工具
对比技术
选择两到三种方法，从精准性、DNA 切割方式、载荷大小、递送方式、临床成熟度和已知风险等方面了解它们的差异。
请在上方选择两种技术，或阅读下方的文字比较。
文字对比
人们最常混淆的概念，在这里得到清晰解答。
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