Widespread spinal cord transduction by intrathecal injection of rAAV delivers efficacious RNAi therapy for amyotrophic lateral sclerosis

Hongyan Wang(University of Massachusetts Chan Medical School), Zuoshang Xu(University of Massachusetts Chan Medical School), Robert H. Brown(University of Massachusetts Chan Medical School), Linghua Qiu, Chunxing Yang(University of Massachusetts Chan Medical School), Qin Su(Jiangsu University), Yansu Guo(Capital Medical University), Guangping Gao(University of Massachusetts Chan Medical School), Joshua Kramer(New England Biolabs (United States)), Bin Yang(California Institute of Technology)
Human Molecular Genetics
September 18, 2013
10.1093/hmg/ddt454
Cited by 92

Related Papers

Viral vector platforms within the gene therapy landscape
|Signal Transduction and Targeted Therapy|2021|1.3k
dSarm/Sarm1 Is Required for Activation of an Injury-Induced Axon Death Pathway
|Science|2012|722
Adeno-associated virus as a delivery vector for gene therapy of human diseases
|Signal Transduction and Targeted Therapy|2024|664
ANG mutations segregate with familial and 'sporadic' amyotrophic lateral sclerosis
|Nature Genetics|2006|657
Amyotrophic Lateral Sclerosis-Associated SOD1 Mutant Proteins Bind and Aggregate with Bcl-2 in Spinal Cord Mitochondria
|Neuron|2004|495