Slow Intrathecal Injection of rAAVrh10 Enhances its Transduction of Spinal Cord and Therapeutic Efficacy in a Mutant SOD1 Model of ALS

•Slow IT injection of rAAV delivers higher transduction to spinal cord compared with the fast injection.•Fast IT injection delivers higher transduction to brain and peripheral tissues compared with the slow injection.•Slow IT injection of a therapeutic rAAV led to a slightly better outcome in SOD1-G...

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Veröffentlicht in:	Neuroscience 2017-12, Vol.365, p.192-205
Hauptverfasser:	Li, Dongxiao, Liu, Chong, Yang, Chunxing, Wang, Dan, Wu, Dongxia, Qi, Yinkuang, Su, Qin, Gao, Guangping, Xu, Zuoshang, Guo, Yansu
Format:	Artikel
Sprache:	eng
Schlagworte:	AAV Adenomatous Polyposis Coli Protein - metabolism amyotrophic lateral sclerosis Amyotrophic Lateral Sclerosis - genetics Amyotrophic Lateral Sclerosis - pathology Amyotrophic Lateral Sclerosis - therapy Animals Body Weight - genetics Calcium-Binding Proteins - metabolism Dependovirus - genetics Disease Models, Animal Ganglia, Spinal - metabolism Ganglia, Spinal - pathology gene therapy Genetic Therapy - methods Glial Fibrillary Acidic Protein - metabolism Green Fluorescent Proteins - genetics Green Fluorescent Proteins - metabolism Injections, Spinal intrathecal Mice Mice, Inbred C57BL Mice, Transgenic Microfilament Proteins - metabolism Mutation - genetics SOD1-G93A Spinal Cord - metabolism Spinal Cord - pathology Superoxide Dismutase - genetics Transduction, Genetic
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