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
相关论文
共 120 条
  • [1] Comparison of the growth hormone, IGF-1 and insulin in cerebrospinal fluid and serum between patients with motor neuron disease and healthy controls
    Bilic, E.
    Bilic, E.
    Rudan, I.
    Kusec, V.
    Zurak, N.
    Delimar, D.
    Zagar, M.
    [J]. EUROPEAN JOURNAL OF NEUROLOGY, 2006, 13 (12) : 1340 - 1345
  • [2] A placebo-controlled trial of insulin-like growth factor-I in amyotrophic lateral sclerosis
    Borasio, GD
    Robberecht, W
    Leigh, PN
    Emile, J
    Guiloff, RJ
    Jerusalem, F
    Silani, V
    Vos, PE
    Wokke, JHJ
    Dobbins, T
    [J]. NEUROLOGY, 1998, 51 (02) : 583 - 586
  • [3] Adult mesenchymal stem cells for tissue engineering versus regenerative medicine
    Caplan, Arnold I.
    [J]. JOURNAL OF CELLULAR PHYSIOLOGY, 2007, 213 (02) : 341 - 347
  • [4] Chen Lin, 2007, Zhongguo Xiu Fu Chong Jian Wai Ke Za Zhi, V21, P961
  • [5] Motor neuron degeneration promotes neural progenitor cell proliferation, migration, and neurogenesis in the spinal cords of amyotrophic lateral sclerosis mice
    Chi, LY
    Ke, Y
    Luo, C
    Li, BL
    Gozal, D
    Kalyanaraman, B
    Liu, R
    [J]. STEM CELLS, 2006, 24 (01) : 34 - 43
  • [6] Functional recovery in rats with ischemic paraplegia after spinal grafting of human spinal stem cells
    Cizkova, D.
    Kakinohana, O.
    Kucharova, K.
    Marsala, S.
    Johe, K.
    Hazel, T.
    Hefferan, M. P.
    Marsala, M.
    [J]. NEUROSCIENCE, 2007, 147 (02) : 546 - 560
  • [7] Restoration of SMN function:: Delivery of a trans-splicing RNA re-directs SMN2 pre-mRNA splicing
    Coady, Tristan H.
    Shababi, Monir
    Tullis, Gregory E.
    Lorson, Christian L.
    [J]. MOLECULAR THERAPY, 2007, 15 (08) : 1471 - 1478
  • [8] Wild-type bone marrow cells ameliorate the phenotype of SOD1-G93A ALS mice and contribute to CNS, heart and skeletal muscle tissues
    Corti, S
    Locatelli, F
    Donadoni, C
    Guglieri, M
    Papadimitriou, D
    Strazzer, S
    Del Bo, R
    Comi, GP
    [J]. BRAIN, 2004, 127 : 2518 - 2532
  • [9] Neural stem cell transplantation can ameliorate the phenotype of a mouse model of spinal muscular atrophy
    Corti, Stefania
    Nizzardo, Monica
    Nardini, Martina
    Donadoni, Chiara
    Salani, Sabrina
    Ronchi, Dario
    Saladino, Francesca
    Bordoni, Andreina
    Fortunato, Francesco
    Del Bo, Roberto
    Papadimitriou, Dimitra
    Locatelli, Federica
    Menozzi, Giorgia
    Strazzer, Sandra
    Bresolin, Nereo
    Comi, Giacomo R.
    [J]. JOURNAL OF CLINICAL INVESTIGATION, 2008, 118 (10) : 3316 - 3330
  • [10] Neural stem cells LewisX+CXCR4+modify disease progression in an amyotrophic lateral sclerosis model
    Corti, Stefania
    Locatelli, Federica
    Papadimitriou, Dimitra
    Del Bo, Roberto
    Nizzardo, Monica
    Nardini, Martina
    Donadoni, Chiara
    Salani, Sabrina
    Fortunato, Francesco
    Strazzer, Sandra
    Bresolin, Nereo
    Comi, Giacomo P.
    [J]. BRAIN, 2007, 130 : 1289 - 1305
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