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CRISPR-Cas9 and Genome Editing

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CRISPR-Cas9

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A molecular tool used to make precise changes in the DNA of organisms, from bacteria to human cells.

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Cas9

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An enzyme that cuts DNA at a specific location dictated by an RNA guide sequence.

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sgRNA (single-guide RNA)

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A synthetic RNA molecule designed to guide Cas9 to a specific location in the genome where a cut is intended.

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PAM (Protospacer Adjacent Motif)

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A short sequence of DNA recognized by Cas9 enzyme to initiate binding and create a double-strand break.

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NHEJ (Non-Homologous End Joining)

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A DNA repair mechanism that often leads to insertions or deletions when used with CRISPR-Cas9, potentially knocking out a gene.

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HDR (Homology-Directed Repair)

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A precise DNA repair mechanism used in conjunction with CRISPR-Cas9 when introducing specific mutations or inserting new genetic material.

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Knockout

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A genetic engineering process where CRISPR-Cas9 is used to disrupt the function of a gene, often by creating small insertions or deletions.

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Knock-in

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A process where CRISPR-Cas9 and HDR are used to insert a specific DNA sequence into a particular genomic location.

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Gene Drive

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A genetic engineering technique that promotes the inheritance of a particular gene or trait to increase its prevalence in a population.

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Off-target effects

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Unintended genetic modifications in non-target locations in the genome caused by CRISPR-Cas9 action.

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On-target efficiency

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The effectiveness with which CRISPR-Cas9 induces editing at the intended target site in the genome.

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Base Editors

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A class of CRISPR-based tools enabling the direct, irreversible conversion of one DNA base pair to another without introducing DNA breaks.

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Prime Editing

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A versatile and precise genome editing technique that uses a Cas9 nickase and a specially engineered reverse transcriptase.

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Cas12a (Cpf1)

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An alternative CRISPR protein to Cas9 that has distinct PAM requirements and produces staggered DNA cuts.

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dCas9 (dead Cas9)

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A modified form of Cas9 that still binds to DNA but no longer cuts, used in gene regulation applications.

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