Issues regarding gene therapy products for Parkinson's disease: The development of CERE-120 (AAV-NTN) as one reference point

被引：25

作者：

Bartus, Raymond T. ^{[1
]}

Herzog, Christopher D. ^{[1
]}

Bishop, Kathie ^{[1
]}

Ostrove, Jeffrey M. ^{[1
]}

Tuszynski, Mark ^{[2
]}

Kordower, Jeffrey H. ^{[3
]}

Gasmi, Mehdi ^{[1
]}

机构：

[1] Ceregene Inc, San Diego, CA 92121 USA

[2] UCSD, Sch Med, La Jolla, CA USA

[3] Rush Univ, Med Ctr, Chicago, IL 60612 USA

来源：

PARKINSONISM & RELATED DISORDERS | 2007年 / 13卷

关键词：

neurotrophic factors; disease progression; neurturin; neuronal restoration; neuroprotection; NEUROTROPHIC FACTOR GDNF; NERVE GROWTH-FACTOR; NIGROSTRIATAL SYSTEM; RHESUS-MONKEYS; INTRACEREBROVENTRICULAR INFUSION; INTRAPUTAMENAL INFUSION; STRIATAL DELIVERY; HUMAN NEURTURIN; VECTOR; BRAIN;

D O I：

10.1016/S1353-8020(08)70052-X

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

Purpose: To develop CERE-120 (AAV-NTN) as a novel therapy for Parkinson's disease (PD) that might restore function of degenerating dopamine neurons and prevent further degeneration. Scope: A nonclinical program demonstrated that NTN expression can be predictably controlled following CERE-120 administration, provides clear evidence of efficacy in numerous animal models and is safe at dose multiples that far exceed those required for efficacy. Preliminary, open label evidence in PD Subjects offers corroborative support for these observations. Conclusions: CERE-120 may represent an important, novel therapy for PD, though the clinical data require confirmation with additional clinical tests, including an ongoing multi-center, double-blincled controlled trial. (C) 2007 Elsevier B. V. All rights reserved.

引用

页码：S469 / S477

页数：9

共 37 条

[1]

ARVANITAKIS Z, 2007, AM AC NEUR ANN M BOS

[2]

Bartus R T, 1999, Curr Opin Drug Discov Devel, V2, P152

[3] NEUROTROPHIC FACTORS - CAN THE DEGENERATING BRAIN BE INDUCED TO HEAL ITSELF [J].