Adeno-associated viral vectors for treatment of myofibrin (MYOC) glaucoma

• Main Authors: PECHAN PETER, SCARIA ABRAHAM, ARDINGER JEFFERY
• Format: Patent
• Language: Chinese; English
• Published: 15.08.2023
• 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; 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 present invention relates to adeno-associated virus vectors for the treatment of myofibrin (MYOC) glaucoma, and provides methods of using adeno-associated virus (AAV) vectors for the treatment of myofibrin (MYOC) glaucoma. In some aspects, the AAV vectors encode R-spinal protein 1 (RSPO1), R-spinal protein 2 (RSPO2), R-spinal protein 3 (RSPO3), or R-spinal protein 4 (RSPO4) and/or RNAi that targets myofibrin (MYOC). In one aspect, viral particles are administered to an eye of a human subject. Virus particles encoding RSPO1, RSPO2, RSPO3, and/or RSPO4, and/or MYOC RNAi are also contemplated. In some aspects, variant AAV2 particles that transduce trabecular meshwork are provided. 本发明涉及用于治疗肌纤蛋白(MYOC)青光眼的腺伴随病毒载体，并提供使用腺伴随病毒(AAV)载体用于治疗肌纤蛋白(MYOC)青光眼的方法。一些方面，AAV载体编码R-脊椎蛋白1(RSPO1)、R-脊椎蛋白2(RSPO2)、R-脊椎蛋白3(RSPO3)或R-脊椎蛋白4(RSPO4)和/或靶向肌纤蛋白(MYOC)的RNAi。一方面，将病毒颗粒施用至人受试者的眼部。编码RSPO1、RSPO2、RSPO3和/或RSPO4和/或MYOC RNAi的病毒颗粒也考虑在内。一些方面，提供了转导小梁网的变体AAV2颗粒。