先导编辑（prime editing）能做什么传统 CRISPR 做不到的事？
What can prime editing do that classic CRISPR cannot?
先导编辑用改造的 Cas9 和一段 pegRNA 直接写入新的 DNA 序列，可做点突变、小片段插入或删除，而不依赖 DNA 双链断裂，灵活度和精度更高。
Prime editing uses a modified Cas9 plus a pegRNA to write new DNA sequences—point mutations, small insertions, or deletions—without relying on double-strand breaks, offering more versatility and precision.
难度 专业 · 适合 大众
CC BY 4.0 · 已核验 · Verified 2026-11-04
来源 · Source
Sakuma,《From nuclease-based gene knock-in to prime editing – promising technologies of precision gene engineering》,Gene and Genome Editing,2022 (DOI: 10.1016/j.ggedit.2022.100017)
Sakuma, 'From nuclease-based gene knock-in to prime editing – promising technologies of precision gene engineering', Gene and Genome Editing, 2022, DOI: 10.1016/j.ggedit.2022.100017
独立核验依据 · Independent verification: 核验依据 1 Crossref DOI 10.1016/j.ggedit.2022.100017
#先导编辑 #基因编辑
碱基编辑（base editing）与 CRISPR 有何不同？
How does base editing differ from CRISPR?
传统 CRISPR 靠切断 DNA 双链来编辑，而碱基编辑不切断双链，直接用酶把单个碱基“改写”（如 C→T 或 A→G），更像精准的笔误修正，有望降低意外损伤的风险。
Classic CRISPR cuts both DNA strands to edit, while base editors do not cut them; they use an enzyme to directly rewrite a single base (e.g., C→T or A→G), like precise typo correction, potentially lowering unintended damage.
难度 专业 · 适合 大众
CC BY 4.0 · 已核验 · Verified 2026-10-04
来源 · Source
Komor AC et al. Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage. Nature (2016) 533:420-424
Komor AC et al. Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage. Nature (2016) 533:420-424
独立核验依据 · Independent verification: 核验依据 1 DOI 10.1038/nature17946
#碱基编辑 #基因编辑
mRNA 癌症疫苗的原理是什么？
How do mRNA cancer vaccines work?
这类疫苗把肿瘤特有的“新抗原”对应的 mRNA 注入体内，训练免疫系统识别并攻击表达这些抗原的癌细胞；目前多用于黑色素瘤等恶性肿瘤的个性化辅助治疗。
These vaccines inject mRNA encoding tumor-specific “neoantigens” so the immune system learns to recognize and attack cancer cells carrying them; they are studied mainly as personalized adjunct therapy in cancers like melanoma.
难度 专业 · 适合 小学低段(6-8)
CC BY 4.0 · 已核验 · Verified 2026-11-03
来源 · Source
Karikó、Weissman 等《RNA 修饰抑制 Toll 样受体激活》(Immunity, 2005)；Pardi、Weissman 等《mRNA 疫苗》(Nature Reviews Drug Discovery, 2018)；美国 CDC《新冠 mRNA 疫苗》
Karikó, Weissman et al., 'Suppression of RNA recognition by Toll-like receptors' (Immunity, 2005); Pardi, Hogan, Porter & Weissman, 'mRNA vaccines' (Nature Reviews Drug Discovery, 2018); US CDC, 'mRNA COVID-19 Vaccines'
独立核验依据 · Independent verification: 核验依据 1 CROSSREF 标识 10.1016/j.immuni.2005.06.008：Suppression of
#mRNA疫苗 #癌症免疫
诱导多能干细胞（iPSC）为什么重要？
Why are induced pluripotent stem cells (iPSCs) important?
iPSC 是把成年体细胞“重编程”回类似胚胎干细胞的状态，能再分化为多种细胞。它绕开了胚胎干细胞的伦理争议，并可用于个体化药物测试和疾病建模。
iPSCs are adult cells reprogrammed to an embryo-like state, able to become many cell types. They avoid embryo ethics concerns and enable personalized drug testing and disease modeling.
难度 专业 · 适合 小学高段(9-12)
CC BY 4.0 · 来源待核验 · Source pending review
来源 · Source
出处备注：著录含书名，但缺版次 / 出版社 / 年份中的一部分，需编辑补全后才能做独立比对。
Takahashi 与 Yamanaka《诱导多能干细胞》(Cell, 2006)；Yamanaka《iPS 细胞》(Nobel Lecture, 2012)；美国 NIH
Takahashi & Yamanaka, 'Induction of pluripotent stem cells' (Cell, 2006); Yamanaka, 'iPS cells' (Nobel Lecture, 2012); US NIH
#iPSC #干细胞
CRISPR 基因编辑是怎样“剪切”DNA 的？
How does CRISPR gene editing “cut” DNA?
CRISPR-Cas9 用一段引导 RNA 找到目标 DNA 序列，再由 Cas9 蛋白像分子剪刀一样切断 DNA 双链；细胞修复断口时可能引入或修正基因。它用于科研与作物改良，治疗应用仍在谨慎推进。
CRISPR-Cas9 uses a guide RNA to find a target DNA sequence, and the Cas9 protein cuts both strands like molecular scissors; cellular repair of the break can disrupt or correct a gene. It is used in research and crops, with therapeutic use advancing cautiously.
难度 进阶 · 适合 大众
CC BY 4.0 · 已核验 · Verified 2026-11-04
来源 · Source
Doudna 与 Charpentier《CRISPR-Cas9 的适应性免疫》(Science, 2012)；Jinek 等《可编程双链 DNA 切割》(Science, 2012)；美国 NIH
Doudna & Charpentier, 'A programmable dual-RNA guide' (Science, 2012); Jinek et al., 'Programmable dual-RNA–Cas9' (Science, 2012); US NIH
独立核验依据 · Independent verification: 核验依据 1 Crossref DOI 10.1126/science.1225829（复合著录按段核验：本标识只担保第二段
#CRISPR #基因编辑
什么是精准医疗（precision medicine）？
What is precision medicine?
精准医疗根据个体的基因、环境和生活方式来预防和治疗疾病，例如用基因检测结果选择更可能有效的靶向药，而不是“一刀切”的方案。
Precision medicine tailors prevention and treatment to an individual's genes, environment, and lifestyle—e.g., using genetic testing to pick targeted drugs more likely to work, rather than one-size-fits-all approaches.
难度 进阶 · 适合 大众
CC BY 4.0 · 来源待核验 · Source pending review
来源 · Source
出处备注：此条未指向带版次的可引用著作（通识性内容、机构文件或未展开的英文书名），本库不提供独立书目出处；如需精确引用，需由编辑补全具体篇目与版次。
Nature Medicine（精准肿瘤学）
Nature Medicine (precision oncology)
#精准医疗 #靶向治疗
什么是 CAR-T 细胞免疫疗法？
What is CAR-T cell immunotherapy?
CAR-T 是把患者自身的 T 细胞取出，在实验室装上识别癌细胞的“嵌合抗原受体”后再回输体内，让免疫细胞精准攻击肿瘤。它已用于某些白血病和淋巴瘤。
In CAR-T therapy, a patient's own T cells are collected, engineered in the lab to carry a chimeric antigen receptor that recognizes cancer cells, then infused back to attack tumors. It is used for some leukemias and lymphomas.
难度 进阶 · 适合 大众
CC BY 4.0 · 来源待核验 · Source pending review
来源 · Source
出处备注：著录含书名，但缺版次 / 出版社 / 年份中的一部分，需编辑补全后才能做独立比对。
June 等《CAR-T 细胞治疗》(NEJM, 2018)；Kalos 等《靶向 CD19 的 CAR-T 治疗白血病》(Science Translational Medicine, 2011)
June et al., 'CAR T-cell therapy' (NEJM, 2018); Kalos et al., 'CD19-targeted CAR T cells in leukemia' (Sci Transl Med, 2011)
#免疫疗法 #CAR-T
什么是 siRNA 和小干扰 RNA 药物？
What are siRNA and small interfering RNA drugs?
siRNA 是一小段 RNA，能与目标信使 RNA 结合并促其降解，从而在“翻译之前”关闭某个基因的表达。基于它的药物已用于治疗某些遗传性转甲状腺素蛋白淀粉样变性。
siRNA is a short RNA that binds a target messenger RNA and triggers its destruction, silencing a gene before it is translated into protein. Such drugs are used for some hereditary transthyretin amyloidosis.
难度 进阶 · 适合 小学低段(6-8)
CC BY 4.0 · 已核验 · Verified 2026-11-02
来源 · Source
Elbashir SM等．21核苷酸RNA双链介导培养哺乳动物细胞的RNA干扰．《自然》,2001；411(6836)：493-498
Elbashir SM, et al. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature. 2001;411(6836):493-498
独立核验依据 · Independent verification: 核验依据 1 CROSSREF 标识 10.1038/35078107：Duplexes of 21-nucleotide R
#siRNA #RNA干扰
药物基因组学如何影响用药？
How does pharmacogenomics influence medication use?
药物基因组学研究基因差异怎样改变人对药物的反应，例如某些人代谢特定药物过快或过慢，据此调整剂量可减少副作用、提高疗效。
Pharmacogenomics studies how genetic differences change drug response—e.g., some people metabolize a drug too fast or too slowly—so dosing can be adjusted to reduce side effects and improve efficacy.
难度 进阶 · 适合 初中(12-15)
CC BY 4.0 · 已核验 · Verified 2026-08-23 · 2 条独立证据
来源 · Source
杨宝峰 主编,《药理学》第9版,人民卫生出版社,2018
Yang Baofeng ed., Pharmacology, 9th ed., PMPH, 2018
独立核验依据 · Independent verification: 核验依据 2 ISBN 9787117266048（豆瓣书目条目: 药理学）
#药物基因组学 #精准医疗
什么是类器官（organoid）？
What is an organoid?
类器官是在实验室里用干细胞培养出的、能模拟真实器官结构和功能的小型三维组织，可用于药物筛选、发育研究和罕见病机制探索。
Organoids are tiny 3D tissues grown from stem cells that mimic the structure and function of real organs, used for drug screening, development studies, and rare-disease mechanisms.
难度 进阶 · 适合 小学低段(6-8)
CC BY 4.0 · 来源待核验 · Source pending review
来源 · Source
出处备注：本题原挂出处与答案内容不符，已撤回；具体书目待补，暂不展示来源。
本题原挂出处与答案内容不符，已撤回；具体书目待补。
Previous citation withdrawn as unsupported for this answer; a specific source is pending.
#类器官 #再生医学
什么是 mRNA 技术？它只能用来做疫苗吗？
What is mRNA technology, and is it only for vaccines?
mRNA（信使核糖核酸）是把遗传指令送到细胞里、让细胞临时生产特定蛋白的技术。除新冠疫苗外，它正被研究用于癌症疫苗、蛋白质替代疗法和罕见病治疗。
mRNA (messenger RNA) delivers genetic instructions that make cells temporarily produce a specific protein. Beyond COVID vaccines, it is being studied for cancer vaccines, protein-replacement therapy, and rare-disease treatment.
难度 入门 · 适合 大众
CC BY 4.0 · 已核验 · Verified 2026-11-03
来源 · Source
Karikó、Weissman 等《RNA 修饰抑制 Toll 样受体激活》(Immunity, 2005)；Pardi、Weissman 等《mRNA 疫苗》(Nature Reviews Drug Discovery, 2018)；美国 CDC《新冠 mRNA 疫苗》
Karikó, Weissman et al., 'Suppression of RNA recognition by Toll-like receptors' (Immunity, 2005); Pardi, Hogan, Porter & Weissman, 'mRNA vaccines' (Nature Reviews Drug Discovery, 2018); US CDC, 'mRNA COVID-19 Vaccines'
独立核验依据 · Independent verification: 核验依据 1 CROSSREF 标识 10.1016/j.immuni.2005.06.008：Suppression of
#mRNA #信使核糖核酸
什么是再生医学？
What is regenerative medicine?
再生医学试图用干细胞、组织工程和生物材料修复或替换受损的组织与器官，例如培养皮肤用于烧伤、用患者细胞造“类器官”来模拟疾病。
Regenerative medicine aims to repair or replace damaged tissues and organs using stem cells, tissue engineering, and biomaterials—e.g., lab-grown skin for burns, or “organoids” from a patient's cells to model disease.
难度 入门 · 适合 大众
CC BY 4.0 · 来源待核验 · Source pending review
来源 · Source
出处备注：著录含书名，但缺版次 / 出版社 / 年份中的一部分，需编辑补全后才能做独立比对。
Murphy & Atala，《组织与器官的3D生物打印》，《自然·生物技术》32卷，773–785页（2014）
Murphy, S.V. & Atala, A. '3D bioprinting of tissues and organs.' Nat. Biotechnol. 32, 773–785 (2014)
#再生医学 #干细胞