Mutant of adeno-associated virus capsid protein, nucleic acid for coding mutant and application of mutant

• Main Authors: PAN OUDONG, YANG JIALI, SHEN WEIRAN, YANG XINGLIN, CAI ZEQI, NIU LELE
• Format: Patent
• Language: Chinese; English
• Published: 15.11.2024
• 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; ORGANIC CHEMISTRY; PEPTIDES; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS; SPIRITS; VINEGAR; WINE

The invention discloses a mutant of adeno-associated virus capsid protein, nucleic acid for encoding the mutant and application of the mutant. The mutant of the capsid protein of the adeno-associated virus has amino acid mutation at one or more of the 1301 < th > site, the 1708 < th > site and the 1711 < th > site relative to the capsid protein of a parent AAV9. Virus particles prepared by using the mutant of adeno-associated virus capsid protein give consideration to both transduction efficiency and targeting, and can provide a safer and more effective gene therapy vector tool for subjects. 本申请公开了一种腺相关病毒衣壳蛋白的突变体、编码其的核酸及应用。该腺相关病毒衣壳蛋白的突变体相对于亲本AAV9的衣壳蛋白在第1301位、第1708位和第1711位中的一位或多位具有氨基酸突变。使用该腺相关病毒衣壳蛋白的突变体制备得到的病毒颗粒，兼顾了转导效率和靶向性，能够为受试者提供更安全、更有效的基因治疗载体工具。