Christian L. Lorson PhD, University of Missouri - Columbia

Andrew
Christian L. Lorson PhD

The Lorson lab is interested in Spinal muscular atrophy (SMA) an inherited motor neuron disease that occurs in 1:6000 child births. Specifically the lab is studying survival motor neuron (SMN) genes by working on the following projects; Bi-functional RNAs delivered via a gene therapy vector, Trans-splicing delivered via a gene therapy vector and SMN-inducing compounds.

The Investigator's Annexe Part of The Investigator's Annexe program.

Products

References

  1. Coady TH, Baughan TD, Shababi M, Passini MA, Lorson CL. Development of a single vector system that enhances trans-splicing of SMN2 transcripts. PLoS One. 2008;3(10):e3468.
  2. Baughan T, Shababi M, Coady TH, Dickson AM, Tullis GE, Lorson CL. Stimulating full-length SMN2 expression by delivering bifunctional RNAs via a viral vector. Mol Ther. 2006 Jul;14(1):54-62.
  3. Mattis VB, Butchbach ME, Lorson CL. Detection of human survival motor neuron (SMN) protein in mice containing the SMN2 transgene: applicability to preclinical therapy development for spinal muscular atrophy. J Neurosci Methods. 2008 Oct 30;175(1):36-43.
  4. Wolstencroft EC, Mattis V, Bajer AA, Young PJ, Lorson CL. A non-sequence-specific requirement for SMN protein activity: the role of aminoglycosides in inducing elevated SMN protein levels. Hum Mol Genet. 2005 May 1;14(9):1199-210.
  5. Mattis VB, Ebert AD, Fosso MY, Chang CW, Lorson CL. Delivery of a read-through inducing compound, TC007, lessens the severity of a spinal muscular atrophy animal model. Hum Mol Genet. 2009 Oct 15;18(20):3906-13.
  6. Mattis VB, Bowerman M, Kothary R, Lorson CL. A SMNDelta7 read-through product confers functionality to the SMNDelta7 protein. Neurosci Lett. 2008 Sep 5;442(1):54-8.
  7. Wolstencroft EC1 Mattis V, Bajer AA, Young PJ, Lorson CL. A non-sequence-specific requirement for SMN protein activity: the role of aminoglycosides in inducing elevated SMN protein levels. Hum Mol Genet. 2005 May 1;14(9):1199-210.
  8. Mattis VB, Butchbach ME, Lorson CL. Detection of human survival motor neuron (SMN) protein in mice containing the SMN2 transgene: applicability to preclinical therapy development for spinal muscular atrophy. J Neurosci Methods. 2008 Oct 30;175(1):36-43.