Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
AssigneesGenethon, Centre National De La Recherche Scientifique
InventorMartine Barkats
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Abstract
The present invention relates to compositions and methods, in particular to methods based on systemic injection of rAAV, for delivering genes to cells of the central nervous system in mammals, such as brain neurons or glial cells, and in particular to motor neurons or glial cells of the spinal cord The invention also relates to methods of treating motor neuron disorders in mammals by expression of therapeutic genes. The invention stems from the unexpected discovery that peripheral injection of AAV vectors leads to a bypass of the blood brain barrier and a massive infection of motor neurons. The invention may be used in any mammal, including human subjects.
Claims13
A method for delivering a transgene across the blood brain barrier to spinal cord motor neurons or glial cells of a human subject, and expressing said transgene, said method comprising intravenously administering, in the absence of a blood-brain barrier disrupting agent, to the subject from 1013 to 1017 viral genomes of a recombinant double stranded self-complementary human serotype 9 adeno-associated virus (scAAV) vector comprising an AAV9-derived capsid and a genome comprising the transgene, such that the scAAV vector crosses the blood brain barrier and spinal cord motor neuron or glial cells of said subject are transduced and express said transgene.
Dependencies are inferred from the claim text.