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Enhancer Restricts Transgene Expression to Motor Neurons for Improved Motor Neuron-Targeted Gene Therapy

Detailed Technology Description
This invention describes a DNA construct for gene expression in spinal motor neurons enabling gene therapy specific for motor neuron diseases.
Others

U.S. 2005/0129672

Identification of a conserved 125 base-pair Hb9 enhancer that specifies gene expression to spinal motor neurons. Dev. Biol. 293(2):474-85.
Telomerase immortalization of neuronally restricted progenitor cells derived from the human fetal spinal cord. Nature Biotech 22(3):297-305.
Enhancer-specified GFP-based FACS purification of human spinal motor neurons from embryonic stem cells. Exp Neurol. 196(2):224-34.

Human embryonic stem cell-derived motor neurons expressing SOD1 mutants exhibit typical signs of motor neuron degeneration linked to ALS. Dis. Model Mech. 2:189-95, 2009.
Directed differentiation of human induced pluripotent stem cells (iPS) generates active motor neurons. Stem Cells 27:806-811, 2009.
*Abstract

The homeobox gene Hb9 is selectively expressed by motor neurons in the developing and mature central nervous system. The inventors identified a conserved regulatory element (enhancer) in the Hb9 promoter region that is sufficient to mediate expression of a gene of interest specifically in motor neurons. It has been demonstrated both in vitro and in a mouse model in vivo that positioning a transgene coding sequence behind this short enhancer sequence leads to selective expression of the transgene in motor neurons. Thus, constructs employing the enhancer can be used in gene therapy for motor neuron diseases. Moreover, the invention employing a reporter gene can be used to isolate motor neurons from a mixed population of human brain and/or spinal cells as well as to follow the differentiation of human embryonic stem cells or induced pluripotential stem cells inducible stem cells into motor neurons.

 

Potential Applications

  • Gene therapy of motor neuron diseases such as amyotrophic lateral sclerosis (ALS) or spinal muscular atrophy (SMA)
  • Track or maximize the differentiation of stem cells into motor neurons
  • Permits patient and disease-specific motor neurons to be identified and isolated for study

 

Advantages

  • Enables smaller constructs for targeting gene expression to motor neurons
  • Limiting transgene expression to motor neurons enables safer gene therapy
*Licensing
Dan-Oscar Antsonda429@cornell.edu212-746-1297
Country/Region
USA

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