ABSTRACT
Gene therapy involves the introduction of exogenous nucleic acids into target patient’s cells to treat diseases linked to aberrant gene expression patterns. The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas9) adaptive immunity system was repurposed to edit eukaryotic genomes in order to correct and/or replace defective genes associated with disease and disorders. However, several technical limitations hinder the use of the CRISPR-Cas9 genome editing system for gene therapy in humans. This chapter summarizes these limitations and provides several showcase examples that may help to better understand how CRISPR-Cas9 strategies can be used for therapeutic purposes.
