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Christian Lorson

Research Profile

Chemicals & Drugs

Disorders

Living Beings

Anatomy

Physiology

Trans Splicing

Procedures

Genetic Therapy

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Melissa S Cobb; Zhihua Feng; Michael L Garcia; Jacqueline J Glascock; Chien-Ping Ko; Brittanie R Martin; Chiara Mazzasette; Madeline R Miller; Erkan Y Osman; Hansjörg Rindt; Ferril F Rose; Monir Shababi; Mary J Wetz; Pei-Fen Yen; Christian L Lorson
Development and characterization of an SMN2-based intermediate mouse model of Spinal Muscular Atrophy.
Hansjörg Rindt; Christina N Thebeau; Gary A Weisman; Pei-Fen Yen; Troy S Peterson; Christian L Lorson
Replacement of huntingtin exon 1 by trans-splicing.
Monique A Lorson; Christian L Lorson
SMN-inducing compounds for the treatment of spinal muscular atrophy.

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All Publications

2013: Melissa S Cobb; Zhihua Feng; Michael L Garcia; Jacqueline J Glascock; Chien-Ping Ko; Brittanie R Martin; Chiara Mazzasette; Madeline R Miller; Erkan Y Osman; Hansjörg Rindt; Ferril F Rose; Monir Shababi; Mary J Wetz; Pei-Fen Yen; Christian L Lorson
Development and characterization of an SMN2-based intermediate mouse model of Spinal Muscular Atrophy.
Human molecular genetics 2013;22(9):1843-55.
2012: Hansjörg Rindt; Christina N Thebeau; Gary A Weisman; Pei-Fen Yen; Troy S Peterson; Christian L Lorson
Replacement of huntingtin exon 1 by trans-splicing.
Cellular and molecular life sciences : CMLS 2012;69(24):4191-204.
2012: Monique A Lorson; Christian L Lorson
SMN-inducing compounds for the treatment of spinal muscular atrophy.
Future medicinal chemistry 2012;4(16):2067-84.
2012: Virginia B Mattis; Cheng-Wei Tom Chang; Christian L Lorson
Analysis of a read-through promoting compound in a severe mouse model of spinal muscular atrophy.
Neuroscience letters 2012;525(1):72-5.
2012: Monir Shababi; James R Sowers; Jacqueline J Glascock; Javad Habibi; Lixin Ma; Christian L Lorson
Partial restoration of cardio-vascular defects in a rescued severe model of spinal muscular atrophy.
Journal of molecular and cellular cardiology 2012;52(5):1074-82.
2012: Hansjörg Rindt; Ferrill F Rose; Spencer M Vale; Desire M Buckley; Michael L Garcia; Megan Krogman; Christian L Lorson
Transgenic inactivation of murine myostatin does not decrease the severity of disease in a model of Spinal Muscular Atrophy.
Neuromuscular disorders : NMD 2012;22(3):277-85.
2012: Jacqueline J Glascock; Megan M Krogman; Erkan Y Osman; Monir Shababi; Mary J Wetz; Christian L Lorson
Decreasing disease severity in symptomatic, Smn(-/-);SMN2(+/+), spinal muscular atrophy mice following scAAV9-SMN delivery.
Human gene therapy 2012;23(3):330-5.
2012: Monir Shababi; Christian L Lorson
Optimization of SMN trans-splicing through the analysis of SMN introns.
Journal of molecular neuroscience : MN 2012;46(3):459-69.
2012: Jacqueline J Glascock; Megan M Krogman; Monir Shababi; Mary J Wetz; Christian L Lorson
Direct central nervous system delivery provides enhanced protection following vector mediated gene replacement in a severe model of spinal muscular atrophy.
Biochemical and biophysical research communications 2012;417(1):376-81.
2011: Monique A Lorson; Clifton N Murphy; Randall S Prather; Melissa S Samuel; Lee Spate; Christian L Lorson; Kevin D Wells
Disruption of the Survival Motor Neuron (SMN) gene in pigs using ssDNA.
Transgenic research 2011;20(6):1293-304.
2011: Jeffrey M Dale; Devin M Barry; Virginia B Garcia; Christian L Lorson; Ferrill F Rose; Hailian Shen; Michael L Garcia
The spinal muscular atrophy mouse model, SMAΔ7, displays altered axonal transport without global neurofilament alterations.
Acta neuropathologica 2011;122(3):331-41.
2011: Tristan H Coady; Christian L Lorson
SMN in spinal muscular atrophy and snRNP biogenesis.
Wiley interdisciplinary reviews. RNA 2011;2(4):546-64.
2011: Cyril Jayakumar Peter; Matthew Evans; Venugopal Thayanithy; Naoko Taniguchi-Ishigaki; Ingolf Bach; Adrianne Kolpak; Gary J. Bassell; wilfried rossoll; Christian L Lorson; Zheng-Zheng Bao; Elliot J Androphy
The COPI vesicle complex binds and moves with survival motor neuron within axons.
Human molecular genetics 2011;20(9):1701-11.
2011: Monir Shababi; Jacqueline Glascock; Christian L Lorson
Combination of SMN trans-splicing and a neurotrophic factor increases the life span and body mass in a severe model of spinal muscular atrophy.
Human gene therapy 2011;22(2):135-44.
2011: Jacqueline J Glascock; Erkan Y Osman; Ferrill F Rose; Monir Shababi; Tristan H Coady; Christian L Lorson
Delivery of therapeutic agents through intracerebroventricular (ICV) and intravenous (IV) injection in mice.
Journal of visualized experiments : JoVE 2011;(56):.
2010: Monir Shababi; Javad Habibi; Hsiao T Yang; Spencer M Vale; Will A Sewell; Christian L Lorson
Cardiac defects contribute to the pathology of spinal muscular atrophy models.
Human molecular genetics 2010;19(20):4059-71.
2010: Monir Shababi; Virginia B Mattis; Christian L Lorson
Therapeutics that directly increase SMN expression to treat spinal muscular atrophy.
Drug news & perspectives 2010;23(8):475-82.
2010: Debra J Shaw; Robert Morse; Adrian G Todd; Paul Eggleton; Christian L Lorson; Philip J Young
Identification of a self-association domain in the Ewing's sarcoma protein: a novel function for arginine-glycine-glycine rich motifs?
Journal of biochemistry 2010;147(6):885-93.
2010: Christian L Lorson; Hansjorg Rindt; Monir Shababi
Spinal muscular atrophy: mechanisms and therapeutic strategies.
Human molecular genetics 2010;19(R1):R111-8.
2010: Tristan H Coady; Christian L Lorson
Trans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy.
The Journal of neuroscience : the official journal of the Society for Neuroscience 2010;30(1):126-30.
2010: Matthew E R Butchbach; Ferrill F Rose; Sarah Rhoades; John Marston; John T McCrone; Rachel Sinnott; Christian L Lorson
Effect of diet on the survival and phenotype of a mouse model for spinal muscular atrophy.
Biochemical and biophysical research communications 2010;391(1):835-40.
2009: Debra J Shaw; Robert Morse; Adrian G Todd; Paul Eggleton; Christian L Lorson; Philip J Young
Identification of a tripartite import signal in the Ewing Sarcoma protein (EWS).
Biochemical and biophysical research communications 2009;390(4):1197-201.
2009: Florence Le Roy; Karine Charton; Christian L Lorson; Isabelle Richard
RNA-targeting approaches for neuromuscular diseases.
Trends in molecular medicine 2009;15(12):580-91.
2009: Virginia B Mattis; Allison D Ebert; Marina Y Fosso; Cheng-Wei Chang; Christian L Lorson
Delivery of a read-through inducing compound, TC007, lessens the severity of a spinal muscular atrophy animal model.
Human molecular genetics 2009;18(20):3906-13.
2009: Travis D Baughan; Alexa Dickson; Erkan Y Osman; Christian L Lorson
Delivery of bifunctional RNAs that target an intronic repressor and increase SMN levels in an animal model of spinal muscular atrophy.
Human molecular genetics 2009;18(9):1600-11.
2009: Ferrill F Rose; Virginia B Mattis; Hansjörg Rindt; Christian L Lorson
Delivery of recombinant follistatin lessens disease severity in a mouse model of spinal muscular atrophy.
Human molecular genetics 2009;18(6):997-1005.
2009: Allison D Ebert; Junying Yu; Ferrill F Rose; Virginia B Mattis; Christian L Lorson; James A Thomson; Clive Svendsen
Induced pluripotent stem cells from a spinal muscular atrophy patient.
Nature 2009;457(7227):277-80.
2009: Virginia B Mattis; Marina Y Fosso; Cheng-Wei Chang; Christian L Lorson
Subcutaneous administration of TC007 reduces disease severity in an animal model of SMA.
BMC neuroscience 2009;10():142.
2008: Alexa Dickson; Erkan Osman; Christian L Lorson
A negatively acting bifunctional RNA increases survival motor neuron both in vitro and in vivo.
Human gene therapy 2008;19(11):1307-15.
2008: Virginia B Mattis; Matthew E R Butchbach; Christian L Lorson
Detection of human survival motor neuron (SMN) protein in mice containing the SMN2 transgene: applicability to preclinical therapy development for spinal muscular atrophy.
Journal of neuroscience methods 2008;175(1):36-43.
2008: Ferrill F Rose; Philip W Meehan; Tristan H Coady; Virginia B Garcia; Michael L Garcia; Christian L Lorson
The Wallerian degeneration slow (Wld(s)) gene does not attenuate disease in a mouse model of spinal muscular atrophy.
Biochemical and biophysical research communications 2008;375(1):119-23.
2008: Monique A Lorson; Alexa M Dickson; Debra J Shaw; Adrian G Todd; Elizabeth C Young; Robert Morse; Catherine Wolstencroft; Christian L Lorson; Philip Young
Identification and characterisation of a nuclear localisation signal in the SMN associated protein, Gemin4.
Biochemical and biophysical research communications 2008;375(1):33-7.
2008: Virginia B Mattis; Melissa Bowerman; Rashmi Kothary; Christian L Lorson
A SMNDelta7 read-through product confers functionality to the SMNDelta7 protein.
Neuroscience letters 2008;442(1):54-8.
2008: Monique A Lorson; Lee D Spate; Randall S Prather; Christian L Lorson
Identification and characterization of the porcine (Sus scrofa) survival motor neuron (SMN1) gene: an animal model for therapeutic studies.
Developmental dynamics : an official publication of the American Association of Anatomists 2008;237(8):2268-78.
2008: Tristan H Coady; Travis D Baughan; Monir Shababi; Marco A Passini; Christian L Lorson
Development of a single vector system that enhances trans-splicing of SMN2 transcripts.
PloS one 2008;3(10):e3468.
2008: Mary S Sakla; Christian L Lorson
Induction of full-length survival motor neuron by polyphenol botanical compounds.
Human genetics 2008;122(6):635-43.
2008: Tatyana Novoyatleva; Bettina Heinrich; Yesheng Tang; Natalya Benderska; Matthew E R Butchbach; Christian L Lorson; Monique A Lorson; Claudia Ben-Dov; Pascale Fehlbaum; Laurent Bracco; Arthur H M Burghes; Mathieu Bollen; Stefan Stamm
Protein phosphatase 1 binds to the RNA recognition motif of several splicing factors and regulates alternative pre-mRNA processing.
Human molecular genetics 2008;17(1):52-70.
2007: Tristan H Coady; Monir Shababi; Gregory E Tullis; Christian L Lorson
Restoration of SMN function: delivery of a trans-splicing RNA re-directs SMN2 pre-mRNA splicing.
Molecular therapy : the journal of the American Society of Gene Therapy 2007;15(8):1471-8.
2006: Virginia B Mattis; Ravi Rai; Jinhua Wang; Cheng-Wei T Chang; Tristan H Coady; Christian L Lorson
Novel aminoglycosides increase SMN levels in spinal muscular atrophy fibroblasts.
Human genetics 2006;120(4):589-601.
2006: Tessa L Carrel; Michelle L McWhorter; Eileen Workman; Honglai Zhang; Elizabeth C Wolstencroft; Christian L Lorson; Gary J. Bassell; Arthur H M Burghes; Christine E Beattie
Survival motor neuron function in motor axons is independent of functions required for small nuclear ribonucleoprotein biogenesis.
The Journal of neuroscience : the official journal of the Society for Neuroscience 2006;26(43):11014-22.
2006: Travis Baughan; Monir Shababi; Tristan H Coady; Alexa M Dickson; Gregory E Tullis; Christian L Lorson
Stimulating full-length SMN2 expression by delivering bifunctional RNAs via a viral vector.
Molecular therapy : the journal of the American Society of Gene Therapy 2006;14(1):54-62.
2005: Elizabeth C Wolstencroft; Virginia Mattis; Anna A Bajer; Philip Young; Christian L Lorson
A non-sequence-specific requirement for SMN protein activity: the role of aminoglycosides in inducing elevated SMN protein levels.
Human molecular genetics 2005;14(9):1199-210.
2005: Philip Young; Ann Newman; Klaus T Jensen; Lisa R Burger; David J Pintel; Christian L Lorson
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.
The Journal of general virology 2005;86(Pt 4):1009-14.
2004: Jean-Francois Masson; Margaret Barnhart; Tina M Battaglia; Glenn E Morris; Ronald A Nieman; Philip Young; Christian L Lorson; Karl Steven Booksh
Monitoring of recombinant survival motor neuron protein using fiber-optic surface plasmon resonance.
The Analyst 2004;129(9):855-9.
2004: Jonathan W Francis; Dayse Figueiredo; Johanna C vanderSpek; Laura M Ayala; Young Seon Kim; Mary P Remington; Philip Young; Christian L Lorson; Shinichiro Ikebe; Paul S Fishman; Robert H Brown
A survival motor neuron:tetanus toxin fragment C fusion protein for the targeted delivery of SMN protein to neurons.
Brain research 2004;995(1):84-96.
2003: Philip Young; Jonathan W Francis; Diane Lince; Keith D Coon; Elliot J Androphy; Christian L Lorson
The Ewing's sarcoma protein interacts with the Tudor domain of the survival motor neuron protein.
Brain research. Molecular brain research 2003;119(1):37-49.
2003: Umrao R Monani; Matthew T Pastore; Tatiana O Gavrilina; Sibylle Jablonka; Thanh T Le; Catia Andreassi; Jennifer M DiCocco; Christian L Lorson; Elliot J Androphy; Michael Sendtner; Michael Podell; Arthur H M Burghes
A transgene carrying an A2G missense mutation in the SMN gene modulates phenotypic severity in mice with severe (type I) spinal muscular atrophy.
The Journal of cell biology 2003;160(1):41-52.
2002: Karen A Wehner; Laura Ayala; Youngseon Kim; Philip Young; Betsy A Hosler; Christian L Lorson; Susan J. Baserga; Jonathan W Francis
Survival motor neuron protein in the nucleolus of mammalian neurons.
Brain research 2002;945(2):160-73.
2002: Philip Young; Klaus T Jensen; Lisa R Burger; David J Pintel; Christian L Lorson
Minute virus of mice small nonstructural protein NS2 interacts and colocalizes with the Smn protein.
Journal of virology 2002;76(12):6364-9.
2002: Philip Young; Klaus T Jensen; Lisa R Burger; David J Pintel; Christian L Lorson
Minute virus of mice NS1 interacts with the SMN protein, and they colocalize in novel nuclear bodies induced by parvovirus infection.
Journal of virology 2002;76(8):3892-904.
2002: Philip Young; Christine J DiDonato; Diane Hu; Rashmi Kothary; Elliot J Androphy; Christian L Lorson
SRp30c-dependent stimulation of survival motor neuron (SMN) exon 7 inclusion is facilitated by a direct interaction with hTra2 beta 1.
Human molecular genetics 2002;11(5):577-87.
2002: Philip Young; Patricia M Day; Jianhua Zhou; Elliot J Androphy; Glenn E Morris; Christian L Lorson
A direct interaction between the survival motor neuron protein and p53 and its relationship to spinal muscular atrophy.
The Journal of biological chemistry 2002;277(4):2852-9.
2001: Christine J DiDonato; Christian L Lorson; Yves De Repentigny; Louise R Simard; C Chartrand; Elliot J Androphy; Rashmi Kothary
Regulation of murine survival motor neuron (Smn) protein levels by modifying Smn exon 7 splicing.
Human molecular genetics 2001;10(23):2727-36.
2001: M L Zhang; Christian L Lorson; Elliot J Androphy; J Zhou
An in vivo reporter system for measuring increased inclusion of exon 7 in SMN2 mRNA: potential therapy of SMA.
Gene therapy 2001;8(20):1532-8.
2000: Philip Young; Nguyen Thi Man; Christian L Lorson; Thanh T Le; Elliot J Androphy; Arthur H M Burghes; Glenn E Morris
The exon 2b region of the spinal muscular atrophy protein, SMN, is involved in self-association and SIP1 binding.
Human molecular genetics 2000;9(19):2869-77.
2000: Yvonne Hofmann; Christian L Lorson; Stefan Stamm; Elliot J Androphy; Brunhilde Wirth
Htra2-beta 1 stimulates an exonic splicing enhancer and can restore full-length SMN expression to survival motor neuron 2 (SMN2).
Proceedings of the National Academy of Sciences of the United States of America 2000;97(17):9618-23.
2000: Christian L Lorson; Elliot J Androphy
An exonic enhancer is required for inclusion of an essential exon in the SMA-determining gene SMN.
Human molecular genetics 2000;9(2):259-65.
1999: J Strasswimmer; Christian L Lorson; DE Breiding; J J Chen; T Le; Arthur H M Burghes; Elliot J Androphy
Identification of survival motor neuron as a transcriptional activator-binding protein.
Human molecular genetics 1999;8(7):1219-26.
1999: Umrao R Monani; Christian L Lorson; DONALD PARSONS; Thomas W Prior; Elliot J Androphy; Arthur H M Burghes; J D McPherson
A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2.
Human molecular genetics 1999;8(7):1177-83.
1999: Christian L Lorson; eric hahnen; Elliot J Androphy; Brunhilde Wirth
A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy.
Proceedings of the National Academy of Sciences of the United States of America 1999;96(11):6307-11.
1998: Christian L Lorson; Elliot J Androphy
The domain encoded by exon 2 of the survival motor neuron protein mediates nucleic acid binding.
Human molecular genetics 1998;7(8):1269-75.
1998: Christian L Lorson; J Strasswimmer; J M Yao; James D Baleja; eric hahnen; Brunhilde Wirth; T Le; Arthur H M Burghes; Elliot J Androphy
SMN oligomerization defect correlates with spinal muscular atrophy severity.
Nature genetics 1998;19(1):63-6.
1998: Christian L Lorson; J Pearson; Lisa R Burger; David J Pintel
An Sp1-binding site and TATA element are sufficient to support full transactivation by proximally bound NS1 protein of minute virus of mice.
Virology 1998;240(2):326-37.
1997: Christian L Lorson; David J Pintel
Characterization of the minute virus of mice P38 core promoter elements.
Journal of virology 1997;71(9):6568-75.
1996: Christian L Lorson; Lisa R Burger; M Mouw; David J Pintel
Efficient transactivation of the minute virus of mice P38 promoter requires upstream binding of NS1.
Journal of virology 1996;70(2):834-42.