Patent ID: 8927514
Filing Date: 2015-01-06
Classification: A61K,C07K,C12N

Abstract:
1. A method of treating a neurological disease or disorder in a subject, comprising systemically administering an AAV vector capable of crossing the blood-brain barrier to the subject, said vector comprising: (a) a recombinant nucleotide sequence which encodes an amino acid sequence of an AAV capsid or modified VP1, VP2, or VP3 portion thereof, wherein said amino acid sequence comprises SEQ ID NO:1 having one or more amino acid substitutions, and wherein the one or more amino acid substitutions comprise (i) an alanine (A) at position 346 in lieu of threonine (T), (ii) an arginine (R) at position 505 in lieu of a glycine (G), or (iii) both (i) and (ii); and (b) one or more therapeutic nucleotide sequences which encode one or more therapeutic agents for treating the neurological disease or disorder, wherein the one or more therapeutic nucleotide sequences are heterologous to the AAV vector; wherein the neurological disease or disorder is a disease or disorder of the brain; and wherein the vector is able to cross the intact blood-brain barrier and transduce a target cell or tissue in the brain with the one or more therapeutic nucleotide sequences, thereby delivering said therapeutic agent to treat the disease or disorder of the brain.