Method for reducing editing window of base editor, base editor and application

• Main Authors: BI CHANGHAO, JIANG GUO, ZHAO DONGDONG, CHEN XUXU, ZHANG XUELI
• Format: Patent
• Language: Chinese; English
• Published: 03.02.2023
• Subjects: BEER; BIOCHEMISTRY; CHEMISTRY; COMPOSITIONS THEREOF; CULTURE MEDIA; ENZYMOLOGY; HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPIRITS; VINEGAR; WINE

The invention belongs to the technical field of biotechnology and gene editing, and particularly relates to a gRNA mutant construction method, a gRNA mutant, a method for reducing an editing window of a base editor, the base editor, separated polynucleotide, a recombinant expression vector, a recombinant host cell, the base editor, a composition and application. And a method for gene editing in a cell or subject, and a method for treating or preventing a disease. When the gRNA mutant is applied to a base editor, a base editing window can be universally narrowed, the specificity of gene editing is improved, specific editing of one base is realized, and the gRNA mutant has a wide application prospect in the aspects of gene therapy, drug screening, animal and plant model construction and the like. 本公开属于生物技术和基因编辑技术领域，具体涉及一种gRNA突变体的构建方法、gRNA突变体、缩小碱基编辑器的编辑窗口的方法、碱基编辑器、分离的多核苷酸、重组表达载体、重组宿主细胞、碱基编辑器、组合物及用途，以及在细胞或受试者中进行基因编辑的方法、治疗或预防疾病的方法。gRNA突变体应用于碱基编辑器中，能够通用性地缩小碱基编辑窗口，提高了基因编辑的特异性，实现对1个碱基的特异性编辑，在基因治疗、药物筛选、动植物模型构建等方面具有广阔的