共 50 条
- [11] A Novel Mutation in Aspartoacylase Gene; Canavan Disease[J]. IRANIAN JOURNAL OF CHILD NEUROLOGY, 2015, 9 (04) : 54 - 57Ashrafi, MahmoodrezaTavasoli, AlirezaKatibeh, PegahAryani, OmidVafaee-Shahi, Mohammad
- [12] Long-Term Follow-Up After Gene Therapy for Canavan Disease[J]. SCIENCE TRANSLATIONAL MEDICINE, 2012, 4 (165)Leone, PaolaShera, DavidMcPhee, Scott W. J.Francis, Jeremy S.Kolodny, Edwin H.Bilaniuk, Larissa T.Wang, Dah-JyuuAssadi, MitraGoldfarb, OlgaGoldman, H. WarrenFreese, AndrewYoung, DeborahDuring, Matthew J.Samulski, R. JudeJanson, Christopher G.
- [13] From Gene Therapy to Gene Enhancement: Optimized Pre-Clinical Gene Therapy Transforms Mice with the Severest Canavan Disease Phenotype Into "Supermice"[J]. MOLECULAR THERAPY, 2015, 23 : S198 - S198Gessler, Dominic J.Li, JiaSu, QinMatalon, ReubenGao, Guangping
- [14] CNS Gene Therapy Spatially and Temporally Modulates Transcriptional Expression of Neurons and Oligodendrocytes in Canavan Disease[J]. MOLECULAR THERAPY, 2023, 31 (04) : 345 - 345Lotun, AnoushkaMooney, MorganGao, GuangpingGessler, Dominic J.
- [15] A novel Aspartoacylase (ASPA) Gene Mutation in Canavan Disease[J]. FETAL AND PEDIATRIC PATHOLOGY, 2012, 31 (04) : 236 - 239Durmaz, Asude AlpmanAkin, HalukOnay, HuseyinVahabi, AliOzkinay, Ferda
- [16] Uncoupling N-acetylaspartate from brain pathology: implications for Canavan disease gene therapy[J]. Acta Neuropathologica, 2018, 135 : 95 - 113Georg von JonquieresZiggy H. T. SpencerBenjamin D. RowlandsClaudia B. KlugmannAndre BongersAnne E. HarastaKristina E. ParleyJennie CederholmOrla TeahanRussell PickfordFabien DelerueLars M. IttnerDominik FröhlichCatriona A. McLeanAnthony S. DonMiriam SchneiderGary D. HousleyCaroline D. RaeMatthias Klugmann
- [17] Uncoupling N-acetylaspartate from brain pathology: implications for Canavan disease gene therapy[J]. ACTA NEUROPATHOLOGICA, 2018, 135 (01) : 95 - 113von Jonquieres, GeorgSpencer, Ziggy H. T.Rowlands, Benjamin D.Klugmann, Claudia B.Bongers, AndreHarasta, Anne E.Parley, Kristina E.Cederholm, JennieTeahan, OrlaPickford, RussellDelerue, FabienIttner, Lars M.Frohlich, DominikMcLean, Catriona A.Don, Anthony S.Schneider, MiriamHousley, Gary D.Rae, Caroline D.Klugmann, Matthias
- [18] A MUTATION OF ASPARTOACYLASE GENE IN A TURKISH PATIENT WITH CANAVAN DISEASE[J]. GENETIC COUNSELING, 2012, 23 (01): : 9 - 12Gungor, H. EkeIscan, A.Cece, H.Calik, M.
- [19] Gene therapy targets sickle-cell disease[J]. NATURE, 2018, 564 (7735) : S12 - S13Nowogrodzki, Anna