Crispr Technology Glossary
21 terms total — 21 showing — Last updated 15 Sep 2026
IND IND
A US FDA regulatory application that must be filed before a novel CRISPR-based therapeutic can be administered to human subjects in clinical trials.
AAV
A small, non-enveloped single-stranded DNA virus widely used as a delivery vector for CRISPR components and gene therapy...
base editing
A CRISPR-derived technique that converts one DNA base to another at a targeted site without introducing a double-strand...
Cas12a
A class 2 CRISPR-associated endonuclease that recognises a T-rich PAM and produces staggered (cohesive) double-strand br...
Cas9
An RNA-guided endonuclease that creates double-strand breaks at targeted genomic loci; the most widely used nuclease in...
CRISPR
A family of DNA sequences in prokaryotes that form the basis of the CRISPR-Cas genome-editing system. In editorial conte...
CRISPRa
A CRISPR-based gene-activation approach that uses dCas9 fused to a transcriptional activator to upregulate gene expressi...
CRISPRi
A CRISPR-based gene-silencing approach that uses dCas9 fused to a transcriptional repressor to reduce gene expression wi...
dCas9
A catalytically inactive form of Cas9 that retains DNA-binding capability but lacks nuclease activity, used to recruit t...
electroporation
A physical delivery method in which brief electrical pulses create transient pores in the cell membrane, allowing CRISPR...
gRNA
A synthetic RNA molecule that directs the Cas nuclease to the correct genomic target sequence. May also appear as 'sgRNA...
HDR
A high-fidelity DNA repair pathway that uses a provided template to introduce precise edits at a Cas-induced break site....
IND
A US FDA regulatory application that must be filed before a novel CRISPR-based therapeutic can be administered to human...
indel
A small insertion or deletion of nucleotides at a genomic locus, typically resulting from error-prone NHEJ repair after...
knockin
The insertion of a specific exogenous DNA sequence into a defined genomic locus, typically via HDR-mediated repair of a...
knockout
The inactivation of a gene through deliberate disruption of its sequence, commonly achieved in CRISPR workflows via NHEJ...
NHEJ
An error-prone DNA repair pathway that ligates broken DNA ends without a homologous template, commonly producing inserti...
off-target effect
Unintended cleavage or modification at genomic sites other than the intended target sequence, a key safety and specifici...
PAM
A short DNA sequence immediately flanking the target site that is required for Cas nuclease recognition and cleavage. Al...
pegRNA
An engineered guide RNA used in prime editing that both targets the Cas9 nickase to the correct genomic location and enc...
prime editing
An advanced CRISPR-based genome-editing approach that uses a prime editor (PE) protein and a pegRNA to install precise e...
RNP
A complex of a Cas protein (such as Cas9) pre-assembled with its guide RNA, used as a delivery format for CRISPR editing...