AB-1005是一种基于腺相关病毒血清型2载体(AAV2)的研究性基因疗法,含有人胶质细胞源性神经营养因子(AAV2-GDNF)转基因。通过磁共振成像(MRI)监测的对流增强给药方式直接进行神经外科注射后,GDNF可以在大脑局部区域实现稳定和连续的表达。在非临床研究中,重组人GDNF能够促进多巴胺能神经元的存活和形态分化。 根据新闻稿...
Delayed delivery of AAV-GDNF prevents nigral neurodegeneration and promotes functional recovery in a rat model of Parkinson’s disease. Gene Ther 2002; 9(6): 381–9Wang L, Muramatsu S, Lu Y, Ikeguchi K, Fujimoto K, Okada T, Mizukami H, Hanazono Y, Kume A, Urano F, Ichinose H, ...
基因疗法获FDA授予再生医学先进疗法认定】2月19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。AB-1005是一种基于腺相关病毒血清型2载体(AAV2)的研究性基因疗法...
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...
(MSA-P).1This marks a significant milestone in the development of AB-1005 gene therapy, an adeno-associated viral vector encoding for glial cell line-derived neurotrophic factor (AAV2-GDNF) that is delivered to the...
We describe a highly-sensitive doxycycline-inducible adeno-associated virus (AAV) vector. This vector allowed for the first time a longitudinal analysis of inducible transgene expression in the brain using bioluminescence imaging. To evaluate the dose range of GDNF biological activity, the inducible ...
神经营养因子是一类能够促进神经细胞存活,生长和分化的蛋白质或多肽,在神经系统的发育,生长和损伤后修复等过程中发挥重要作用.它们通过与相应的受体或受体复合物结合进行信号转导,发挥其生物学效应.胶质细胞源性神经营养因子(Glial cell line-derived neurotrophic factor,GDNF)是1993年Lin等首先在大鼠胶质细胞系B49......
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
RedmondJr.,D.E.,Mcentire,etc.Comparison of fetal mesencephalic grafts, AAV-delivered GDNF, and both combined in an MPTP-induced nonhuman primate Parkinson's model[J].2013,12(12).Redmond, D. E., McEntire, C. R., Kingsbery, J. P., Leranth, C., Elsworth, J. D., Bjugstad, K. B....