Delayed delivery of AAV-GDNF prevents nigral neurodegeneration and promotes functional recovery in a rat model of Parkinson's disease. Gene Ther 2002;9:381-389.Wang L, Muramatsu S, Lu Y, et al. 2002. Delayed de
【帕金森病基因疗法获FDA授予再生医学先进疗法认定】北京时间2025年2月20日,拜耳(Bayer)的基因治疗子公司AskBio宣布,用于治疗帕金森病的在研基因疗法AB-1005已获得美国FDA授予的再生医学先进疗法(RMAT)认定。AB-1005是旨在减缓帕金森病情进展并改善患者运动结果的试验性基因疗法。
We first generated three AAV vectors, expressing GDNF, IL-10, or GAD65, which have demonstrated analgesic effects in previous studies, to determine the effects on neuroprotection, neuroinflammation, and neural hyperactivity, respectively. We utilized the TF injection method to administer these alone an...
28 GDNF could also be regulated at the post-transcriptional level, using a fusion with a destabilizing domain.29,30 The herein described highly Dox-sensitive AAV inducible vector combines the advantages of the high Dox-sensitivity of the rtTA-V16 mutant20 and the tightness of the autoregulatory...
山东大学博士学位论文Tet-On/Off系统对AAV介导的TH和GDNF表达调控的研究姓名: 陈腾申请学位级别: 博士专业: 神经外科指导教师: 吴承远; 小泽敬也20030420
(2016) A regulatable AAV vector mediating GDNF biological effects at clinically-approved sub-antimicrobial doxycycline doses. Mol Ther Methods Clin Dev 5:16027.Chtarto, A.; Humbert-Claude, M.; Bockstael, O.; Das, A.T.; Boutry, S.; Breger, L.S.; Klaver, B.; Melas, C.; Barroso-...
AAV mediated GDNF secretion from retinal glia slows down retinal degeneration in a rat model of retinitis pigmentosa. Mol Ther 2011;19:1602-8.Dalkara D, Kolstad KD, Guerin KI, Hoffmann NV, Visel M, et al. (2011) AAV Mediated GDNF Secretion From Retinal Glia Slows Down Retinal Degener- ...
Human neural progenitor cell grafts project neuritic processes along host circuitry concomitantly with AAV-5 GDNF chemotaxis in the MPTP-lesioned primate midbrain. Abstract from American Society for Neural Therapy and Repair. Cell Transplant 2010; 19: 367....
163. Delivery of a Therapeutic Molecule to the Brain for the Treatment of Neurodegenerative Diseases: Production of AAV-5-GDNF by a GMP-Compliant and Scalable Production Platform Results in a Highly Efficient Gene Therapy Vectordoi:10.1016/s1525-0016(16)35967-6None...
Methods A 63-year-old man with advanced PD received AAV2-GDNF in a clinical trial. He died from pneumonia after anterior cervical discectomy and fusion 45months later. An autopsy included brain examination for GDNF transgene expression. Putaminal catecholamine concentrations were compared to in vivo...