CRISPR Decoded

New to all of this从 DNA 是什么开始，循序渐进。无需任何背景知识。 A patient or family哪些已获批准，哪些仍处于试验阶段，以及如何加以区分。 关注行业动态谁在研发什么，处于哪个阶段。仅供参考，不构成任何建议。 关注食品与农业基因编辑农作物与牲畜，以及各国法规为何有所不同。

CRISPR-Cas9A protein that can be programmed with a short RNA guide to find one specific sequence in a genome and cut it. Hiroshi Nishimasu, F. Ann Ran,... · CC BY-SA 3.0 Base EditingChemically converts one DNA letter into another at a chosen position, without cutting both strands of the double helix. Prime EditingWrites a new stretch of DNA sequence directly into a chosen site, using a guide that carries the replacement text with it. CRISPR-Cas12A family of CRISPR nucleases that cut DNA leaving staggered ends, use a different sequence requirement from Cas9, and can be smaller. CRISPR-Cas13Targets RNA rather than DNA, so it can silence a gene's message without touching the genome itself. Cas14 and miniature Cas systemsUnusually small CRISPR nucleases, roughly a third the size of Cas9, discovered in archaea and bacteriophages. RNA EditingChanges letters in the RNA message rather than in the DNA, so the effect is real but temporary and the genome is untouched. 并排对比精准性、切割方式、载荷大小、递送方式、成熟度、风险

Sickle Cell DiseaseBlood disorders SpicyMilkBoy · CC BY-SA 4.0 Beta ThalassemiaBlood disorders Dr Graham Beards · CC BY-SA 4.0 ATTR AmyloidosisMetabolic disease Hereditary AngioedemaRare genetic disease LucyHAE · CC BY-SA 3.0 High Cholesterol and Familial HypercholesterolemiaCardiovascular disease CancerCancer Mikael Häggström, M.D. Author info... · CC0 Leukemia and LymphomaCancer https://wellcomeimages.org/indexplu... · CC BY 4.0 HIVInfectious disease Photo Credit: C. Goldsmith Content... · Public domain Duchenne Muscular DystrophyMuscular disease Dr. Edwin P. Ewing, Jr. · Public domain Cystic FibrosisRare genetic disease

CRSP CRISPR-Cas9, ex vivo and in vivo CRISPR TherapeuticsCRSP · Zug, Switzerland NTLA In vivo CRISPR-Cas9, lipid nanoparticle Intellia TherapeuticsNTLA · Cambridge, Massachusetts BEAM Base editing Beam TherapeuticsBEAM · Cambridge, Massachusetts EDIT In vivo CRISPR editing Editas MedicineEDIT · Cambridge, Massachusetts PRME Prime editing Prime MedicinePRME · Cambridge, Massachusetts VERV In vivo base editing Verve Therapeutics私有 · Boston, Massachusetts CRBU chRDNA hybrid guides, Cas12a Caribou BiosciencesCRBU · Berkeley, California SANA Hypoimmune cell engineering Sana BiotechnologySANA · Seattle, Washington

Jennifer DoudnaStructural biochemist who, with Emmanuelle Charpentier, showed that Cas9 could be programmed with a single guide RNA to cut chosen DNA, and shared the 2020 Nobel Prize in Chemistry for it. Christopher Michel · CC BY-SA 4.0 Emmanuelle CharpentierMicrobiologist who discovered tracrRNA, the missing component of the CRISPR-Cas9 system, and shared the 2020 Nobel Prize in Chemistry with Jennifer Doudna. Bianca Fioretti, Hallbauer & Fioretti · CC BY-SA 4.0 Feng ZhangBioengineer who led one of the first demonstrations of CRISPR genome editing in human cells and discovered several new CRISPR systems including Cas12a and Cas13. PuppyEggs · CC BY-SA 4.0 David LiuChemical biologist whose laboratory invented base editing and prime editing, the two techniques that changed gene editing from cutting to writing. ServiceAT · CC BY-SA 4.0 George ChurchGeneticist whose laboratory independently demonstrated CRISPR editing in human cells in 2013, and a founder of an unusually large number of genomics companies. Christopher Michel · CC BY-SA 4.0 Virginijus ŠikšnysLithuanian biochemist who independently showed that Cas9 could be programmed to cut DNA, publishing in 2012, and whose contribution is often omitted from popular accounts. Gie2016 · CC BY-SA 4.0

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Start here What is DNA?DNA is a long chemical chain that stores the instructions for building and running a living…

Start here What is a gene?A gene is a stretch of DNA that carries the instructions for making one particular thing the… WIC Start here What is a chromosome?A chromosome is one very long DNA molecule packaged with proteins; humans normally have 46, in… WIG Start here What is a genome?A genome is the complete set of genetic instructions in an organism: every chromosome, every… WIR Start here What is RNA?RNA is DNA's working copy: a temporary, single-stranded version the cell makes when it needs to… WIM Start here What is a mutation?A mutation is a change in DNA sequence. Most do nothing, some cause disease, and a few are…

背后的研究 里程碑论文以通俗语言摘要呈现，每篇均附原始发表链接。 基因编辑的历史 从DNA结构的发现到首个获批的CRISPR药物——带领我们走到今天的历史事件。

农业农作物、牲畜及各国不同的监管规则 伦理正反两方的论点 投资这个行业如何融资——仅供了解，不构成建议。 世界地图基因编辑在哪里发生