Targeted retrograde gene delivery to motor neurons
AssigneeThe Salk Institute for Biological Studies
InventorsBrian K. Kaspar, Fred H. Gage
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Abstract
Methods are disclosed for delivering a heterologous gene to a cell body of a neuron by contacting a muscle tissue innervated by the neuron with a viral vector comprising a heterologous gene, wherein the viral vector enters said neuron and is retrogradely moved to the cell body. Additionally, methods for expressing secreted proteins from a nerve cell body as well as methods for treating neurodegenerative disorders such as amyotrophic lateral sclerosis are described.
Claims26
A method for delivering a heterologous gene to a cell body of a neuron, comprising:
- contacting a muscle tissue innervated by said neuron with a viral vector comprising a heterologous gene, wherein said viral vector enters said neuron and is retrogradely moved to the cell body.
A method for treating a neurodegenerative disease in a subject in need thereof, wherein said disease affects a target neuron, said method comprising:
- contacting a muscle tissue innervated by said target neuron with a viral vector comprising a heterologous gene, wherein said viral vector enters said neuron and is retrogradely moved to said cell body, and wherein expression of said heterologous gene results in a reduction in said neurodegenerative disease.
A method for expressing a protein that is secreted from a target neuron, said method comprising:
- contacting a muscle tissue innervated by said neuron with a viral vector comprising a heterologous gene encoding said protein, wherein said viral vector enters said neuron and is retrogradely moved to the cell body where said gene is expressed.
A method of treating a mammal having a neurodegenerative disease, said method comprising:
- selecting a muscle that participates in respiration in said mammal, wherein said muscle is innervated by a target neuron comprising a synaptic region and a cellular portion;
- and
- contacting a muscle that participates in respiration in said mammal with a viral vector comprising a heterologous gene, wherein said viral vector enters a synaptic region of said neuron and is retrogradely moved to the cell body of said target neuron wherein expression of said gene modulates respiration.
Dependencies are inferred from the claim text.