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Christian Lorson

Christian Lorson

Professor, Veterinary Pathobiology

Phone: 573-884-2219 or 573-884-2680 (lab)
lorsonc@missouri.edu
471G Bond Life Sciences Center

Christian Lorson is a professor of veterinary pathobiology and molecular microbiology and immunology (MMI). The Lorson lab focuses on spinal muscular atrophy (SMA), a devastating disease which affects one in 6,000 live births, and is the leading genetic cause of infantile deaths. SMA develops in children who are missing a specific gene called SMN1. The disease is characterized by degeneration and loss of lower spinal motor neurons, causing muscle weakness and atrophy, and often resulting in respiratory failure. In 2008, the Lorson lab investigated a compound that increases muscle mass. In 2010, the lab team, while studying mice, discovered a relationship between SMA and cardiac problems, which suggests that SMA is a multi-system disease and not confined to the nervous system. Currently, the lab is collaborating with several groups to develop new drugs that will encourage the body to produce more SMN protein, with a goal of moving closer toward clinical trial.

News about Christian Lorson

Research Topics

Short-duration splice promoting compound enables a tunable mouse model of spinal muscular atrophy. Rietz A, Hodgetts KJ, Lusic H, Quist KM, Osman EY, Lorson CL, Androphy EJ. Life Science Alliance; 4(1):e202000889.

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Infectivity of SARS-CoV-2: there Is Something More than D614G?. Kannan SR, Spratt AN, Quinn TP, Heng X, Lorson CL, Sönnerborg A, Byrareddy SN, Singh K. Journal of Neuroimmune Pharmacology; 15(4):574-577.

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AAV9-DOK7 gene therapy reduces disease severity in Smn2B/- SMA model mice. Kaifer KA, Villalón E, Smith CE, Simon ME, Marquez J, Hopkins AE, Morcos TI, Lorson CL. Biochemical and Biophysical Research Communications; 530(1):107-114.

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Minor snRNA gene delivery improves the loss of proprioceptive synapses on SMA motor neurons. Osman EY, van Alstyne M, Yen P-F, Lotti F, Feng Z, Ling KKY, Ko C-P, Pellizzoni L, Lorson CL. JCI Insight; 5(12):e130574.

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Development of a novel severe mouse model of spinal muscular atrophy with respiratory distress type 1: FVB-nmd. Shababi M, Smith CE, Kacher M, Alrawi Z, Villalón E, Davis D, Bryda EC, Lorson CL. Biochemical and Biophysical Research Communications; 520(2):341-346.

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Functional characterization of SMN evolution in mouse models of SMA. Osman EY, Bolding MR, Villalón E, Kaifer KA, Lorson ZC, Tisdale S, Hao Y, Conant GC, Pires JC, Pellizzoni L, Lorson CL. Scientific Reports; 9(1):9472.

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Intraperitoneal delivery of a novel drug‐like compound improves disease severity in severe and intermediate mouse models of Spinal Muscular Atrophy. Osman EY, Rietz A, Kline RA, Cherry JJ, Hodgetts KJ, Lorson CL, Androphy EJ. Scientific Reports; 9(1):1633.

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AAV9-Stathmin1 gene delivery improves disease phenotype in an intermediate mouse model of spinal muscular atrophy. Villalón E, Kline RA, Smith CE, Lorson ZC, Osman EY, O'day S, Murray LM, Lorson CL. Human Molecular Genetics; 28(22):3742-3754.

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AAV9-mediated delivery of miR-23a reduces disease severity in Smn2B/-SMA model mice. Kaifer KA, Villalón E, O'Brien BS, Sison SL, Smith CE, Simon ME, Marquez J, O'Day S, Hopkins AE, Neff R, Rindt H, Ebert AD, Lorson CL. Human Molecular Genetics; 28(19):3199-3210.

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Muscle fiber-type selective propensity to pathology in the nmd mouse model of SMARD1. Villalón E, Lee NN, Marquez J, Lorson CL. Biochemical and Biophysical Research Communications; 516(1):313-319.

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A Direct Comparison of IV and ICV Delivery Methods for Gene Replacement Therapy in a Mouse Model of SMARD1. Shababi M, Villalón E, Kaifer KA, DeMarco V, Lorson CL. Molecular Therapy - Methods and Clinical Development; 10:348-360.

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Selective vulnerability in neuronal populations in nmd/SMARD1 mice. Villalón E, Shababi M, Kline R, Lorson ZC, Florea KM, Lorson CL. Human Molecular Genetics; 27(4):679-690.

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Self-oligomerization regulates stability of survival motor neuron protein isoforms by sequestering an SCFSlmb degron. Gray KM, Kaifer KA, Baillat D, Wen Y, Bonacci TR, Ebert AD, Raimer AC, Spring AM, Have ST, Glascock JJ, Gupta K, Van Duyne GD, Emanuele MJ, Lamond AI, Wagner EJ, Lorson CL, Gregory Matera A. Molecular Biology of the Cell; 29(2):96-110.

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Optimization of a series of heterocycles as survival motor neuron gene transcription enhancers. Choi S, Calder AN, Miller EH, Anderson KP, Fiejtek DK, Rietz A, Li H, Cherry JJ, Quist KM, Xing X, Glicksman MA, Cuny GD, Lorson CL, Androphy EA, Hodgetts KJ. Bioorganic and Medicinal Chemistry Letters; 27(23):5144-5148.

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Optimization of trans-splicing for Huntington’s disease RNA therapy. Rindt H, Tom CM, Lorson CL, Mattis VB. Frontiers in Neuroscience; 11(OCT):544.

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Astrocyte-produced miR-146a as a mediator of motor neuron loss in spinal muscular atrophy. Sison SL, Patitucci TN, Seminary ER, Villalon E, Lorson CL, Ebert AD. Human Molecular Genetics; 26(17):3409-3420.

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Analysis of Azithromycin Monohydrate as a Single or a Combinatorial Therapy in a Mouse Model of Severe Spinal Muscular Atrophy. Osman EY, Washington CW, Simon ME, Megiddo D, Greif H, Lorson CL. Journal of Neuromuscular Diseases; 4(3):237-249.

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Discovery of a Small Molecule Probe That Post-Translationally Stabilizes the Survival Motor Neuron Protein for the Treatment of Spinal Muscular Atrophy. Rietz A, Li H, Quist KM, Cherry JJ, Lorson CL, Burnett BG, Kern NL, Calder AN, Fritsche M, Lusic H, Boaler PJ, Choi S, Xing X, Glicksman MA, Cuny GD, Androphy EJ, Hodgetts KJ. Journal of Medicinal Chemistry; 60(11):4594-4610.

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Comparison of independent screens on differentially vulnerable motor neurons reveals alpha-synuclein as a common modifier in motor neuron diseases. Kline RA, Kaifer KA, Osman EY, Carella F, Tiberi A, Ross J, Pennetta G, Lorson CL, Murray LM. PLoS Genetics; 13(3):e1006680.

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SMN deficiency negatively impacts red pulp macrophages and spleen development in mouse models of spinal muscular atrophy. Khairallah M-T, Astroski J, Custer SK, Androphy EJ, Franklin CL, Lorson CL. Human Molecular Genetics; 26(5):932-941.

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Gene Therapy for Spinal Muscular Atrophy. Miller MR, Osman EY, Lorson CL. Molecular and Cellular Therapies for Motor Neuron Diseases; 233-250.

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Contributions of Different Cell Types to Spinal Muscular Atrophy Pathogenesis. Deguise MO, Patitucci TN, Ebert AD, Lorson CL, Kothary R. Spinal Muscular Atrophy: Disease Mechanisms and Therapy; 167-181.

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Retraction: The SMN structure reveals its crucial role in snRNP assembly [Human Molecular Genetics, 24, 8, 2138-46]doi: 10.1093/hmg/ddu734. Seng CO, Magee C, Young PJ, Lorson CL, Allen JP. Human Molecular Genetics; 25(24):5516.

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Optimization of morpholino antisense oligonucleotides targeting the intronic repressor element1 in spinal muscular atrophy. Osman EY, Washington CW, Kaifer KA, Mazzasette C, Patitucci TN, Florea KM, Simon ME, Ko C-P, Ebert AD, Lorson CL. Molecular Therapy; 24(9):1592-1601.

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Rescue of a mouse model of spinal muscular atrophy with respiratory distress type 1 by AAV9-IGHMBP2 is dose dependent. Shababi M, Feng Z, Villalon E, Sibigtroth CM, Osman EY, Miller MR, Williams-Simon PA, Lombardi A, Sass TH, Atkinson AK, Garcia ML, Ko C-P, Lorson CL. Molecular Therapy; 24(5):855-866.

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Placental development in a mouse model of spinal muscular atrophy. Van Gronigen Caesar G, Dale JM, Osman EY, Garcia ML, Lorson CL, Schulz LC. Biochemical and Biophysical Research Communications; 470(1):82-87.

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Astrocytes influence the severity of spinal muscular atrophy. Rindt H, Feng Z, Mazzasette C, Glascock JJ, Valdivia D, Pyles N, Crawford TO, Swoboda KJ, Patitucci TN, Ebert AD, Sumner CJ, Ko C-P, Lorson CL. Human Molecular Genetics; 24(14):4094-4102.

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The SMN structure reveals its crucial role in snRNP assembly (RETRACTED). Seng CO, Magee C, Young PJ, Lorson CL, Allen JP. Human Molecular Genetics; 24(8):2138-2146.

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Gene therapy in spinal muscular atrophy (SMA) models using intracerebroventricular injection into neonatal mice. Shababi M, Osman EY, Lorson CL. Neuromethods; 98:297-320.

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Morpholino antisense oligonucleotides targeting intronic repressor Element1 improve phenotype in SMA mouse models. Osman EY, Miller MR, Robbins KL, Lombardi AM, Atkinson AK, Brehm AJ, Lorson CL. Human Molecular Genetics; 23(18):4832-4845.

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Defining the therapeutic window in a severe animal model of spinal muscular atrophy. Robbins KL, Glascock JJ, Osman EY, Miller MR, Lorson CL. Human Molecular Genetics; 23(17):4559-4568.

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Spinal muscular atrophy: A motor neuron disorder or a multi-organ disease?. Shababi M, Lorson CL, Rudnik-Schöneborn SS. Journal of Anatomy; 224(1):15-28.

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Restoration of SMN to Emx-1 expressing cortical neurons is not sufficient to provide benefit to a severe mouse model of Spinal Muscular Atrophy. Taylor AS, Glascock JJ, Rose FF Jr, Lutz C, Lorson CL. Transgenic Research; 22(5):1029-1036.

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Infantile spinale Muskelatrophie: mehr als eine Motoneuronerkrankung? [Infantile spinal muscular atrophy: More than a motor neuron disease?]. Rudnik-Schöneborn S, Lorson CL, Shababi M. Medizinische Genetik; 25(3):347-351.

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Enhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug-like compounds. Cherry JJ, Osman EY, Evans MC, Choi S, Xing X, Cuny GD, Glicksman MA, Lorson CL, Androphy EJ. EMBO Molecular Medicine; 5(7):1103-1118.

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Development and characterization of an SMN2-based intermediate mouse model of spinal muscular atrophy. Cobb MS, Rose FF, Rindt H, Glascock JJ, Shababi M, Miller MR, Osman EY, Yen P-F, Garcia ML, Martin BR, Wetz MJ, Mazzasette C, Feng Z, Ko C-P, Lorson CL. Human Molecular Genetics; 22(9):1843-1855.

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Replacement of huntingtin exon 1 by trans-splicing. Rindt H, Yen P-F, Thebeau CN, Peterson TS, Weisman GA, Lorson CL. Cellular and Molecular Life Sciences; 69(24):4191-4204.

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SMN-inducing compounds for the treatment of spinal muscular atrophy. Lorson MA, Lorson CL. Future Medicinal Chemistry; 4(16):2067-2084.

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Analysis of a read-through promoting compound in a severe mouse model of spinal muscular atrophy. Mattis VB, Tom Chang C-W, Lorson CL. Neuroscience Letters; 525(1):72-75.

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Partial restoration of cardio-vascular defects in a rescued severe model of spinal muscular atrophy. Shababi M, Habibi J, Ma L, Glascock JJ, Sowers JR, Lorson CL. Journal of Molecular and Cellular Cardiology; 52(5):1074-1082.

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Transgenic inactivation of murine myostatin does not decrease the severity of disease in a model of Spinal Muscular Atrophy. Rindt H, Buckley DM, Vale SM, Krogman M, Rose FF, Garcia ML, Lorson CL. Neuromuscular Disorders; 22(3):277-285.

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Decreasing disease severity in symptomatic, Smn-/-;SMN2 ++, spinal muscular atrophy mice following scAAV9-SMN delivery. Glascock JJ, Osman EY, Wetz MJ, Krogman MM, Shababi M, Lorson CL. Human Gene Therapy; 23(3):330-335.

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Optimization of SMN trans-splicing through the analysis of SMN introns. Shababi M, Lorson CL. Journal of Molecular Neuroscience; 46(3):459-469.

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Direct central nervous system delivery provides enhanced protection following vector mediated gene replacement in a severe model of Spinal Muscular Atrophy. Glascock JJ, Shababi M, Wetz MJ, Krogman MM, Lorson CL. Biochemical and Biophysical Research Communications; 417(1):376-381.

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Bifunctional RNAs targeting the intronic splicing silencer N1 increase SMN levels and reduce disease severity in an animal model of spinal muscular atrophy. Osman EY, Yen P-F, Lorson CL. Molecular Therapy; 20(1):119-126.

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Disruption of the Survival Motor Neuron (SMN) gene in pigs using ssDNA. Lorson MA, Spate LD, Samuel MS, Murphy CN, Lorson CL, Prather RS, Wells KD. Transgenic Research; 20(6):1293-1304.

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Delivery of therapeutic agents through intracerebroventricular (ICV) and intravenous (IV) injection in mice. Glascock JJ, Osman EY, Coady TH, Rose FF, Shababi M, Lorson CL. Journal of Visualized Experiments; 56:e2968.

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The spinal muscular atrophy mouse model, SMAΔ7, displays altered axonal transport without global neurofilament alterations. Dale JM, Shen H, Barry DM, Garcia VB, Rose FF Jr, Lorson CL, Garcia ML. Acta Neuropathologica; 122(3):331-341.

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SMN in spinal muscular atrophy and snRNP biogenesis. Coady TH, Lorson CL. Wiley Interdisciplinary Reviews: RNA; 2(4):546-564.

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The COPI vesicle complex binds and moves with survival motor neuron within axons. Peter CJ, Evans M, Thayanithy V, Taniguchi-Ishigaki N, Bach I, Kolpak A, Bassell GJ, Rossoll W, Lorson CL, Bao Z-Z, Androphy EJ. Human Molecular Genetics; 20(9):1701-1711.

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Combination of SMN trans-splicing and a neurotrophic factor increases the life span and body mass in a severe model of spinal muscular atrophy. Shababi M, Glascock J, Lorson CL. Human Gene Therapy; 22(2):135-144.

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Therapeutics that directly increase SMN expression to treat spinal muscular atrophy. Shababi M, Mattis VB, Lorson CL. Drug News and Perspectives; 23(8):475-482.

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Cardiac defects contribute to the pathology of spinal muscular atrophy models. Shababi M, Habibi J, Yang HT, Vale SM, Sewell WA, Lorson CL. Human Molecular Genetics; 19(20):4059-4071.

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Identification of a self-association domain in the Ewing’s sarcoma protein: A novel function for arginine-glycine-glycine rich motifs?. Shaw DJ, Morse R, Todd AG, Eggleton P, Lorson CL, Young PJ. Journal of Biochemistry; 147(6):885-893.

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Spinal muscular atrophy: Mechanisms and therapeutic strategies. Lorson CL, Rindt H, Shababi M. Human Molecular Genetics; 19(R1):R111-R118.

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Trans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy. Coady TH, Lorson CL. Journal of Neuroscience; 30(1):126-130.

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Effect of diet on the survival and phenotype of a mouse model for spinal muscular atrophy. Butchbach MER, Rose FF Jr, Rhoades S, Marston J, McCrone JT, Sinnott R, Lorson CL. Biochemical and Biophysical Research Communications; 391(1):835-840.

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Identification of a tripartite import signal in the Ewing Sarcoma protein (EWS). Shaw DJ, Morse R, Todd AG, Eggleton P, Lorson CL, Young PJ. Biochemical and Biophysical Research Communications; 390(4):1197-1201.

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RNA-targeting approaches for neuromuscular diseases. Le Roy F, Charton K, Lorson CL, Richard I. Trends in Molecular Medicine; 15(12):580-591.

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Subcutaneous administration of TC007 reduces disease severity in an animal model of SMA. Mattis VB, Fosso MY, Chang C-W, Lorson CL. BMC Neuroscience; 10:142.

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Delivery of a read-through inducing compound, TC007, lessens the severity of a spinal muscular atrophy animal model. Mattis VB, Ebert AD, Fosso MY, Chang C-W, Lorson CL. Human Molecular Genetics; 18(20):3906-3913.

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Delivery of bifunctional RNAs that target an intronic repressor and increase SMN levels in an animal model of spinal muscular atrophy. Baughan TD, Dickson A, Osman EY, Lorson CL. Human Molecular Genetics; 18(9):1600-1611.

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Delivery of recombinant follistatin lessens disease severity in a mouse model of spinal muscular atrophy. Rose FF Jr, Mattis VB, Rindt H, Lorson CL. Human Molecular Genetics; 18(6):997-1005.

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Induced pluripotent stem cells from a spinal muscular atrophy patient. Ebert AD, Yu J, Rose FF Jr, Mattis VB, Lorson CL, Thomson JA, Svendsen CN. Nature; 457(7227):277-280.

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A negatively acting bifunctional RNA increases survival motor neuron both in vitro and in vivo. Dickson A, Osman E, Lorson CL. Human Gene Therapy; 19(11):1307-1315.

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Detection of human survival motor neuron (SMN) protein in mice containing the SMN2 transgene: Applicability to preclinical therapy development for spinal muscular atrophy. Mattis VB, Butchbach MER, Lorson CL. Journal of Neuroscience Methods; 175(1):36-43.

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Development of a single vector system that enhances Trans-splicing of SMN2 transcripts. Coady TH, Baughan TD, Shababi M, Passini MA, Lorson CL. PLoS ONE; 3(10):e3468.

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Identification and characterisation of a nuclear localisation signal in the SMN associated protein, Gemin4. Lorson MA, Dickson AM, Shaw DJ, Todd AG, Young EC, Morse R, Wolstencroft C, Lorson CL, Young PJ. Biochemical and Biophysical Research Communications; 375(1):33-37.

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The Wallerian degeneration slow (Wlds) gene does not attenuate disease in a mouse model of spinal muscular atrophy. Rose FF Jr, Meehan PW, Coady TH, Garcia VB, Garcia ML, Lorson CL. Biochemical and Biophysical Research Communications; 375(1):119-123.

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A SMNΔ7 read-through product confers functionality to the SMNΔ7 protein. Mattis VB, Bowerman M, Kothary R, Lorson CL. Neuroscience Letters; 442(1):54-58.

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Identification and characterization of the porcine (Sus scrofa) survival motor neuron (SMN1) gene: An animal model for therapeutic studies. Lorson MA, Spate LD, Prather RS, Lorson CL. Developmental Dynamics; 237(8):2268-2278.

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Protein phosphatase 1 binds to the RNA recognition motif of several splicing factors and regulates alternative pre-mRNA processing. Novoyatleva T, Heinrich B, Tang Y, Benderska N, Butchbach MER, Lorson CL, Lorson MA, Ben-Dov C, Fehlbaum P, Bracco L, Burghes AHM, Bollen M, Stamm S. Human Molecular Genetics; 17(1):52-70.

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Induction of full-length survival motor neuron by polyphenol botanical compounds. Sakla MS, Lorson CL. Human Genetics; 122(6):635-643.

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Restoration of SMN function: Delivery of a trans-splicing RNA Re-directs SMN2 pre-mRNA splicing. Coady TH, Shababi M, Tullis GE, Lorson CL. Molecular Therapy; 15(8):1471-1478.

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Novel aminoglycosides increase SMN levels in spinal muscular atrophy fibroblasts. Mattis VB, Rai R, Wang J, Chang C-WT, Coady T, Lorson CL. Human Genetics; 120(4):589-601.

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Survival motor neuron function in motor axons is independent of functions required for small nuclear ribonucleoprotein biogenesis. Carrel TL, McWhorter ML, Workman E, Zhang H, Wolstencroft EC, Lorson C, Bassell GJ, Burghes AHM, Beattie CE. Journal of Neuroscience; 26(43):11014-11022.

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Surface plasmon resonance sensors for detection of survival of motor neuron protein. Mitropanopoulos M, Campanaro B, Malone M, Masson J-F, Nieman RA, Lorson CL, Booksh KS. Proceedings of SPIE - The International Society for Optical Engineering; 6380:638008.

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Stimulating Full-Length SMN2 Expression by Delivering Bifunctional RNAs via a Viral Vector. Baughan T, Shababi M, Coady TH, Dickson AM, Tullis GE, Lorson CL. Molecular Therapy; 14(1):54-62.

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A non-sequence-apecific requirement for SMN protein activity: The role of aminoglycosides in inducing elevated SMN protein levels. Wolstencroft EC, Mattis V, Bajer AA, Young PJ, Lorson CL. Human Molecular Genetics; 14(9):1199-1210.

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Minute virus of mice small non-structural protein NS2 localizes within, but is not required for the formation of, Smn-associated autonomous parvovirus-associated replication bodies. Young PJ, Newman A, Jensen KT, Burger LR, Pintel DJ, Lorson CL. Journal of General Virology; 86(4):1009-1014.

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Monitoring of recombinant survival motor neuron protein using fiber-optic surface plasmon resonance. Masson J-F, Barnhart M, Battaglia TM, Morris GE, Nieman RA, Young PJ, Lorson CL, Booksh KS. Analyst; 129(9):855-859.

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A survival motor neuron:tetanus toxin fragment C fusion protein for the targeted delivery of SMN protein to neurons. Francis JW, Figueiredo D, VanderSpek JC, Ayala LM, Kim YS, Remington MP, Young PJ, Lorson CL, Ikebe S, Fishman PS, Brown RH Jr. Brain Research; 995(1):84-96.

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The Ewing’s sarcoma protein interacts with the Tudor domain of the survival motor neuron protein. Young PJ, Francis JW, Lince D, Coon K, Androphy EJ, Lorson CL. Molecular Brain Research; 119(1):37-49.

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A transgene carrying an A2G missense mutation in the SMN gene modulates phenotypic severity in mice with severe (type I) spinal muscular atrophy. Monani UR, Pastore MT, Gavrilina TO, Jablonka S, Le TT, Andreassi C, DiCocco JM, Lorson C, Androphy EJ, Sendtner M, Podell M, Burghes AHM. Journal of Cell Biology; 160(1):41-52.

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Survival motor neuron protein in the nucleolus of mammalian neurons. Wehner KA, Ayala L, Kim Y, Young PJ, Hosler BA, Lorson CL, Baserga SJ, Francis JW. Brain Research; 945(2):160-173.

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Minute virus of mice small nonstructural protein NS2 interacts and colocalizes with the Smn protein. Young PJ, Jensen KT, Burger LR, Pintel DJ, Lorson CL. Journal of Virology; 76(12):6364-6369.

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Minute virus of mice NS1 interacts with the SMN protein, and they colocalize in novel nuclear bodies induced by parvovirus infection. Young PJ, Jensen KT, Burger LR, Pintel DJ, Lorson CL. Journal of Virology; 76(8):3892-3904.

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SRp30c-dependent stimulation of survival motor neuron (SMN) exon 7 inclusion is facilitated by a direct interaction with hTra2β1. Young PJ, DiDonato CJ, Hu D, Kothary R, Androphy EJ, Lorson CL. Human Molecular Genetics; 11(5):577-587.

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A direct interaction between the survival motor neuron protein and p53 and its relationship to spinal muscular atrophy. Young PJ, Day PM, Zhou J, Androphy EJ, Morris GE, Lorson CL. Journal of Biological Chemistry; 277(4):2852-2859.

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An in vivo reporter system for measuring increased inclusion of exon 7 in SMN2 mRNA: Potential therapy of SMA. Zhang ML, Lorson CL, Androphy EJ, Zhou J. Gene Therapy; 8(20):1532-1538.

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Regulation of murine survival motor neuron (Smn) protein levels by modifying Smn exon 7 splicing. DiDonato CJ, Lorson CL, De Repentigny Y, Simard L, Chartrand C, Androphy EJ, Kothary R. Human Molecular Genetics; 10(23):2727-2736.

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Erratum: The exon 2b region of the spinal muscular atophy protein, SMN, is involved in self-association and SIP1 binding (Human Molecular Genetics (2000) vol. 9 (2869-2877)). Young PJ, Man NT, Lorson CL, Le TT, Androphy EJ, Burghes AHM, Morris GE. Human Molecular Genetics; 10(1):88.

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The exon 2b region of the spinal muscular atrophy protein, SMN, is involved in self-association and SIP1 binding. Young PJ, Man NT, Lorson CL, Le TT, Androphy EJ, Burghes AHM, Morris GE. Human Molecular Genetics; 9(19):2869-2877.

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Htra2-β1 stimulates an exonic splicing enhancer and can restore full-length SMN expression to survival motor neuron 2 (SMN2). Hofmann Y, Lorson CL, Stamm S, Androphy EJ, Wirth B. Proceedings of the National Academy of Sciences of the United States of America; 97(17):9618-9623.

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An exonic enhancer is required for inclusion of an essential exon in the SMA-determining gene SMN. Lorson CL, Androphy EJ. Human Molecular Genetics; 9(2):259-266.

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Identification of survival motor neuron as a transcriptional activator-binding protein. Strasswimmer J, Lorson CL, Breiding DE, Chen JJ, Le T, Burghes AHM, Androphy EJ. Human Molecular Genetics; 8(7):1219-1226.

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A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2. Monani UR, Lorson CL, Parsons DW, Prior TW, Androphy EJ, Burghes AHM, McPherson JD. Human Molecular Genetics; 8(7):1177-1183.

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A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy. Lorson CL, Hahnen E, Androphy EJ, Wirth B. Proceedings of the National Academy of Sciences of the United States of America; 96(11):6307-6311.

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The domain encoded by exon 2 of the survival motor neuron protein mediates nucleic acid binding. Lorson CL, Androphy EJ. Human Molecular Genetics; 7(8):1269-1275.

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SMN oligomerization defect correlates with spinal muscular atrophy severity. Lorson CL, Strasswimmer J, Yao J-M, Baleja JD, Hahnen E, Wirth B, Le T, Burghes AHM, Androphy EJ. Nature Genetics; 19(1):63-66.

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An SP1-binding site and TATA element are sufficient to support full transactivation by proximally bound NS1 protein of minute virus of mice. Lorson C, Pearson J, Burger L, Pintel DJ. Virology; 240(2):326-337.

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Corrigendum: The domain encoded by exon 2 of the survival motor neuron protein mediates nucleic acid binding (Human Molecular Genetics (1998) 7 (1269-1275)). Lorson CL, Androphy EJ. Human Molecular Genetics; 7(11):1831.

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Characterization of the minute virus of mice P38 core promoter elements. Lorson C, Pintel DJ. Journal of Virology; 71(9):6568-6575.

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Efficient transactivation of the minute virus of mice P38 promoter requires upstream binding of NS1. Lorson C, Burger LR, Mouw M, Pintel DJ. Journal of Virology; 70(2):834-842.

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