Gene therapy

• Main Authors: HOLLOWOOD, COLLEEN, FENNELL THOMAS, GROENDAHL CHRISTIAN
• Format: Patent
• Language: Chinese; English
• Published: 05.01.2024
• 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

An AAV vector comprising a nucleotide sequence encoding factor I or factor H or a fragment or derivative thereof. The vector is useful for treating or preventing complement-mediated ocular conditions. 一种AAV载体，所述AAV载体包含编码因子I或因子H或者其片段或衍生物的核苷酸序列。该载体用于治疗或预防补体介导的眼部病症。