High isomerohydrolase activity mutants of mammalian rpe65

• Main Authors: MA JIAN-XING, TAKAHASHI YUSUKE, MOISEYEV GENNADIY
• Format: Patent
• Language: Chinese; English
• Published: 31.05.2017
• Subjects: AGRICULTURE; ANIMAL HUSBANDRY; BEER; BIOCHEMISTRY; BIOCIDES, e.g. AS DISINFECTANTS, AS PESTICIDES, AS HERBICIDES; CHEMISTRY; COMPOSITIONS THEREOF; CULTURE MEDIA; ENZYMES; ENZYMOLOGY; FISHING; FORESTRY; HUMAN NECESSITIES; HUNTING; HYGIENE; MEDICAL OR VETERINARY SCIENCE; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; PEST REPELLANTS OR ATTRACTANTS; PLANT GROWTH REGULATORS; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PRESERVATION OF BODIES OF HUMANS OR ANIMALS OR PLANTS OR PARTSTHEREOF; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPIRITS; TRAPPING; VINEGAR; WINE

Mutant mammalian RPE65 proteins and portions thereof, and nucleic acids encoding the mutants, for use in treating a condition related to retinal degeneration in a subject, the mutant mammalian RPE65 proteins or portions thereof having isomerohydrolase activity. A gene therapy method of treating a condition related to retinal degeneration in a mammalian subject in need of such treatment, comprising administering to the subject a therapeutically- effective amount of a vector comprising a nucleic acid encoding a mutant mammalian RPE65 protein or a portion thereof. A method of treating a condition related to retinal degeneration in a subject in need of such treatment, comprising administering to the subject a therapeutically-effective amount of at least one of a mutant mammalian RPE65 protein or a portion thereof. 突变的哺乳动物RPE65蛋白及其部分以及编码突变体的核酸，用于在受试者中治疗与视网膜变性相关的病症，该突变的哺乳动物RPE65蛋白或其部分具有异构水解酶活性。在需要这种治疗的哺乳动物受试者中治疗与视网膜变性相关的病症的基因治疗方法，包括向所述受试者施用治疗有效量的包括编码突变的哺乳动物RPE65蛋白或其部分的核酸的载体。在需要这种治疗的受试者中治疗与视网膜变性相关的病症的方法，包括向所述受试者施用治疗