Targeted retrograde gene delivery for neuronal protection
AssigneeThe Salk Institute for Biological Studies
InventorsBrian K. Kaspar, Fred H. Gage, Daniel A. Peterson
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Abstract
Methods are disclosed for transducing neurons with heterologous genes using retrograde viral transport. The methods disclosed employ substantially non-toxic vectors, such as adeno-associated virus vectors, that are capable of retrograde axonal transport to introduce and express genes in the neurons. This method has applications in the mapping of neural pathways, in stimulating or inhibiting the growth of neurons, and in the treatment of various neurodegenerative diseases such as Alzheimer's disease, Parkinson's disease, and amyotrophic lateral sclerosis.
Claims27
A method for transducing a neuron with a heterologous gene, wherein said neuron has a synaptic portion and a cellular portion, comprising:
- providing a viral vector comprising a heterologous gene to be transduced into a neuron;
- and
- contacting the synaptic portion of said neuron with said viral vector under conditions whereby said contacting results in transduction of the viral vector into said synaptic portion, and retrograde movement of said viral vector from the synaptic portion to the cellular portion, wherein said heterologous gene is incorporated into the genome of the neuron.
A method for increasing the proliferation of a nerve cell, wherein said nerve cell has a synaptic portion and a cellular portion, comprising:
- providing a viral vector comprising a growth factor gene to be transduced into said nerve cell;
- contacting the synaptic end of said nerve cell with said viral vector under conditions whereby said contacting results in transduction of the viral vector into said synaptic end, and retrograde movement of said viral vector from the synaptic end to the cellular end of said nerve cell;
- and
- incubating said nerve cell under conditions whereby said growth factor gene is expressed by said nerve cell.
A method for treating a neurodegenerative disease in a human, comprising:
- identifying a human patient in need of treatment for said neurodegenerative disease;
- providing a viral vector comprising a therapeutic gene to be transduced into a synaptic end of said target neurons of said patient;
- and
- introducing said viral vector into a terminal field of said target neurons of said patient under conditions whereby said contacting results in transduction of the viral vector into the synaptic end of said target neurons, and wherein said viral vectors migrate from the synaptic end to the cellular end of said target neurons.
Dependencies are inferred from the claim text.