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:

Lorson Lab Google Scholar:

Lorson lab Twitter:

Lorson lab Give Direct (Fund to Cure SMARD):

Research Emphasis:
Translational Medicine; Gene Therapy; Neurodegeneration; Spinal Muscular Atrophy; Spinal Muscular Atrophy with Respiratory Distress; CMT; Molecular Genetics; RNA Processing; 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), Charcot-Marie-Tooth (CMT), 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. 5) Novel SMARD1 patient-based models of disease. 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 2017-2021:

Distinct Metabolic Profile Associated with a Fatal Outcome in COVID-19 Patients during the Early Epidemic in Italy.
Saccon E, Bandera A, Sciumè M, Mikaeloff F, Lashari AA, Aliberti S, Sachs MC, Billi F, Blasi F, Gabriel EE, Costantino G, De Roberto P, Krishnan S, Gori A, Peyvandi F, Scudeller L, Canetta C, Lorson CL, Valenti L, Singh K, Baldini L, Fracchiolla NS; COVID-19 Network Working Group,, Neogi U.
Microbiol Spectr. 2021 Sep 1:e0054921. doi: 10.1128/Spectrum.00549-21. Online ahead of print.
PMID: 34468185

Evolutionary analysis of the Delta and Delta Plus variants of the SARS-CoV-2 viruses.
Kannan SR, Spratt AN, Cohen AR, Naqvi SH, Chand HS, Quinn TP, Lorson CL, Byrareddy SN, Singh K.
J Autoimmun. 2021 Aug 11;124:102715. doi: 10.1016/j.jaut.2021.102715. Online ahead of print.
PMID: 34399188

Evolution, correlation, structural impact and dynamics of emerging SARS-CoV-2 variants.
Spratt AN, Kannan SR, Woods LT, Weisman GA, Quinn TP, Lorson CL, Sönnerborg A, Byrareddy SN, Singh K.
Comput Struct Biotechnol J. 2021;19:3799-3809. doi: 10.1016/j.csbj.2021.06.037. Epub 2021 Jun 24.
PMID: 34188776

Survival motor neuron deficiency slows myoblast fusion through reduced myomaker and myomixer expression.
McCormack NM, Villalón E, Viollet C, Soltis AR, Dalgard CL, Lorson CL, Burnett BG.
J Cachexia Sarcopenia Muscle. 2021 Aug;12(4):1098-1116. doi: 10.1002/jcsm.12740. Epub 2021 Jun 11.
PMID: 34115448

Coronavirus helicases: attractive and unique targets of antiviral drug-development and therapeutic patents.
Spratt AN, Gallazzi F, Quinn TP, Lorson CL, Sönnerborg A, Singh K.
Expert Opin Ther Pat. 2021 Apr;31(4):339-350. doi: 10.1080/13543776.2021.1884224. Epub 2021 Apr 21.
PMID: 33593200

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 Sci Alliance. 2020 Nov 24;4(1):e202000889. doi: 10.26508/lsa.202000889. Print 2021 Jan.
PMID: 33234679

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.
J Neuroimmune Pharmacol. 2020 Dec;15(4):574-577. doi: 10.1007/s11481-020-09954-3. Epub 2020 Sep 15.
PMID: 32930936

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.
Biochem Biophys Res Commun. 2020 Sep 10;530(1):107-114. doi: 10.1016/j.bbrc.2020.07.031. Epub 2020 Jul 30.
PMID: 32828271

Minor snRNA gene delivery improves the loss of proprioceptive synapses on SMA motor neurons.
Osman EY, Van Alstyne M, Yen PF, Lotti F, Feng Z, Ling KK, Ko CP, Pellizzoni L, Lorson CL.
JCI Insight. 2020 Jun 18;5(12):e130574. doi: 10.1172/jci.insight.130574.
PMID: 32516136

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.
Biochem Biophys Res Commun. 2019 Dec 3;520(2):341-346. doi: 10.1016/j.bbrc.2019.10.032. Epub 2019 Oct 8.
PMID: 31604525

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.
Hum Mol Genet. 2019 Nov 15;28(22):3742-3754. doi: 10.1093/hmg/ddz188.
PMID: 31363739

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.
Sci Rep. 2019 Jul 1;9(1):9472. doi: 10.1038/s41598-019-45822-8.
PMID: 31263170

Muscle fiber-type selective propensity to pathology in the nmd mouse model of SMARD1.
Villalón E, Lee NN, Marquez J, Lorson CL.
Biochem Biophys Res Commun. 2019 Aug 13;516(1):313-319. doi: 10.1016/j.bbrc.2019.06.117. Epub 2019 Jun 28.
PMID: 31256932

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.
Hum Mol Genet. 2019 Oct 1;28(19):3199-3210. doi: 10.1093/hmg/ddz142.
PMID: 31211843

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.
Sci Rep. 2019 Feb 7;9(1):1633. doi: 10.1038/s41598-018-38208-9.
PMID: 30733501

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.
Mol Ther Methods Clin Dev. 2018 Aug 17;10:348-360. doi: 10.1016/j.omtm.2018.08.005. eCollection 2018 Sep 21.
PMID: 30202772

Selective vulnerability in neuronal populations in nmd/SMARD1 mice.
Villalón E, Shababi M, Kline R, Lorson ZC, Florea KM, Lorson CL.
Hum Mol Genet. 2018 Feb 15;27(4):679-690. doi: 10.1093/hmg/ddx434.
PMID: 29272405

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, Matera AG.
Mol Biol Cell. 2018 Jan 15;29(2):96-110. doi: 10.1091/mbc.E17-11-0627. Epub 2017 Nov 22.
PMID: 29167380

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.
PMID: 29103974

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.
PMID: 29066943

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.
PMID: 28637335

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.
PMID: 28598854

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.
PMID: 28481536

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.
PMID: 28362802

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 DDW, Lorson CL.
JCI Insight. 2017 Mar 9;2(5):e89970. doi: 10.1172/jci.insight.89970.
PMID: 28289706

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.
PMID: 28062667

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