Christian L. Lorson, PhD


Curators’ Distinguished Professor
Associate Vice Chancellor for Research
Associate Dean for Research and Graduate Studies, College of Veterinary Medicine
Director, Spinal Cord Injury/Disease Research Program (SCIDRP)
Professor, Department of Veterinary Pathobiology

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

Building Address: Bond Life Sciences Center, Room 471-F, 1201 East Rollins Street
Phone Number: 573-884-2219
Lab Websites:

Lorson Lab Google Scholar

Lorson lab Twitter

Lorson lab Give Direct (Fund to Cure SMARD)


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.


All publications

Potential therapeutic targets for Mpox: the evidence to date.
Byrareddy SN, Sharma K, Sachdev S, Reddy AS, Acharya A, Klaustermeier KM, Lorson CL, Singh K. Expert Opin Ther Targets. 2023 Jul 4:1-13. doi: 10.1080/14728222.2023.2230361. Online ahead of print. PMID: 37368464

Normal muscle fiber type distribution is recapitulated in aged ephrin-A3 -/- mice that previously lacked most slow myofibers.
Arpke RW, Moritz TC, Hahn KL, Stark DA, Villalón E, Lorson CL, Cornelison D. Am J Physiol Cell Physiol. 2023 Mar 1;324(3):C718-C727. doi: 10.1152/ajpcell.00519.2022. Epub 2023 Jan 30. PMID: 36717102

ABT1 modifies SMARD1 pathology via interactions with IGHMBP2 and stimulation of ATPase and helicase activity.
Vadla GP, Ricardez Hernandez SM, Mao J, Garro-Kacher MO, Lorson ZC, Rice RP, Hansen SA, Lorson CL, Singh K, Lorson MA. JCI Insight. 2023 Jan 24;8(2):e164608. doi: 10.1172/jci.insight.164608. PMID: 36480289 

Mutations in the monkeypox virus replication complex: Potential contributing factors to the 2022 outbreak.
Kannan SR, Sachdev S, Reddy AS, Kandasamy SL, Byrareddy SN, Lorson CL, Singh K. J Autoimmun. 2022 Dec;133:102928. doi: 10.1016/j.jaut.2022.102928. Epub 2022 Oct 14. PMID: 36252459

Continued Complexity of Mutations in Omicron Sublineages.
Spratt AN, Kannan SR, Sharma K, Sachdev S, Kandasamy SL, Sönnerborg A, Lorson CL, Singh K. Biomedicines. 2022 Oct 16;10(10):2593. doi: 10.3390/biomedicines10102593. PMID: 36289855 

Motor unit recovery following Smn restoration in mouse models of spinal muscular atrophy.
Comley LH, Kline RA, Thomson AK, Woschitz V, Landeros EV, Osman EY, Lorson CL, Murray LM. Hum Mol Genet. 2022 Sep 10;31(18):3107-3119. doi: 10.1093/hmg/ddac097. PMID: 35551393 

A combinatorial approach increases SMN level in SMA model mice.
Dumas SA, Villalón E, Bergman EM, Wilson KJ, Marugan JJ, Lorson CL, Burnett BG. Hum Mol Genet. 2022 Aug 25;31(17):2989-3000. doi: 10.1093/hmg/ddac068. PMID: 35419606 

Friend or Foe(tal): challenges in development of a large animal model for pre-clinical fetal gene therapy.
Finkel RS, Lorson CL. Gene Ther. 2022 Jun;29(6):316-318. doi: 10.1038/s41434-022-00327-4. Epub 2022 Mar 8. PMID: 35256766 

Complex Mutation Pattern of Omicron BA.2: Evading Antibodies without Losing Receptor Interactions.
Kannan SR, Spratt AN, Sharma K, Goyal R, Sönnerborg A, Apparsundaram S, Lorson CL, Byrareddy SN, Singh K.Int J Mol Sci. 2022 May 16;23(10):5534. doi: 10.3390/ijms23105534.PMID: 35628343 

CRISPR-Mediated Enzyme Fragment Complementation Assay for Quantification of the Stability of Splice Isoforms.
Tang Z, Hegde S, Zhao J, Zhu S, Johnson KA, Lorson CL, Wang J. Chembiochem. 2022 May 4;23(9):e202200012. doi: 10.1002/cbic.202200012. Epub 2022 Mar 14. PMID: 35235240

The Ighmbp2D564N mouse model is the first SMARD1 model to demonstrate respiratory defects.
Smith CE, Lorson MA, Ricardez Hernandez SM, Al Rawi Z, Mao J, Marquez J, Villalón E, Keilholz AN, Smith CL, Garro-Kacher MO, Morcos T, Davis DJ, Bryda EC, Nichols NL, Lorson CL. Hum Mol Genet. 2022 Apr 22;31(8):1293-1307. doi: 10.1093/hmg/ddab317. PMID: 34726235 

The adeno-associated virus 2 genome and Rep 68/78 proteins interact with cellular sites of DNA damage.
Boftsi M, Whittle FB, Wang J, Shepherd P, Burger LR, Kaifer KA, Lorson CL, Joshi T, Pintel DJ, Majumder K. Hum Mol Genet. 2022 Mar 21;31(6):985-998. doi: 10.1093/hmg/ddab300.PMID: 34652429

Trans cohort metabolic reprogramming towards glutaminolysis in long-term successfully treated HIV-infection.
Mikaeloff F, Svensson Akusjärvi S, Ikomey GM, Krishnan S, Sperk M, Gupta S, Magdaleno GDV, Escós A, Lyonga E, Okomo MC, Tagne CT, Babu H, Lorson CL, Végvári Á, Banerjea AC, Kele J, Hanna LE, Singh K, de Magalhães JP, Benfeitas R, Neogi U. Commun Biol. 2022 Jan 11;5(1):27. doi: 10.1038/s42003-021-02985-3. PMID: 35017663 

Defining the optimal dose and therapeutic window in SMA with respiratory distress type I model mice, FVB/NJ-Ighmpb2  nmd-2J.
Shababi M, Smith CE, Ricardez Hernandez SM, Marquez J, Al Rawi Z, Villalón E, Farris KD, Garro-Kacher MO, Lorson CL. Mol Ther Methods Clin Dev. 2021 Aug 8;23:23-32. doi: 10.1016/j.omtm.2021.07.008. eCollection 2021 Dec 10. PMID: 34553000 

Discovery and Evaluation of Entry Inhibitors for SARS-CoV-2 and Its Emerging Variants.
Acharya A, Pandey K, Thurman M, Klug E, Trivedi J, Sharma K, Lorson CL, Singh K, Byrareddy SN. J Virol. 2021 Nov 23;95(24):e0143721. doi: 10.1128/JVI.01437-21. Epub 2021 Sep 22. PMID: 34550770 

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