Means and methods for AAV gene therapy in humans

A method of treating a human disease comprising administering an AAV5 gene therapy vector to the central nervous system (CNS) of a human in need thereof,

• wherein the human is not subjected to a pre-screening with an assay to determine anti-AAV5 antibodies;
• and
• wherein the human has not been subjected to a medical treatment with an AAV5 gene therapy vector prior to the medical treatment.

A method of treating a human disease comprising administering an AAV5 gene therapy vector to the central nervous system (CNS) of a human in need thereof,

• wherein said human is subjected to a pre-screening with an assay to determine anti-AAV5 antibodies,
• wherein said human has not been subjected to a medical treatment with an AAV5 gene therapy vector prior to said medical treatment, said human having an anti-AAV5 antibody level of at most the 100th percentile of anti-AAV5 antibody levels as observed in the human population not before treated with an AAV5 vector, and
• wherein said human tested positive for anti-AAV5 antibodies.

A method for treating a human patient eligible for a medical treatment with an AAV5 gene therapy vector, the method comprising the steps of:

• (i) providing a serum sample from a human patient having Huntington's disease,
• (ii) determining the anti-AAV5 antibody titer, wherein the human patient is eligible for a medical treatment if (i) the total anti-AAV5 antibody titer has a value in the range of 0.02-5 as determined by a total anti-AAV5 (TAb) assay and/or (ii) the anti-AAV5 antibody titer has a value in the range of 3-5,000 as determined with a neutralizing anti-AAV5 antibody assay;
• (iii) administering the AAV5 gene therapy vector to the eligible human patient.