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CNS-targeted Viral Delivery of G-CSF in an Animal Model for ALS: Improved Efficacy and Preservation of the Neuromuscular Unit
被引:47
作者:

Henriques, Alexandre
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机构:
SYGNIS Biosci, Dept Mol Neurol, D-69120 Heidelberg, Germany
INSERM, U692, Strasbourg, France
Univ Strasbourg, Fac Med, UMRS692, Strasbourg, France SYGNIS Biosci, Dept Mol Neurol, D-69120 Heidelberg, Germany

Pitzer, Claudia
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SYGNIS Biosci, Dept Mol Neurol, D-69120 Heidelberg, Germany SYGNIS Biosci, Dept Mol Neurol, D-69120 Heidelberg, Germany

Dittgen, Tanjew
论文数: 0 引用数: 0
h-index: 0
机构:
SYGNIS Biosci, Dept Mol Neurol, D-69120 Heidelberg, Germany SYGNIS Biosci, Dept Mol Neurol, D-69120 Heidelberg, Germany

Klugmann, Matthias
论文数: 0 引用数: 0
h-index: 0
机构:
Univ New S Wales, Sch Med Sci, Dept Physiol, Translat Neurosci Facil, Sydney, NSW, Australia SYGNIS Biosci, Dept Mol Neurol, D-69120 Heidelberg, Germany

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Schneider, Armin
论文数: 0 引用数: 0
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SYGNIS Biosci, Dept Mol Neurol, D-69120 Heidelberg, Germany SYGNIS Biosci, Dept Mol Neurol, D-69120 Heidelberg, Germany
机构:
[1] SYGNIS Biosci, Dept Mol Neurol, D-69120 Heidelberg, Germany
[2] INSERM, U692, Strasbourg, France
[3] Univ Strasbourg, Fac Med, UMRS692, Strasbourg, France
[4] Univ New S Wales, Sch Med Sci, Dept Physiol, Translat Neurosci Facil, Sydney, NSW, Australia
关键词:
AMYOTROPHIC-LATERAL-SCLEROSIS;
COLONY-STIMULATING FACTOR;
PARKINSONS-DISEASE;
MOTOR-NEURONS;
GENE-TRANSFER;
MOUSE MODEL;
TRANSDUCTION;
MICE;
EXPRESSION;
TRIAL;
D O I:
10.1038/mt.2010.271
中图分类号:
Q81 [生物工程学(生物技术)];
Q93 [微生物学];
学科分类号:
071005 ;
0836 ;
090102 ;
100705 ;
摘要:
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease characterized by progressive loss of motoneurons. We have recently uncovered a new neurotrophic growth factor, granulocyte-colony stimulating factor (G-CSF), which protects alpha-motoneurons, improves functional outcome, and increases life expectancy of SOD-1 (G93A) mice when delivered subcutaneously. However, chronic systemic delivery of G-CSF is complicated by elevation of neutrophilic granulocytes. Here, we used adeno-associated virus (AAV) to directly target and confine G-CSF expression to the spinal cord. Whereas intramuscular injection of AAV failed to transduce motoneurons retrogradely, and caused a high systemic load of G-CSF, intraspinal delivery led to a highly specific enrichment of G-CSF in the spinal cord with moderate peripheral effects. Intraspinal delivery improved motor functions, delayed disease progression, and increased survival by 10%, longer than after systemic delivery. Mechanistically, we could show that G-CSF in addition to rescuing motoneurons improved neuromuscular junction (NMJ) integrity and enhanced motor axon regeneration after nerve crush injury. Collectively, our results show that intraspinal delivery improves efficacy of G-CSF treatment in an ALS mouse model while minimizing the systemic load of G-CSF, suggesting a new therapeutic option for ALS treatment.
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页码:284 / 292
页数:9
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