Recombinant adeno-associated vectors for targeted treatment
AssigneeCity of Hope
InventorsSaswati CHATTERJEE, Laura SMITH, Kamehameha WONG
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AssigneeCity of Hope
InventorsSaswati CHATTERJEE, Laura SMITH, Kamehameha WONG
Estimating the term…
Novel adeno-associated virus (AAV) vectors in nucleotide and amino acid forms and uses thereof are provided. The isolates show specific tropism for certain target tissues, such as blood stem cells, liver, heart and joint tissue, and may be used to transduce stem cells for introduction of genes of interest into the target tissues. Certain of the vectors are able to cross tightly controlled biological junctions, such as the blood-brain barrier, which open up additional novel uses and target organs for the vectors, providing for additional methods of gene therapy and drug delivery.
A method of treating a neurological disease or disorder in a subject, comprising administering a vector capable of crossing the blood-brain barrier, blood-ocular barrier, or blood-nerve barrier, said vector comprising one or more therapeutic nucleotide sequences for treating the neurological disease or disorder.
A method of treating a disease or disorder in a subject, comprising administering a vector comprising one or more therapeutic nucleotide sequences selected from the following:
A method of treating a brain or muscle disease or disorder in a subject, comprising administering a vector of AAVHSC15 comprising a therapeutic nucleotide sequence for treating the brain or heart disease or disorder.
Dependencies are inferred from the claim text.