Christian L. Lorson

Publications

• Partial Restoration of Cardio-vascular Defects in a Rescued Severe Model of Spinal Muscular Atrophy
  Journal of Molecular and Cellular Cardiology. Jan, 2012  |   Pubmed ID: 22285962
• 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. Jan, 2012  |   Pubmed ID: 22172949
• Bifunctional RNAs Targeting the Intronic Splicing Silencer N1 Increase SMN Levels and Reduce Disease Severity in an Animal Model of Spinal Muscular Atrophy
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Jan, 2012  |   Pubmed ID: 22031236
• Decreasing Disease Severity in Symptomatic, Smn(-/-);SMN2(+/+), Spinal Muscular Atrophy Mice Following ScAAV9-SMN Delivery
  Human Gene Therapy. Jan, 2012  |   Pubmed ID: 22029744
• Transgenic Inactivation of Murine Myostatin Does Not Decrease the Severity of Disease in a Model of Spinal Muscular Atrophy
  Neuromuscular Disorders : NMD. Nov, 2011  |   Pubmed ID: 22079083
• SMN in Spinal Muscular Atrophy and SnRNP Biogenesis
  Wiley Interdisciplinary Reviews. RNA. Jul-Aug, 2011  |   Pubmed ID: 21957043
• Optimization of SMN Trans-Splicing Through the Analysis of SMN Introns
  Journal of Molecular Neuroscience : MN. Aug, 2011  |   Pubmed ID: 21826391
• The Spinal Muscular Atrophy Mouse Model, SMAΔ7, Displays Altered Axonal Transport Without Global Neurofilament Alterations
  Acta Neuropathologica. Sep, 2011  |   Pubmed ID: 21681521
• Disruption of the Survival Motor Neuron (SMN) Gene in Pigs Using SsDNA
  Transgenic Research. Dec, 2011  |   Pubmed ID: 21350916
• The COPI Vesicle Complex Binds and Moves with Survival Motor Neuron Within Axons
  Human Molecular Genetics. May, 2011  |   Pubmed ID: 21300694
• 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. Feb, 2011  |   Pubmed ID: 20804424
• Therapeutics That Directly Increase SMN Expression to Treat Spinal Muscular Atrophy
  Drug News & Perspectives. Oct, 2010  |   Pubmed ID: 21031163
• Cardiac Defects Contribute to the Pathology of Spinal Muscular Atrophy Models
  Human Molecular Genetics. Oct, 2010  |   Pubmed ID: 20696672
• Spinal Muscular Atrophy: Mechanisms and Therapeutic Strategies
  Human Molecular Genetics. Apr, 2010  |   Pubmed ID: 20392710
• Identification of a Self-association Domain in the Ewing's Sarcoma Protein: a Novel Function for Arginine-glycine-glycine Rich Motifs?
  Journal of Biochemistry. Jun, 2010  |   Pubmed ID: 20211855
• 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. Jan, 2010  |   Pubmed ID: 20053895
• Effect of Diet on the Survival and Phenotype of a Mouse Model for Spinal Muscular Atrophy
  Biochemical and Biophysical Research Communications. Jan, 2010  |   Pubmed ID: 19945425
• Subcutaneous Administration of TC007 Reduces Disease Severity in an Animal Model of SMA
  BMC Neuroscience. 2009  |   Pubmed ID: 19948047
• RNA-targeting Approaches for Neuromuscular Diseases
  Trends in Molecular Medicine. Dec, 2009  |   Pubmed ID: 19906562
• Identification of a Tripartite Import Signal in the Ewing Sarcoma Protein (EWS)
  Biochemical and Biophysical Research Communications. Dec, 2009  |   Pubmed ID: 19879242
• Delivery of a Read-through Inducing Compound, TC007, Lessens the Severity of a Spinal Muscular Atrophy Animal Model
  Human Molecular Genetics. Oct, 2009  |   Pubmed ID: 19625298
• Delivery of Bifunctional RNAs That Target an Intronic Repressor and Increase SMN Levels in an Animal Model of Spinal Muscular Atrophy
  Human Molecular Genetics. May, 2009  |   Pubmed ID: 19228773
• Induced Pluripotent Stem Cells from a Spinal Muscular Atrophy Patient
  Nature. Jan, 2009  |   Pubmed ID: 19098894
• Delivery of Recombinant Follistatin Lessens Disease Severity in a Mouse Model of Spinal Muscular Atrophy
  Human Molecular Genetics. Mar, 2009  |   Pubmed ID: 19074460
• A Negatively Acting Bifunctional RNA Increases Survival Motor Neuron Both in Vitro and in Vivo
  Human Gene Therapy. Nov, 2008  |   Pubmed ID: 19848583
• Development of a Single Vector System That Enhances Trans-splicing of SMN2 Transcripts
  PloS One. 2008  |   Pubmed ID: 18941511
• 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. Oct, 2008  |   Pubmed ID: 18771690
• The Wallerian Degeneration Slow (Wld(s)) Gene Does Not Attenuate Disease in a Mouse Model of Spinal Muscular Atrophy
  Biochemical and Biophysical Research Communications. Oct, 2008  |   Pubmed ID: 18680723
• Identification and Characterisation of a Nuclear Localisation Signal in the SMN Associated Protein, Gemin4
  Biochemical and Biophysical Research Communications. Oct, 2008  |   Pubmed ID: 18675250
• 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. Aug, 2008  |   Pubmed ID: 18651653
• A SMNDelta7 Read-through Product Confers Functionality to the SMNDelta7 Protein
  Neuroscience Letters. Sep, 2008  |   Pubmed ID: 18601974
• Induction of Full-length Survival Motor Neuron by Polyphenol Botanical Compounds
  Human Genetics. Jan, 2008  |   Pubmed ID: 17962980
• Protein Phosphatase 1 Binds to the RNA Recognition Motif of Several Splicing Factors and Regulates Alternative Pre-mRNA Processing
  Human Molecular Genetics. Jan, 2008  |   Pubmed ID: 17913700
• 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. Aug, 2007  |   Pubmed ID: 17551501
• Novel Aminoglycosides Increase SMN Levels in Spinal Muscular Atrophy Fibroblasts
  Human Genetics. Nov, 2006  |   Pubmed ID: 16951947
• Stimulating Full-length SMN2 Expression by Delivering Bifunctional RNAs Via a Viral Vector
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Jul, 2006  |   Pubmed ID: 16580882
• A Non-sequence-specific Requirement for SMN Protein Activity: the Role of Aminoglycosides in Inducing Elevated SMN Protein Levels
  Human Molecular Genetics. May, 2005  |   Pubmed ID: 15790598
• 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. Apr, 2005  |   Pubmed ID: 15784894
• Monitoring of Recombinant Survival Motor Neuron Protein Using Fiber-optic Surface Plasmon Resonance
  The Analyst. Sep, 2004  |   Pubmed ID: 15343403
• A Survival Motor Neuron:tetanus Toxin Fragment C Fusion Protein for the Targeted Delivery of SMN Protein to Neurons
  Brain Research. Jan, 2004  |   Pubmed ID: 14644474
• The Ewing's Sarcoma Protein Interacts with the Tudor Domain of the Survival Motor Neuron Protein
  Brain Research. Molecular Brain Research. Nov, 2003  |   Pubmed ID: 14597228
• Survival Motor Neuron Protein in the Nucleolus of Mammalian Neurons
  Brain Research. Aug, 2002  |   Pubmed ID: 12126878
• Minute Virus of Mice Small Nonstructural Protein NS2 Interacts and Colocalizes with the Smn Protein
  Journal of Virology. Jun, 2002  |   Pubmed ID: 12021369
• Minute Virus of Mice NS1 Interacts with the SMN Protein, and They Colocalize in Novel Nuclear Bodies Induced by Parvovirus Infection
  Journal of Virology. Apr, 2002  |   Pubmed ID: 11907229
• SRp30c-dependent Stimulation of Survival Motor Neuron (SMN) Exon 7 Inclusion is Facilitated by a Direct Interaction with HTra2 Beta 1
  Human Molecular Genetics. Mar, 2002  |   Pubmed ID: 11875052
• A Direct Interaction Between the Survival Motor Neuron Protein and P53 and Its Relationship to Spinal Muscular Atrophy
  The Journal of Biological Chemistry. Jan, 2002  |   Pubmed ID: 11704667