Исчерпывающий путеводитель по редактированию генов.
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the crystal structure of Streptococcus pyogenes Cas9 in complex with sgRNA and its target DNA at 2.5 A ˚ resolution CRISPR-Cas9A protein that can be programmed with a short RNA guide to find one specific sequence in a genome and cut it. Approved A DNA helix with one letter being chemically changed Base EditingChemically converts one DNA letter into another at a chosen position, without cutting both strands of the double helix. Phase III A prime editor writing new letters into a DNA strand 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 A Cas12 enzyme on DNA, scattering fragments 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 A Cas13 enzyme cutting a strand of RNA CRISPR-Cas13Targets RNA rather than DNA, so it can silence a gene's message without touching the genome itself. Phase I A tiny Cas14 enzyme bound to a DNA helix Cas14 and miniature Cas systemsUnusually small CRISPR nucleases, roughly a third the size of Cas9, discovered in archaea and bacteriophages. Discovery An enzyme changing a letter on a strand of RNA 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 A DNA helix studded with glowing chemical tags 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 A deactivated Cas9 on DNA beaming light to switch a gene on CRISPRa (gene activation)Uses a disabled CRISPR protein to switch a gene up rather than to cut it. Research tool A deactivated Cas9 sitting on a DNA strand, blocking it 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 TALEN protein arms gripping a DNA helix 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 proteins binding along a DNA helix Zinc Finger NucleasesThe first programmable gene-editing tool to reach patients: small, engineered proteins that grip DNA in three-letter blocks. Phase III A recombinase enzyme swapping DNA segments Recombinases and integrasesEnzymes that cut and rejoin DNA at defined sites, able to insert whole genes rather than edit single letters. Preclinical A transposase moving a DNA segment to a new place 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 A viral capsid delivering a gene into a cell Gene Therapy (gene addition)Delivers a working copy of a gene into cells without changing the existing genome. It is the older approach that gene editing is often confused with. Approved An RNA-guided complex unwinding a strand of RNA RNA InterferenceUses the cell's own machinery to destroy a specific RNA message, lowering a protein's level without touching DNA. Approved Modular components being assembled on a bench Synthetic BiologyDesigning genetic parts and circuits so that cells perform new functions: the engineering discipline that gene editing serves as a tool. Phase III