Department of Veterinary Pathobiology

Christian Lorson
Associate Dean for Research and Graduate Studies

  • Ph.D. 1997, University of Missouri-Columbia
  • B.A. 1991, Colorado College

Building Address: Bond LSC 471-G
Phone Number: 573-884-2219
Lab Website:

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Research Emphasis:
Translational Medicine; Gene Therapy; Neurodegeneration; Spinal Muscular Atrophy; Spinal Muscular Atrophy with Respiratory Distress; Molecular Genetics; RNA Processing; Alternative Splicing; Animal Models of Disease

The Lorson lab is comprised of several research teams, largely centered on translational medicine and neurodegenerative diseases, such as Spinal Muscular Atrophy (SMA), SMA with Respiratory Distress (SMARD), Huntington’s Disease, and others. Current projects include: 1) the development of a novel drug for SMA – a splice-switching antisense oligonucleotide – that dramatically extends survival in SMA animal models from a single dose at birth. 2) Identification of genes and pathways that improve the SMA/SMARD phenotypes, such as Plastin-3 or alpha-synuclein and then validating these factors using AAV-mediated gene therapy. 3) Small molecule development in collaboration with the Androphy lab (IUPUI) and the Hodgetts lab (LDDN/Harvard). 4) Gene replacement for SMARD and development of new animal models to push gene therapy towards the clinic. Overall, the Lorson lab is a highly dynamic, interactive, team-centric group that leverages molecular genetics/biology to develop tools and novel therapeutics for devastating genetic diseases.

Selected Publications 2016/2017:

Selective vulnerability in neuronal populations in nmd/SMARD1 mice.
Villalón E, Shababi M, Kline R, Lorson ZC, Florea KM, Lorson CL. Hum Mol Genet. 2017 Dec 20. doi: 10.1093/hmg/ddx434. [Epub ahead of print]

Self-oligomerization regulates stability of Survival Motor Neuron (SMN) 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, Matera AG. Mol Biol Cell. 2017 Nov 22. pii: mbc.E17-11-0627. doi: 10.1091/mbc.E17-11-0627. [Epub ahead of print]

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. Bioorg Med Chem Lett. 2017 Dec 1;27(23):5144-5148. doi: 10.1016/j.bmcl.2017.10.066. Epub 2017 Oct 26.

Optimization of trans-Splicing for Huntington's Disease RNA Therapy.
Rindt H, Tom CM, Lorson CL, Mattis VB. Front Neurosci. 2017 Oct 10;11:544. doi: 10.3389/fnins.2017.00544. eCollection 2017.

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. Hum Mol Genet. 2017 Sep 1;26(17):3409-3420. doi: 10.1093/hmg/ddx230.

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. J Neuromuscul Dis. 2017;4(3):237-249. doi: 10.3233/JND-170230.

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. J Med Chem. 2017 Jun 8;60(11):4594-4610. doi: 10.1021/acs.jmedchem.6b01885. Epub 2017 May 19.

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 Genet. 2017 Mar 31;13(3):e1006680. doi: 10.1371/journal.pgen.1006680. eCollection 2017 Mar.

Plastin-3 extends survival and reduces severity in mouse models of spinal muscular atrophy.
Kaifer KA, Villalón E, Osman EY, Glascock JJ, Arnold LL, Cornelison DD, Lorson CL. JCI Insight. 2017 Mar 9;2(5):e89970. doi: 10.1172/jci.insight.89970.

SMN deficiency negatively impacts red pulp macrophages and spleen development in mouse models of spinal muscular atrophy.
Khairallah MT, Astroski J, Custer SK, Androphy EJ, Franklin CL, Lorson CL. Hum Mol Genet. 2017 Mar 1;26(5):932-941. doi: 10.1093/hmg/ddx008.

Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy.
Osman EY, Washington CW 3rd, Kaifer KA, Mazzasette C, Patitucci TN, Florea KM, Simon ME, Ko CP, Ebert AD, Lorson CL. Mol Ther. 2016 Sep;24(9):1592-601. doi: 10.1038/mt.2016.145. Epub 2016 Jul 9.

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 CP, Lorson CL. Mol Ther. 2016 May;24(5):855-66. doi: 10.1038/mt.2016.33. Epub 2016 Feb 10.

Placental development in a mouse model of spinal muscular atrophy.
Van Gronigen Caesar G, Dale JM, Osman EY, Garcia ML, Lorson CL, Schulz LC. Biochem Biophys Res Commun. 2016 Jan 29;470(1):82-7. doi: 10.1016/j.bbrc.2015.12.120. Epub 2015 Dec 31.

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