Stem cell technology for the study and treatment of motor neuron diseases

被引：52
作者：
Lunn, J. Simon [1 ]
Sakowski, Stacey A. [1 ]
Federici, Thais [2 ]
Glass, Jonathan D. [3 ]
Boulis, Nicholas M. [2 ]
Feldman, Eva L. [1 ]
机构：
[1] Univ Michigan, Dept Neurol, Ann Arbor, MI 48109 USA
[2] Emory Univ, Dept Neurosurg, Atlanta, GA 30322 USA
[3] Emory Univ, Dept Neurol, Atlanta, GA 30322 USA
来源：
REGENERATIVE MEDICINE | 2011年 / 6卷 / 02期
关键词：
amyotrophic lateral sclerosis; induced pluripotent stem cells; motor neuron disease; Phase I clinical trial; spinal muscular atrophy; stem cells; translational medicine; AMYOTROPHIC-LATERAL-SCLEROSIS; SPINAL MUSCULAR-ATROPHY; ENDOTHELIAL GROWTH-FACTOR; PLACEBO-CONTROLLED TRIAL; BETA-LACTAM ANTIBIOTICS; CORD BLOOD-CELLS; RAT MODEL; MOUSE MODEL; FACTOR-I; EXTENDS SURVIVAL;
D O I：
10.2217/RME.11.6
中图分类号：
Q813 [细胞工程];
学科分类号：
摘要：
Amyotrophic lateral sclerosis and spinal muscular atrophy are devastating neurodegenerative diseases that lead to the specific loss of motor neurons. Recently, stem cell technologies have been developed for the investigation and treatment of both diseases. Here we discuss the different stem cells currently being studied for mechanistic discovery and therapeutic development, including embryonic, adult and induced pluripotent stem cells. We also present supporting evidence for the utilization of stem cell technology in the treatment of amyotrophic lateral sclerosis and spinal muscular atrophy, and describe key issues that must be considered for the transition of stem cell therapies for motor neuron diseases from bench to bedside. Finally, we discuss the first-in-human Phase I trial currently underway examining the safety and feasibility of intraspinal stem cell injections in amyotrophic lateral sclerosis patients as a foundation for translating stem cell therapies for various neurological diseases.
引用
收藏
页码：201 / 213
页数：13
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