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Blood|March 1, 2024
Sickle cell disease iPSC-derived sensory neurons exhibit increased excitability and sensitization to patient plasmaReilly L Allison, Emily Welby, Vanessa Ehlers, et al.
Journal of Cardiovascular Pharmacology and Therapeutics|March 5, 2016
Nicorandil, a Nitric Oxide Donor and ATP-Sensitive Potassium Channel Opener, Protects Against Dystrophin-Deficient CardiomyopathyMuhammad Z Afzal, Melanie Reiter, Courtney Gastonguay, et al.
Cells|March 4, 2020
Motor Neuron Generation from iPSCs from Identical Twins Discordant for Amyotrophic Lateral SclerosisEmily R Seminary, Stephanie Santarriaga, Lynn Wheeler, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|July 13, 2016
Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular AtrophyErkan Y Osman, Charles W Washington, Kevin A Kaifer, et al.
Proceedings of the National Academy of Sciences of the United States of America|November 17, 2023
Mitigating aberrant Cdk5 activation alleviates mitochondrial defects and motor neuron disease symptoms in spinal muscular atrophyNimrod Miller, Zhaofa Xu, Katharina A Quinlan, et al.
Human Molecular Genetics|April 26, 2015
Astrocytes influence the severity of spinal muscular atrophyHansjörg Rindt, Zhihua Feng, Chiara Mazzasette, et al.
Human Molecular Genetics|June 19, 2019
AAV9-mediated delivery of miR-23a reduces disease severity in Smn2B/-SMA model miceKevin A Kaifer, Eric Villalón, Benjamin S O'Brien, et al.
Molecular Cell|July 21, 2018
SRCP1 Conveys Resistance to Polyglutamine AggregationStephanie Santarriaga, Holly N Haver, Adam J Kanack, et al.
Stem Cell Research|March 12, 2013
EZ spheres: a stable and expandable culture system for the generation of pre-rosette multipotent stem cells from human ESCs and iPSCsAllison D Ebert, Brandon C Shelley, Amanda M Hurley, et al.
Biology Open|August 19, 2024
High mobility group box 1 (HMGB1) is a potential disease biomarker in cell and mouse models of Duchenne muscular dystrophyRebecca A Slick, Jessica Sutton, Margaret Haberman, et al.
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