La guida definitiva all'editing genico.
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CRISPR-Cas9A protein that can be programmed with a short RNA guide to find one specific sequence in a genome and cut it. Approved Base EditingChemically converts one DNA letter into another at a chosen position, without cutting both strands of the double helix. Phase III Prime EditingWrites a new stretch of DNA sequence directly into a chosen site, using a guide that carries the replacement text with it. Phase I/II CRISPR-Cas12A family of CRISPR nucleases that cut DNA leaving staggered ends, use a different sequence requirement from Cas9, and can be smaller. Phase I/II CRISPR-Cas13Targets RNA rather than DNA, so it can silence a gene's message without touching the genome itself. Phase I Cas14 and miniature Cas systemsUnusually small CRISPR nucleases — roughly a third the size of Cas9 — discovered in archaea and bacteriophages. Discovery RNA EditingChanges letters in the RNA message rather than in the DNA, so the effect is real but temporary and the genome is untouched. Phase I/II Epigenetic EditingChanges how loudly a gene is expressed by rewriting the chemical marks on and around it, without altering a single letter of DNA sequence. Preclinical CRISPRa (gene activation)Uses a disabled CRISPR protein to switch a gene up rather than to cut it. Research tool CRISPRi (gene silencing)Parks a disabled CRISPR protein on a gene to block it from being read, turning it down without changing it. Research tool TALENsCustom-built proteins that recognise DNA letter by letter and cut — the technique CRISPR largely displaced, still in clinical use. Phase I/II Zinc Finger NucleasesThe first programmable gene-editing tool to reach patients — small, engineered proteins that grip DNA in three-letter blocks. Phase III Recombinases and integrasesEnzymes that cut and rejoin DNA at defined sites, able to insert whole genes rather than edit single letters. Preclinical TransposasesEnzymes that cut a segment of DNA out of one place and paste it into another — nature's own copy-and-paste. Phase I/II Gene Therapy (gene addition)Delivers a working copy of a gene into cells without changing the existing genome — the older approach that gene editing is often confused with. Approved RNA InterferenceUses the cell's own machinery to destroy a specific RNA message, lowering a protein's level without touching DNA. Approved Synthetic BiologyDesigning genetic parts and circuits so that cells perform new functions — the engineering discipline that gene editing serves as a tool. Phase III