CRISPR-ERA for switching off (onco)genes

Abstract

[EN]Genome editing nucleases like the popular CRISPR/Cas9 allow generate knock - out cell lines and nulls zygotes by inducing site - specific DSB within a genome. In most cases, when a DNA template is not present, the DSB is repaired by non - homologous end joining (NHEJ) resulting in small nucleotide insertions or deletions that can be used to construct knockout alleles. However, for se veral reasons, these mutations do not produce the desired null result in all cases, generating a similar protein with functional activity. That undesirable effect could limit the therapeutic efficiency of gene therapy strategies focused on abrogating oncog ene expression by CRISPR/Cas9 and should be taken in account. This chapter reviews the irruption of CRISPR technology for gene silencing and its application in gene therapy.