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V targets DNA; type VI targets RNA and type III targets both the DNA and
RNA (Samai et al., 2015; Koonin et al., 2017). In several organisms, Cas loci
do not fit into current classification due to their complex modular structure
and variability, e.g., target of Cas13d is not DNA, but RNA. Class 2 includes
Cas9, Cas12, and Cas13 endonucleases which are widely used as genome
editing tools (Wu et al., 2018).
11.3 MECHANISM OF ACTION OF CRISPR/Cas SYSTEMS
CRISPR/Cas system has a basis from the natural phenomena of bacterial
and archaeal adaptive immune system for invasion against external plasmids
or sequences of viruses (Marraffini & Sontheimer, 2010). CRISPR/Cas
editing system has two components which comprise Cas endonuclease and
single guide RNA (sgRNA). Cas-sgRNA complex binds to the target DNA
sequences. The sgRNA is made of CRISPR RNA (crRNA) with program
mable 20-nt RNA target sequences and trans-activating crRNA (tracrRNA).
The sgRNA recognizes and directs Cas protein to specific genomic target
(Hsu et al., 2014). Cas induces double-strand break (DSB) at the loci of
interest in the genome. The specificity of the system is due to 20 nt elon
gated replaceable guide sequence within sgRNA, complementary to the
target sequence (Li et al., 2013). The most generally used Cas protein is
Cas9 endonuclease derived from the gram-positive Streptococcus pyogenes
bacterium. Cas9 endonuclease cleavage is based on Protospacer Adjacent
Motif (PAM, 5’-NGG-3’) present directly downstream of 20-nt target DNA
sequence (Zhang et al., 2018) and PAM requirement is a bottleneck in it.
PAM sequence varies on the basis of type of CRISPR/Cas, e.g., in Cas9 vari
ants and Cas9 orthologs (Leenay & Beisel, 2017). The Cas9 endonuclease
cleaves at 3-nt upstream of PAM within DNA target site, generating blunt
ended double-strand breaks (DSBs) (Jiang & Doudna, 2017). CRISPR from
Prevotella and Francisella (Cpf1) known as Cas12a endonuclease of class 2
type V lacks HNH domain, needs T rich PAM and generates staggered cuts
for the plant genetic manipulations (Zetsche et al., 2015; Stella et al., 2017).
DSBs activated intrinsic DNA repair mechanism of the cell through NHEJ
or homology-directed repair (HDR) to generate gene knockout or gene
knock-in. HDR requires repair donor DNA template and leads to knock-in.
NHEJ is most dominant and active error prone DNA repair pathway and
generates insertions and deletions (indels) or substitutions resulting in the
gene knockout or gene silencing (Rong & Golic, 2000; Hasley et al., 2021).