Christian L. Lorson

Affiliations: 
University of Missouri - Columbia, Columbia, MO, United States 
Area:
Microbiology Biology, Neuroscience Biology, Molecular Biology, Genetics
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"Christian Lorson"
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Publications

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Shababi M, Smith CE, Kacher M, et al. (2019) Development of a novel severe mouse model of spinal muscular atrophy with respiratory distress type 1: FVB-nmd. Biochemical and Biophysical Research Communications
Kaifer KA, Villalón E, O'Brien BS, et al. (2019) AAV9-Mediated Delivery of miR-23a Reduces Disease Severity in Smn2B-/SMA Model Mice. Human Molecular Genetics
Shababi M, Villalón E, Kaifer KA, et al. (2018) A Direct Comparison of IV and ICV Delivery Methods for Gene Replacement Therapy in a Mouse Model of SMARD1. Molecular Therapy. Methods & Clinical Development. 10: 348-360
Villalón E, Shababi M, Kline R, et al. (2017) Selective vulnerability in neuronal populations in nmd/SMARD1 mice. Human Molecular Genetics
Rindt H, Tom CM, Lorson CL, et al. (2017) Optimization of trans-Splicing for Huntington's Disease RNA Therapy. Frontiers in Neuroscience. 11: 544
Shababi M, Feng Z, Villalon E, et al. (2016) Rescue of a Mouse Model of Spinal Muscular Atrophy with Respiratory Distress type 1 (SMARD1) by AAV9-IGHMBP2 is Dose Dependent. Molecular Therapy : the Journal of the American Society of Gene Therapy
Rindt H, Feng Z, Mazzasette C, et al. (2015) Astrocytes influence the severity of spinal muscular atrophy. Human Molecular Genetics. 24: 4094-102
Seng CO, Magee C, Young PJ, et al. (2015) The SMN structure reveals its crucial role in snRNP assembly. Human Molecular Genetics. 24: 2138-46
Osman EY, Miller MR, Robbins KL, et al. (2014) Morpholino antisense oligonucleotides targeting intronic repressor Element1 improve phenotype in SMA mouse models. Human Molecular Genetics. 23: 4832-45
Robbins KL, Glascock JJ, Osman EY, et al. (2014) Defining the therapeutic window in a severe animal model of spinal muscular atrophy. Human Molecular Genetics. 23: 4559-68
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