SG11201700724PA - High isomerohydrolase activity mutants of mammalian rpe65 - Google Patents

High isomerohydrolase activity mutants of mammalian rpe65

Info

Publication number
SG11201700724PA
SG11201700724PA SG11201700724PA SG11201700724PA SG11201700724PA SG 11201700724P A SG11201700724P A SG 11201700724PA SG 11201700724P A SG11201700724P A SG 11201700724PA SG 11201700724P A SG11201700724P A SG 11201700724PA SG 11201700724P A SG11201700724P A SG 11201700724PA
Authority
SG
Singapore
Prior art keywords
activity mutants
isomerohydrolase activity
mammalian rpe65
rpe65
mammalian
Prior art date
Application number
SG11201700724PA
Inventor
Jian-Xing Ma
Yusuke Takahashi
Gennadiy Moiseyev
Original Assignee
Univ Oklahoma
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Univ Oklahoma filed Critical Univ Oklahoma
Publication of SG11201700724PA publication Critical patent/SG11201700724PA/en

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/18—Carboxylic ester hydrolases (3.1.1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/01—Carboxylic ester hydrolases (3.1.1)
    • C12Y301/01064—Retinoid isomerohydrolase (3.1.1.64)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
SG11201700724PA 2014-07-31 2015-07-27 High isomerohydrolase activity mutants of mammalian rpe65 SG11201700724PA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201462031472P 2014-07-31 2014-07-31
PCT/US2015/042263 WO2016018816A1 (en) 2014-07-31 2015-07-27 High isomerohydrolase activity mutants of mammalian rpe65

Publications (1)

Publication Number Publication Date
SG11201700724PA true SG11201700724PA (en) 2017-02-27

Family

ID=55218217

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11201700724PA SG11201700724PA (en) 2014-07-31 2015-07-27 High isomerohydrolase activity mutants of mammalian rpe65

Country Status (10)

Country Link
US (1) US10494619B2 (en)
EP (1) EP3188762A4 (en)
JP (1) JP2017522895A (en)
KR (1) KR20170036085A (en)
CN (1) CN106794262A (en)
AU (1) AU2015296861A1 (en)
BR (1) BR112017001909A2 (en)
CA (1) CA2956588A1 (en)
SG (1) SG11201700724PA (en)
WO (1) WO2016018816A1 (en)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107287238B (en) * 2016-04-11 2020-10-16 厦门继景生物技术有限责任公司 Gene vector and gene therapy medicine for treating Leber congenital amaurosis 2 type disease
AU2017258776B2 (en) * 2016-04-28 2023-07-13 Spark Therapeutics, Inc. Relative potency assay for viral vector encoding isomerohydrolases
GB2595197B (en) 2017-04-21 2022-02-23 Zenimax Media Inc Systems and methods for encoder-guided adaptive-quality rendering
US20210254037A1 (en) * 2018-06-14 2021-08-19 Avixgen Inc. Pharmaceutical composition comprising fusion protein of cell-penetrating peptide and rpe65 for treatment of leber's congenital amaurosis
CN111118017B (en) * 2018-11-01 2023-10-03 上海朗昇生物科技有限公司 Carrier for treating Leber congenital amaurosis and application thereof
US20220267777A1 (en) * 2019-07-10 2022-08-25 Emendobio Inc. Differential knockout of a heterozygous allele of rpe65
US20240060989A1 (en) 2019-10-04 2024-02-22 Novartis Ag Methods for measuring cralbp activity
CN111876418B (en) * 2020-07-17 2024-02-02 北京希诺因生物科技有限公司 Method for establishing congenital black Mongolian model dogs

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US4394448A (en) 1978-02-24 1983-07-19 Szoka Jr Francis C Method of inserting DNA into living cells
US5288641A (en) 1984-06-04 1994-02-22 Arch Development Corporation Herpes Simplex virus as a vector
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5665577A (en) 1989-02-06 1997-09-09 Dana-Farber Cancer Institute Vectors containing HIV packaging sequences, packaging defective HIV vectors, and uses thereof
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US5676954A (en) 1989-11-03 1997-10-14 Vanderbilt University Method of in vivo delivery of functioning foreign genes
US5672510A (en) 1990-01-19 1997-09-30 Genetic Therapy, Inc. Retroviral vectors
US5817491A (en) 1990-09-21 1998-10-06 The Regents Of The University Of California VSV G pseusdotyped retroviral vectors
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WO1996018418A1 (en) 1994-12-12 1996-06-20 Genetic Therapy, Inc. Improved adenoviral vectors and producer cells
US5707865A (en) 1994-12-21 1998-01-13 Kohn; Donald B. Retroviral vectors for expression in embryonic cells
US5622856A (en) 1995-08-03 1997-04-22 Avigen High efficiency helper system for AAV vector production
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
ZA969435B (en) 1995-11-16 1997-06-02 Antonomous University Of Barce Treatment of diabetes with a glucokinase gene
IT237434Y1 (en) 1997-04-17 2000-09-13 Philco Italiana WASHING MACHINE WITH TROLLEY FOR ITS MOVEMENT
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
CN1313873A (en) 1998-07-13 2001-09-19 表达遗传学公司 Polyester analogus of poly-L-Lysine as a soluble, biodegradable gene delivery carrier
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CA2833870C (en) * 2011-04-22 2020-03-10 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof

Also Published As

Publication number Publication date
WO2016018816A1 (en) 2016-02-04
EP3188762A4 (en) 2018-01-10
AU2015296861A1 (en) 2017-02-16
BR112017001909A2 (en) 2019-09-17
US10494619B2 (en) 2019-12-03
CN106794262A (en) 2017-05-31
US20170226490A1 (en) 2017-08-10
CA2956588A1 (en) 2016-02-04
EP3188762A1 (en) 2017-07-12
JP2017522895A (en) 2017-08-17
KR20170036085A (en) 2017-03-31

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