, ... Neurobiology of disease 96, 323-334, 2016 | 34 | 2016 |
Functional regulatory regions of human transcription factor MEF2C CG Janson, Y Chen, Y Li, D Leifer Molecular brain research 97 (1), 70-82, 2001 | 33 | 2001 |