C. Lorson

发表

Htra2-beta 1 stimulates an exonic splicing enhancer and can restore full-length SMN expression to survival motor neuron 2 (SMN2).

S. Stamm, Y. Hofmann, C. Lorson, 2000, Proceedings of the National Academy of Sciences of the United States of America.

A SMNΔ7 read-through product confers functionality to the SMNΔ7 protein

M. Bowerman, C. Lorson, V. Mattis, 2008, Neuroscience Letters.

A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy.

C. Lorson, E. Androphy, B. Wirth, 1999, Proceedings of the National Academy of Sciences of the United States of America.

SRp30c-dependent stimulation of survival motor neuron (SMN) exon 7 inclusion is facilitated by a direct interaction with hTra2 beta 1.

C. Lorson, E. Androphy, R. Kothary, 2002, Human molecular genetics.

The domain encoded by exon 2 of the survival motor neuron protein mediates nucleic acid binding.

C. Lorson, E. Androphy, 1998, Human molecular genetics.

The SMN structure reveals its crucial role in snRNP assembly.

C. Lorson, C. Seng, James P. Allen, 2015, Human molecular genetics.

Minor snRNA gene delivery improves the loss of proprioceptive synapses on SMA motor neurons.

C. Lorson, Meaghan Van Alstyne, L. Pellizzoni, 2020, JCI insight.

Comparison of independent screens on differentially vulnerable motor neurons reveals alpha-synuclein as a common modifier in motor neuron diseases

C. Lorson, G. Pennetta, R. Kline, 2017, PLoS genetics.

A transgene carrying an A2G missense mutation in the SMN gene modulates phenotypic severity in mice with severe (type I) spinal muscular atrophy

M. Podell, C. Lorson, E. Androphy, 2003, The Journal of cell biology.

Efficient transactivation of the minute virus of mice P38 promoter requires upstream binding of NS1

C. Lorson, D. Pintel, L. Burger, 1996, Journal of virology.

Analysis of Azithromycin Monohydrate as a Single or a Combinatorial Therapy in a Mouse Model of Severe Spinal Muscular Atrophy.

C. Lorson, E. Osman, H. Greif, 2017, Journal of neuromuscular diseases.

Novel aminoglycosides increase SMN levels in spinal muscular atrophy fibroblasts

C. Lorson, V. Mattis, Virginia B. Mattis, 2006, Human Genetics.

Trans-Splicing-Mediated Improvement in a Severe Mouse Model of Spinal Muscular Atrophy

C. Lorson, Tristan H. Coady, 2010, The Journal of Neuroscience.

Transgenic inactivation of murine myostatin does not decrease the severity of disease in a model of Spinal Muscular Atrophy

C. Lorson, H. Rindt, M. L. Garcia, 2012, Neuromuscular Disorders.

Rescue of a Mouse Model of Spinal Muscular Atrophy With Respiratory Distress Type 1 by AAV9-IGHMBP2 Is Dose Dependent.

C. Lorson, P. Williams-Simon, E. Villalón, 2016, Molecular therapy : the journal of the American Society of Gene Therapy.

Self-oligomerization regulates stability of survival motor neuron protein isoforms by sequestering an SCFSlmb degron

Michael J. Emanuele, E. Wagner, C. Lorson, 2018, Molecular biology of the cell.

SCFSlmb recognizes a conserved degron within the survival motor neuron (SMN) self-interaction domain to mediate ubiquitylation of SMN and SMNΔ7 isoforms

Michael J. Emanuele, E. Wagner, C. Lorson, 2016 .

CRISPR‐Mediated Enzyme Fragment Complementation Assay for Quantification of the Stability of Splice Isoforms

C. Lorson, Jingxin Wang, Shoutian Zhu, 2022, Chembiochem : a European journal of chemical biology.

Self-oligomerization regulates stability of Survival Motor Neuron (SMN) protein isoforms by sequestering an SCFSlmb degron

Michael J. Emanuele, E. Wagner, C. Lorson, 2017, bioRxiv.

The spinal muscular atrophy mouse model, SMAΔ7, displays altered axonal transport without global neurofilament alterations

C. Lorson, D. Barry, V. B. Garcia, 2011, Acta Neuropathologica.

Complex Mutation Pattern of Omicron BA.2: Evading Antibodies without Losing Receptor Interactions

S. Apparsundaram, C. Lorson, A. Sönnerborg, 2022, International journal of molecular sciences.

The Wallerian degeneration slow (Wld(s)) gene does not attenuate disease in a mouse model of spinal muscular atrophy.

C. Lorson, V. B. Garcia, Tristan H. Coady, 2008, Biochemical and biophysical research communications.

SMN deficiency negatively impacts red pulp macrophages and spleen development in mouse models of spinal muscular atrophy

C. Lorson, E. Androphy, C. Franklin, 2017, Human molecular genetics.

Partial restoration of cardio-vascular defects in a rescued severe model of spinal muscular atrophy.

C. Lorson, J. Sowers, J. Habibi, 2012, Journal of molecular and cellular cardiology.

Muscle fiber-type selective propensity to pathology in the nmd mouse model of SMARD1.

C. Lorson, J. Marquez, E. Villalón, 2019, Biochemical and biophysical research communications.

Induction of full-length survival motor neuron by polyphenol botanical compounds

C. Lorson, Mary S. Sakla, 2007, Human Genetics.

Detection of human survival motor neuron (SMN) protein in mice containing the SMN2 transgene: Applicability to preclinical therapy development for spinal muscular atrophy

C. Lorson, V. Mattis, M. Butchbach, 2008, Journal of Neuroscience Methods.

Survival motor neuron deficiency slows myoblast fusion through reduced myomaker and myomixer expression

C. Lorson, Barrington G. Burnett, C. Dalgard, 2021, Journal of cachexia, sarcopenia and muscle.

Edinburgh Research Explorer Comparison of independent screens on differentially vulnerable motor neurons reveals alpha-synuclein as a common modifier in motor neuron diseases

C. Lorson, G. Pennetta, R. Kline, 2017 .

The COPI vesicle complex binds and moves with survival motor neuron within axons.

C. Lorson, E. Androphy, V. Thayanithy, 2011, Human molecular genetics.

Identification and characterisation of a nuclear localisation signal in the SMN associated protein, Gemin4.

C. Lorson, R. Morse, M. Lorson, 2008, Biochemical and biophysical research communications.

Identification and characterization of the porcine (Sus scrofa) survival motor neuron (SMN1) gene: An animal model for therapeutic studies

C. Lorson, L. Spate, R. Prather, 2008, Developmental dynamics : an official publication of the American Association of Anatomists.

Effect of diet on the survival and phenotype of a mouse model for spinal muscular atrophy.

C. Lorson, J. McCrone, M. Butchbach, 2010, Biochemical and biophysical research communications.

The Ewing's sarcoma protein interacts with the Tudor domain of the survival motor neuron protein.

C. Lorson, E. Androphy, K. Coon, 2003, Brain research. Molecular brain research.

A Direct Interaction between the Survival Motor Neuron Protein and p53 and Its Relationship to Spinal Muscular Atrophy*

C. Lorson, E. Androphy, P. Day, 2002, The Journal of Biological Chemistry.

A non-sequence-specific requirement for SMN protein activity: the role of aminoglycosides in inducing elevated SMN protein levels.

C. Lorson, V. Mattis, A. Bajer, 2005, Human molecular genetics.

AAV9-Stathmin1 gene delivery improves disease phenotype in an intermediate mouse model of Spinal Muscular Atrophy.

C. Lorson, R. Kline, L. Murray, 2019, Human molecular genetics.

Intraperitoneal delivery of a novel drug‐like compound improves disease severity in severe and intermediate mouse models of Spinal Muscular Atrophy

C. Lorson, E. Androphy, R. Kline, 2019, Scientific Reports.

The exon 2b region of the spinal muscular atrophy protein, SMN, is involved in self-association and SIP1 binding.

C. Lorson, E. Androphy, G. Morris, 2000, Human molecular genetics.

The exon 2 b region of the spinal muscular atrophy protein , SMN , is involved in self-association and SIP 1 binding

C. Lorson, E. Androphy, G. Morris, 2000 .

Defining the optimal dose and therapeutic window in SMA with respiratory distress type I model mice, FVB/NJ-Ighmpb2nmd-2J

C. Lorson, J. Marquez, E. Villalón, 2021, Molecular therapy. Methods & clinical development.

Identification of a tripartite import signal in the Ewing Sarcoma protein (EWS).

C. Lorson, R. Morse, P. Eggleton, 2009, Biochemical and biophysical research communications.

Morpholino antisense oligonucleotides targeting intronic repressor Element1 improve phenotype in SMA mouse models.

C. Lorson, E. Osman, M. Miller, 2014, Human molecular genetics.

Minute Virus of Mice Small Nonstructural Protein NS2 Interacts and Colocalizes with the Smn Protein

C. Lorson, K. Jensen, D. Pintel, 2002, Journal of Virology.

Minute Virus of Mice NS1 Interacts with the SMN Protein, and They Colocalize in Novel Nuclear Bodies Induced by Parvovirus Infection

C. Lorson, K. Jensen, D. Pintel, 2002, Journal of Virology.

Gene Therapy for Spinal Muscular Atrophy

C. Lorson, E. Osman, M. Miller, 2017 .

Subcutaneous administration of TC007 reduces disease severity in an animal model of SMA

C. Lorson, V. Mattis, Virginia B. Mattis, 2009, BMC Neuroscience.

Plastin-3 extends survival and reduces severity in mouse models of spinal muscular atrophy.

C. Lorson, D. Cornelison, E. Villalón, 2017, JCI insight.

Optimization of a series of heterocycles as survival motor neuron gene transcription enhancers.

C. Lorson, M. Glicksman, G. Cuny, 2017, Bioorganic & medicinal chemistry letters.

A negatively acting bifunctional RNA increases survival motor neuron both in vitro and in vivo.

C. Lorson, Alexa M Dickson, E. Osman, 2008, Human gene therapy.

Continued Complexity of Mutations in Omicron Sublineages

C. Lorson, A. Sönnerborg, S. Sachdev, 2022, Biomedicines.

A Direct Interaction between the Survival Motor Neuron Protein and p 53 and Its Relationship to Spinal Muscular Atrophy *

C. Lorson, E. Androphy, P. Day, 2002 .

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.

C. Lorson, K. Jensen, D. Pintel, 2005, The Journal of general virology.

Placental development in a mouse model of spinal muscular atrophy.

C. Lorson, L. Schulz, E. Osman, 2016, Biochemical and biophysical research communications.

Analysis of a read-through promoting compound in a severe mouse model of spinal muscular atrophy

C. Lorson, V. Mattis, Cheng-Wei Chang, 2012, Neuroscience Letters.

Motor unit recovery following Smn restoration in mouse models of spinal muscular atrophy

C. Lorson, R. Kline, L. Murray, 2022, Human molecular genetics.

A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2.

J. McPherson, D. Parsons, C. Lorson, 1999, Human molecular genetics.

Short-duration splice promoting compound enables a tunable mouse model of spinal muscular atrophy

C. Lorson, E. Androphy, H. Lusic, 2020, Life Science Alliance.

Gene Therapy in Spinal Muscular Atrophy (SMA) Models Using Intracerebroventricular Injection into Neonatal Mice

C. Lorson, E. Osman, M. Shababi, 2015 .

Identification of a self-association domain in the Ewing's sarcoma protein: a novel function for arginine-glycine-glycine rich motifs?

C. Lorson, R. Morse, P. Eggleton, 2010, Journal of biochemistry.

Delivery of therapeutic agents through intracerebroventricular (ICV) and intravenous (IV) injection in mice.

C. Lorson, E. Osman, Tristan H. Coady, 2011, Journal of visualized experiments : JoVE.

Development of a Single Vector System that Enhances Trans-Splicing of SMN2 Transcripts

C. Lorson, M. Passini, Tristan H. Coady, 2008, PloS one.

Optimization of SMN Trans-Splicing Through the Analysis of SMN Introns

C. Lorson, M. Shababi, Monir Shababi, 2011, Journal of Molecular Neuroscience.

An exonic enhancer is required for inclusion of an essential exon in the SMA-determining gene SMN.

C. Lorson, E. Androphy, 2000, Human molecular genetics.

Therapeutics that directly increase SMN expression to treat spinal muscular atrophy.

C. Lorson, V. Mattis, M. Shababi, 2010, Drug news & perspectives.

AAV9-DOK7 gene therapy reduces disease severity in Smn2B/- SMA model mice.

C. Lorson, J. Marquez, E. Villalón, 2020, Biochemical and biophysical research communications.

An Sp1-binding site and TATA element are sufficient to support full transactivation by proximally bound NS1 protein of minute virus of mice.

C. Lorson, D. Pintel, J. Pearson, 1998, Virology.

Induced pluripotent stem cells from a spinal muscular atrophy patient

James A. Thomson, Clive N. Svendsen, C. Lorson, 2009, Nature.

RNA-targeting approaches for neuromuscular diseases.

C. Lorson, I. Richard, K. Charton, 2009, Trends in molecular medicine.

Evolution, correlation, structural impact and dynamics of emerging SARS-CoV-2 variants

C. Lorson, A. Sönnerborg, S. Byrareddy, 2021, Computational and Structural Biotechnology Journal.

Factors Associated with Emerging and Re-emerging of SARS-CoV-2 Variants

C. Lorson, A. Sönnerborg, S. Byrareddy, 2021, bioRxiv.

Regulation of murine survival motor neuron (Smn) protein levels by modifying Smn exon 7 splicing.

C. Lorson, E. Androphy, L. Simard, 2001, Human molecular genetics.

Evolutionary analysis of the Delta and Delta Plus variants of the SARS-CoV-2 viruses

C. Lorson, S. Byrareddy, T. Quinn, 2021, Journal of Autoimmunity.

Delivery of recombinant follistatin lessens disease severity in a mouse model of spinal muscular atrophy.

C. Lorson, V. Mattis, H. Rindt, 2009, Human molecular genetics.

Spinal muscular atrophy: a motor neuron disorder or a multi‐organ disease?

C. Lorson, S. Rudnik-Schöneborn, M. Shababi, 2014, Journal of anatomy.

ABT1 modifies SMARD1 pathology via interactions with IGHMBP2 and stimulation of ATPase and helicase activity

Sarah A. Hansen, C. Lorson, M. Lorson, 2022, JCI insight.

Transcription factor FOXL 2 protects granulosa cells from stress and delays cell cycle : role of its regulation by the SIRT 1 deacetylase

C. Lorson, E. Androphy, B. Benayoun, 2011 .

AAV9-Mediated Delivery of miR-23a Reduces Disease Severity in Smn2B-/SMA Model Mice.

C. Lorson, A. Ebert, H. Rindt, 2019, Human molecular genetics.

Astrocyte-produced miR-146a as a mediator of motor neuron loss in spinal muscular atrophy

C. Lorson, A. Ebert, Teresa N. Patitucci, 2017, Human molecular genetics.

Characterization of the minute virus of mice P38 core promoter elements

C. Lorson, D. Pintel, 1997, Journal of virology.

Trans cohort metabolic reprogramming towards glutaminolysis in long-term successfully treated HIV-infection

Gustavo Daniel Vega Magdaleno, C. Lorson, J. de Magalhães, 2022, Communications Biology.

Trans Cohorts Metabolomic Modulation Following Long-Term Successful Therapy in HIV-Infection

C. Lorson, J. de Magalhães, Á. Végvári, 2021 .

Infectivity of SARS-CoV-2: there Is Something More than D614G?

C. Lorson, A. Sönnerborg, S. Byrareddy, 2020, Journal of Neuroimmune Pharmacology.

Optimization of trans-Splicing for Huntington's Disease RNA Therapy

C. Lorson, V. Mattis, H. Rindt, 2017, Front. Neurosci..

Contributions of Different Cell Types to Spinal Muscular Atrophy Pathogenesis

C. Lorson, R. Kothary, A. Ebert, 2017 .

Disruption of the Survival Motor Neuron (SMN) gene in pigs using ssDNA

C. Lorson, L. Spate, C. Murphy, 2011, Transgenic Research.

Htra 2-b 1 stimulates an exonic splicing enhancer and can restore full-length SMN expression to survival motor neuron 2 ( SMN 2 )

S. Stamm, Y. Hofmann, C. Lorson, 2000 .

Functional characterization of SMN evolution in mouse models of SMA

C. Lorson, Y. Hao, G. Conant, 2019, Scientific Reports.

Intraperitoneal delivery of a novel drug‐like compound improves disease severity in severe and intermediate mouse models of Spinal Muscular Atrophy

C. Lorson, E. Androphy, R. Kline, 2019, Scientific Reports.

Discovery of a Small Molecule Probe That Post-Translationally Stabilizes the Survival Motor Neuron Protein for the Treatment of Spinal Muscular Atrophy.

C. Lorson, E. Androphy, M. Glicksman, 2017, Journal of medicinal chemistry.

Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy.

C. Lorson, A. Ebert, Teresa N. Patitucci, 2016, Molecular therapy : the journal of the American Society of Gene Therapy.

Defining the therapeutic window in a severe animal model of spinal muscular atrophy.

C. Lorson, E. Osman, M. Miller, 2014, Human molecular genetics.

Enhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug-like compounds

C. Lorson, E. Androphy, M. Glicksman, 2013, EMBO molecular medicine.

Development and characterization of an SMN2-based intermediate mouse model of Spinal Muscular Atrophy.

C. Lorson, H. Rindt, E. Osman, 2013, Human molecular genetics.

Development and characterization of an SMN2-based intermediate mouse model of Spinal Muscular Atrophy.

C. Lorson, H. Rindt, E. Osman, 2013, Human molecular genetics.

SMN-inducing compounds for the treatment of spinal muscular atrophy.

C. Lorson, M. Lorson, Monique A. Lorson, 2012, Future medicinal chemistry.

Direct central nervous system delivery provides enhanced protection following vector mediated gene replacement in a severe model of spinal muscular atrophy.

C. Lorson, Megan M. Krogman, M. Shababi, 2012, Biochemical and biophysical research communications.

Bifunctional RNAs targeting the intronic splicing silencer N1 increase SMN levels and reduce disease severity in an animal model of spinal muscular atrophy.

C. Lorson, E. Osman, Pei-Fen Yen, 2012, Molecular therapy : the journal of the American Society of Gene Therapy.

SMN in spinal muscular atrophy and snRNP biogenesis

C. Lorson, Tristan H. Coady, 2011, Wiley interdisciplinary reviews. RNA.

Cardiac defects contribute to the pathology of spinal muscular atrophy models.

C. Lorson, W. Sewell, J. Habibi, 2010, Human molecular genetics.

Delivery of a read-through inducing compound, TC007, lessens the severity of a spinal muscular atrophy animal model.

C. Lorson, V. Mattis, A. Ebert, 2009, Human molecular genetics.

Delivery of bifunctional RNAs that target an intronic repressor and increase SMN levels in an animal model of spinal muscular atrophy.

C. Lorson, E. Osman, Travis D. Baughan, 2009, Human molecular genetics.

Protein phosphatase 1 binds to the RNA recognition motif of several splicing factors and regulates alternative pre-mRNA processing.

S. Stamm, C. Lorson, C. Ben-Dov, 2008, Human molecular genetics.

Restoration of SMN function: delivery of a trans-splicing RNA re-directs SMN2 pre-mRNA splicing.

C. Lorson, G. Tullis, Tristan H. Coady, 2007, Molecular therapy : the journal of the American Society of Gene Therapy.

Survival Motor Neuron Function in Motor Axons Is Independent of Functions Required for Small Nuclear Ribonucleoprotein Biogenesis

C. Lorson, C. Beattie, G. Bassell, 2006, The Journal of Neuroscience.

Stimulating full-length SMN2 expression by delivering bifunctional RNAs via a viral vector.

C. Lorson, G. Tullis, Tristan H. Coady, 2006, Molecular therapy : the journal of the American Society of Gene Therapy.

SMN (Survival Motor Neuron) Gene and Protein

C. Lorson, E. Androphy, 2002 .

SMN oligomerization defect correlates with spinal muscular atrophy severity

C. Lorson, E. Androphy, B. Wirth, 1998, Nature Genetics.

Coronavirus helicases: attractive and unique targets of antiviral drug-development and therapeutic patents

C. Lorson, A. Sönnerborg, T. Quinn, 2021, Expert opinion on therapeutic patents.

Survival motor neuron protein in the nucleolus of mammalian neurons

C. Lorson, S. Baserga, Karen A. Wehner, 2002, Brain Research.

muscular atrophy Delivery of recombinant follistatin lessens disease severity in a mouse model of spinal including high-resolution figures

C. Lorson, F. F. Rose, Y. Qiu, 2008 .

A survival motor neuron:tetanus toxin fragment C fusion protein for the targeted delivery of SMN protein to neurons

C. Lorson, M. Remington, P. Fishman, 2004, Brain Research.

Combination of SMN trans-splicing and a neurotrophic factor increases the life span and body mass in a severe model of spinal muscular atrophy.

C. Lorson, M. Shababi, J. Glascock, 2011, Human gene therapy.

Mutations in the monkeypox virus replication complex: Potential contributing factors to the 2022 outbreak

C. Lorson, S. Byrareddy, S. Sachdev, 2022, Journal of autoimmunity.

Discovery and Evaluation of Entry Inhibitors for SARS-CoV-2 and Its Emerging Variants

C. Lorson, S. Byrareddy, J. Trivedi, 2021, Journal of virology.

An exonic enhancer is required for an essential exon in the SMA determining gene SMN

C. Lorson, E. Androphy, 2000 .

Spinal muscular atrophy: mechanisms and therapeutic strategies.

C. Lorson, H. Rindt, M. Shababi, 2010, Human molecular genetics.

Development of a novel severe mouse model of spinal muscular atrophy with respiratory distress type 1: FVB-nmd.

C. Lorson, E. Bryda, E. Villalón, 2019, Biochemical and biophysical research communications.

Optimization of SMN Trans-Splicing Through the Analysis of SMN Introns

C. Lorson, M. Shababi, 2012, Journal of Molecular Neuroscience.

Replacement of huntingtin exon 1 by trans-splicing

C. Lorson, H. Rindt, G. Weisman, 2012, Cellular and Molecular Life Sciences.

Identification of survival motor neuron as a transcriptional activator-binding protein.

C. Lorson, E. Androphy, J. Strasswimmer, 1999, Human molecular genetics.

Monitoring of recombinant survival motor neuron protein using fiber-optic surface plasmon resonance.

Jean-Francois Masson, Karl S Booksh, Tina M. Battaglia, 2004, The Analyst.

Astrocytes influence the severity of spinal muscular atrophy.

T. Crawford, C. Lorson, K. Swoboda, 2015, Human molecular genetics.

Selective vulnerability in neuronal populations in nmd/SMARD1 mice

C. Lorson, R. Kline, E. Villalón, 2018, Human molecular genetics.

Replacement of huntingtin exon 1 by trans-splicing

C. Lorson, H. Rindt, G. Weisman, 2012, Cellular and Molecular Life Sciences.

Potential therapeutic targets for Mpox: the evidence to date

C. Lorson, S. Byrareddy, S. Sachdev, 2023, Expert opinion on therapeutic targets.

Discovery and in-vitro evaluation of potent SARS-CoV-2 entry inhibitors

C. Lorson, S. Byrareddy, J. Trivedi, 2021, bioRxiv.

Decreasing disease severity in symptomatic, Smn(-/-);SMN2(+/+), spinal muscular atrophy mice following scAAV9-SMN delivery.

C. Lorson, E. Osman, Megan M. Krogman, 2012, Human gene therapy.

A Direct Comparison of IV and ICV Delivery Methods for Gene Replacement Therapy in a Mouse Model of SMARD1

C. Lorson, E. Villalón, Kevin A. Kaifer, 2018, Molecular therapy. Methods & clinical development.

Functional characterization of SMN evolution in mouse models of SMA

C. Lorson, Y. Hao, G. Conant, 2019, Scientific Reports.

Subcutaneous administration of TC007 reduces disease severity in an animal model of SMA

C. Lorson, V. Mattis, Cheng-Wei Chang, 2009 .

Distinct Metabolic Profile Associated with a Fatal Outcome in COVID-19 Patients during the Early Epidemic in Italy

C. Lorson, L. Baldini, A. Lashari, 2021, Microbiology spectrum.

A distinct metabolic profile associated with a fatal outcome in COVID-19 patients during early epidemic in Italy

L. N. Valenti, C. Lorson, L. Baldini, 2021, medRxiv.

The adeno-associated virus 2 (AAV2) genome and rep 68/78 proteins interact with cellular sites of DNA damage.

T. Joshi, C. Lorson, Juexin Wang, 2021, Human molecular genetics.

Induced pluripotent stem cells from a spinal muscular atrophy patient

C. Lorson, J. Thomson, Junying Yu, 2009, Nature.

Morpholino antisense oligonucleotides targeting intronic repressor Element1 improve phenotype in SMA mouse models.

C. Lorson, Abby Lombardi, Arleigh K Atkinson, 2014, Human molecular genetics.

An in vivo reporter system for measuring increased inclusion of exon 7 in SMN2 mRNA: potential therapy of SMA

C. Lorson, J. Zhou, E. Androphy, 2001, Gene Therapy.

Red cabbage juice-mediated gut microbiota modulation improves intestinal epithelial homeostasis and ameliorates colitis

Siu Hung Joshua Chan, C. Lorson, S. Batra, 2023, bioRxiv.

Defining the therapeutic window in a severe animal model of spinal muscular atrophy.

C. Lorson, J. Glascock, Kate L. Robbins, 2014, Human molecular genetics.