Moniemäksinen geeniterapia lentiviraalisesti toimitetuilla Cas9(D10A):lla ja HDR-tehostajilla

The accumulation of mutations is the primary generator of hereditary diseases as well as ostensibly much of the symptomatology of aging. Various methods for the therapeutic repair of mutations have been devised, but most have focused on the repair of known hereditary diseases of fixed location, with none having demonstrated reliable capacity to repair long segments of mutated DNA of unknown content, the likes of which arise in various loci as a result of natural aging, in adult mammals. This thesis attempts to address this by devising a gene editor capable of repairing multibase randomly-mutated segments, combined with a vehicle that can be used to deliver said gene editor to as many long-lived human cells in vivo as possible. The gene editor was designed successfully and tested with a fluorescent reporter cell line, but functional editing could not be observed in initial experiments. Reasons for this result are considered in the Discussion section.