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CRISPR & Genome Editing Glossary

Master CRISPR-Cas genome editing vocabulary — from Cas nucleases and guide RNA design to base editing and prime editing.

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CRISPR

Clustered Regularly Interspaced Short Palindromic Repeats — a bacterial immune system adapted for precise genome editing.

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CRISPR Screening

Genome-wide loss- or gain-of-function screens using CRISPR libraries to identify genes driving phenotypes.

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Cas9

The CRISPR-associated nuclease that creates double-strand breaks at target sites directed by a guide RNA.

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CRISPR-Cas12

A CRISPR nuclease (Cpf1) that cuts DNA with staggered ends and recognizes T-rich PAM sequences, expanding target range.

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CRISPR-Cas13

An RNA-targeting CRISPR nuclease that cleaves single-stranded RNA, enabling transcriptome editing and diagnostics.

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Genome Editing

Technologies that make precise changes to DNA sequences in living organisms, including insertions, deletions, and replacements.

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Base Editing

CRISPR-derived technique that converts one DNA base to another (e.g., C→T or A→G) without double-strand breaks.

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Prime Editing

A versatile editing method using a Cas9-reverse transcriptase fusion and pegRNA to make all 12 possible point mutations plus insertions/deletions.

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RNA Interference

A biological pathway where small RNA molecules (siRNA, miRNA) silence gene expression by degrading mRNA or blocking translation.

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Gene Therapy

The therapeutic delivery of genetic material into cells to treat or prevent disease by correcting defective genes.

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Gene

A segment of DNA that encodes a functional product (protein or RNA) and is the basic unit of heredity.

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Gene Expression

The process by which information from a gene is used to synthesize functional products, primarily proteins.

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Exon

A coding region of a gene that is retained in the final mature mRNA and typically translated into protein.

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Intron

A non-coding intervening sequence removed by RNA splicing during mRNA maturation.

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Codon

A three-nucleotide sequence in mRNA that specifies a particular amino acid or a stop signal during translation.

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mRNA

Messenger RNA — the intermediate molecule that carries protein-coding information from DNA to the ribosome.

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RNA

Ribonucleic acid — a versatile nucleic acid involved in gene regulation, protein synthesis, and genome editing.

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tRNA

Transfer RNA — adaptor molecules that carry specific amino acids to the ribosome during protein synthesis.

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Transcription

The process of copying a DNA sequence into an RNA molecule by RNA polymerase.

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Translation

The ribosome-mediated synthesis of a polypeptide chain from the information encoded in mRNA.

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Promoter

A DNA region upstream of a gene where RNA polymerase and transcription factors bind to initiate transcription.

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Enhancer

A regulatory DNA element that increases gene transcription, often located far from the target gene.

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Mutation

A permanent alteration in the DNA sequence that can affect gene function and may be harmless, beneficial, or harmful.

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Allele

One of two or more alternative forms of a gene that arise by mutation and occupy the same chromosomal locus.

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Genotype

The genetic constitution of an organism, including all alleles present at specific loci.

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Phenotype

The observable characteristics of an organism resulting from the interaction of its genotype with the environment.

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