조작된 산성 알파-글루코시다제 변이체

• Main Authors: ZHU YU, HUISMAN GJALT W, VROOM JONATHAN, ALAOUI ISMAILI MOULAY HICHAM, HALLOWS WILLIAM CASEY, HOMAN DAVID WILLIAM, CHNG CHINPING, BOTHAM RACHEL CATHLEEN, DELLAS NIKKI, SILVERMAN ADAM P, LAO JESSICA P
• Format: Patent
• Language: Korean
• Published: 23.09.2022
• 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; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS; SPIRITS; VINEGAR; WINE

본 발명은 조작된 산성 알파-글루코시다제(GAA) 폴리펩티드 및 그의 조성물을 제공한다. 일부 실시양태에서, 조작된 GAA 폴리펩티드는 증가된 발현, 중성 pH에서의 안정성, 및 세포 용해물 중 활성을 제공하도록 최적화된 것이다. 본 발명은 또한 치료 및 다른 목적을 위해 조작된 GAA 폴리펩티드를 포함하는 조성물을 사용하는 방법을 제공한다. The present invention provides engineered acid alpha-glucosidase (GAA) polypeptides and compositions thereof. In some embodiments, the engineered GAA polypeptides have been optimized to provide increased expression, stability at neutral pH, and activity in cell lysates. The invention also provides methods for utilization of the compositions comprising the engineered GAA polypeptides for therapeutic and other purposes.