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The Journal of Biological Chemistry|March 28, 2008
Therapeutic gene silencing delivered by a chemically modified small interfering RNA against mutant SOD1 slows amyotrophic lateral sclerosis progressionHongyan Wang, Animesh Ghosh, Huricha Baigude, et al.
Proceedings of the National Academy of Sciences of the United States of America|April 6, 2006
Spinal cord endoplasmic reticulum stress associated with a microsomal accumulation of mutant superoxide dismutase-1 in an ALS modelHitoshi Kikuchi, Gabriele Almer, Satoshi Yamashita, et al.
Neuroscience|October 13, 2017
Slow Intrathecal Injection of rAAVrh10 Enhances its Transduction of Spinal Cord and Therapeutic Efficacy in a Mutant SOD1 Model of ALSDongxiao Li, Chong Liu, Chunxing Yang, et al.
Molecular Neurobiology|January 7, 2014
Oxidative stress and autophagic alteration in brainstem of SOD1-G93A mouse model of ALSTing An, Pengxiao Shi, Weisong Duan, et al.
Aging Cell|August 26, 2003
Selective silencing by RNAi of a dominant allele that causes amyotrophic lateral sclerosisHongliu Ding, Dianne S Schwarz, Alex Keene, et al.
Human Molecular Genetics|October 11, 2013
Widespread spinal cord transduction by intrathecal injection of rAAV delivers efficacious RNAi therapy for amyotrophic lateral sclerosisHongyan Wang, Bin Yang, Linghua Qiu, et al.
ACS Macro Letters|June 2, 2022
Development of Excipient-Free Freeze-Dryable Unimolecular Hyperstar Polymers for Efficient siRNA SilencingLiang Zhao, Xiang Wu, Xiaofeng Wang, et al.
Human Molecular Genetics|May 13, 2015
Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespanCara M Weismann, Jennifer Ferreira, Allison M Keeler, et al.
Proceedings of the National Academy of Sciences of the United States of America|February 23, 2013
Reactive astrocytes secrete lcn2 to promote neuron deathFangfang Bi, Cao Huang, Jianbin Tong, et al.
Plos Genetics|September 13, 2006
Designing siRNA that distinguish between genes that differ by a single nucleotideDianne S Schwarz, Hongliu Ding, Lori Kennington, et al.
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