METHOD OF PREDICTING THE LIKELIHOOD OF SUCCESS OF GENE THERAPY

摘要

A method of predicting the likelihood of success of a gene therapy procedure wherein the method involves inducing DNA damage in a cell sample from an individual and assessing the ability of the cell to repair the cell damage to determine whether the individual could tolerate DNA damage caused by a gene therapy vector. In preferred embodiments, the ability of the individuals cells to repair DNA damage is assessed by detecting, and monitoring the presence of a marker of DNA damage repair such as phosphorylated histone H2AX (gamma H2AX) or phosphorylated 53BP1 in the sample.