PE20190393A1 - Ensayo de potencia relativa para un vector viral que codifica isomero hidrolasas - Google Patents

Ensayo de potencia relativa para un vector viral que codifica isomero hidrolasas

Info

Publication number
PE20190393A1
PE20190393A1 PE2018002238A PE2018002238A PE20190393A1 PE 20190393 A1 PE20190393 A1 PE 20190393A1 PE 2018002238 A PE2018002238 A PE 2018002238A PE 2018002238 A PE2018002238 A PE 2018002238A PE 20190393 A1 PE20190393 A1 PE 20190393A1
Authority
PE
Peru
Prior art keywords
isomer
protein
reaction product
hydrolases
viral vector
Prior art date
Application number
PE2018002238A
Other languages
English (en)
Inventor
Linda Couto
Original Assignee
Spark Therapeutics Inc
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 Spark Therapeutics Inc filed Critical Spark Therapeutics Inc
Publication of PE20190393A1 publication Critical patent/PE20190393A1/es

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/34—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving hydrolase
    • C12Q1/44—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving hydrolase involving esterase
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/34—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving hydrolase
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/045—Hydroxy compounds, e.g. alcohols; Salts thereof, e.g. alcoholates
    • A61K31/07—Retinol compounds, e.g. vitamin A
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B01—PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01D—SEPARATION
    • B01D15/00—Separating processes involving the treatment of liquids with solid sorbents; Apparatus therefor
    • B01D15/08—Selective adsorption, e.g. chromatography
    • B01D15/26—Selective adsorption, e.g. chromatography characterised by the separation mechanism
    • B01D15/32—Bonded phase chromatography
    • B01D15/325—Reversed phase
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B01—PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01D—SEPARATION
    • B01D15/00—Separating processes involving the treatment of liquids with solid sorbents; Apparatus therefor
    • B01D15/08—Selective adsorption, e.g. chromatography
    • B01D15/26—Selective adsorption, e.g. chromatography characterised by the separation mechanism
    • B01D15/38—Selective adsorption, e.g. chromatography characterised by the separation mechanism involving specific interaction not covered by one or more of groups B01D15/265 - B01D15/36
    • B01D15/3804—Affinity chromatography
    • B01D15/3819—Affinity chromatography of the nucleic acid-nucleic acid binding protein type
    • 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)
    • 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)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/533—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving isomerase
    • 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
    • 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
    • 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
    • C12N2750/14143—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
    • C12Y—ENZYMES
    • C12Y203/00—Acyltransferases (2.3)
    • C12Y203/01—Acyltransferases (2.3) transferring groups other than amino-acyl groups (2.3.1)
    • C12Y203/01135—Phosphatidylcholine--retinol O-acyltransferase (2.3.1.135)
    • 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)
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/90—Enzymes; Proenzymes
    • G01N2333/914—Hydrolases (3)
    • G01N2333/916—Hydrolases (3) acting on ester bonds (3.1), e.g. phosphatases (3.1.3), phospholipases C or phospholipases D (3.1.4)
    • G01N2333/918—Carboxylic ester hydrolases (3.1.1)
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/16—Ophthalmology
    • G01N2800/164—Retinal disorders, e.g. retinopathy

Landscapes

  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Analytical Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Immunology (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Information Retrieval, Db Structures And Fs Structures Therefor (AREA)

Abstract

Referido a un metodo para medir y/o detectar la actividad de isomero hidrolasas, que comprende: (a) poner en contacto celulas que expresan lecitina retinol aciltransferasa (LRAT) con un vector viral adenoasociado (AAV) que comprende un transgen que codifica una proteina isomero hidrolasa (tal como, RPE65) bajo condiciones que permiten la transduccion celular; (b) incubar las celulas transducidas bajo condiciones que permiten la expresion de la proteina; (c) recolectar y lisar las celulas transducidas para producir un extracto que comprende la proteina; (d) incubar el extracto con un sustrato durante un periodo y bajo condiciones que permiten la conversion del sustrato por la proteina, a un producto de reaccion; (e) someter el producto de reaccion a cromatografia de columna, obteniendose un producto de reaccion purificado; y (f) someter el anterior producto a espectrometria de masa, cuantificandose el producto de reaccion, midiendose asi la actividad del isomero hidrolasa.
PE2018002238A 2016-04-28 2017-04-28 Ensayo de potencia relativa para un vector viral que codifica isomero hidrolasas PE20190393A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201662328916P 2016-04-28 2016-04-28

Publications (1)

Publication Number Publication Date
PE20190393A1 true PE20190393A1 (es) 2019-03-13

Family

ID=60161213

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2018002238A PE20190393A1 (es) 2016-04-28 2017-04-28 Ensayo de potencia relativa para un vector viral que codifica isomero hidrolasas

Country Status (18)

Country Link
US (1) US20210348210A1 (es)
EP (1) EP3448371A4 (es)
JP (1) JP7067843B2 (es)
KR (1) KR20190020661A (es)
CN (1) CN109689033A (es)
AU (2) AU2017258776B2 (es)
BR (1) BR112018072268A2 (es)
CA (1) CA3022386A1 (es)
CL (1) CL2018003079A1 (es)
CO (1) CO2018012509A2 (es)
IL (1) IL262583B2 (es)
MX (1) MX2018013205A (es)
MY (1) MY194096A (es)
PE (1) PE20190393A1 (es)
PH (1) PH12018502291A1 (es)
RU (1) RU2756403C2 (es)
SG (1) SG11201809388SA (es)
WO (1) WO2017190081A1 (es)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20240060989A1 (en) 2019-10-04 2024-02-22 Novartis Ag Methods for measuring cralbp activity
WO2023099791A1 (en) * 2021-12-03 2023-06-08 Dsm Ip Assets B.V. Novel compositions comprising trans retinol
WO2023099793A1 (en) * 2021-12-03 2023-06-08 Dsm Ip Assets B.V. Novel compositions comprising retinoids

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SU873121A1 (ru) * 1979-08-20 1981-10-15 Институт биохимии АН ЛитССР Способ определени активности гидролаз
AU714271B2 (en) * 1996-09-27 1999-12-23 Unilever Plc Skin care compositions containing combinations of compounds for mimicking the effect on skin of retinoic acid
US6071410A (en) * 1998-11-16 2000-06-06 Varian, Inc. Recovery of organic solutes from aqueous solutions
CN1997359A (zh) * 2004-02-17 2007-07-11 哈佛大学校长及研究员协会 包括黄斑变性在内的眼科疾病的处理
KR20140140616A (ko) * 2004-06-18 2014-12-09 유니버시티 오브 워싱톤 스루 이츠 센터 포 커머셜리제이션 시각 이상 치료를 위한 레티날 유도체 및 이를 이용하기 위한 방법
BRPI0515104A (pt) * 2004-09-09 2008-07-08 Univ Washington método para produzir todo-trans-(13,14)-diidrorretinol, polipeptìdeo isolado, polinucleotìdeo isolado, construção de expressão, célula procariótica ou eucariótica cultivada, vetor, célula hospedeira isolada, método para produzir um polipeptìdeo retsat, anticorpo, método para identificar agonistas ou antagonistas de um polipeptìdeo retsat eucariótico, composição farmacêutica, e, métodos para tratar uma doença neoplásica em um indivìduo mamìfero, para tratar doença retinal ou cegueira em um indivìduo mamìfero, para tratar um estado de doença retinal ou cegueira em um indivìduo mamìfero, para tratar doença auto imune em um indivìduo mamìfero e para tratar uma condição ou distúrbio de pele em um indivìduo mamìfero
AU2009352678B2 (en) * 2009-09-15 2015-05-21 Eluminex Biosciences (Suzhou) Limited Pharmaceutical formulations comprising 9-cis-retinyl esters in a lipid vehicle
US20140357611A1 (en) * 2009-12-08 2014-12-04 Case Western Reserve University Compounds and methods of treating ocular disorders
JP6600624B2 (ja) * 2013-05-31 2019-10-30 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア アデノ随伴ウイルス変異体及びその使用方法
WO2015184453A1 (en) 2014-05-30 2015-12-03 Case Western Reserve University Retinylamine derivitives for treatment of ocular disorders
WO2016018816A1 (en) * 2014-07-31 2016-02-04 The Board Of Regents Of The University Of Oklahoma High isomerohydrolase activity mutants of mammalian rpe65
GB201502137D0 (en) * 2015-02-09 2015-03-25 Ucl Business Plc Treatment

Also Published As

Publication number Publication date
AU2017258776A1 (en) 2018-11-29
BR112018072268A2 (pt) 2019-02-12
EP3448371A1 (en) 2019-03-06
KR20190020661A (ko) 2019-03-04
JP7067843B2 (ja) 2022-05-16
US20210348210A1 (en) 2021-11-11
RU2756403C2 (ru) 2021-09-30
SG11201809388SA (en) 2018-11-29
MY194096A (en) 2022-11-11
RU2018141675A3 (es) 2020-09-14
PH12018502291A1 (en) 2019-07-08
RU2018141675A (ru) 2020-05-29
CN109689033A (zh) 2019-04-26
JP2019514375A (ja) 2019-06-06
IL262583B1 (en) 2023-06-01
EP3448371A4 (en) 2019-09-11
CO2018012509A2 (es) 2019-04-30
IL262583A (en) 2018-12-31
CA3022386A1 (en) 2017-11-02
WO2017190081A1 (en) 2017-11-02
AU2023203939A1 (en) 2023-09-14
AU2017258776B2 (en) 2023-07-13
CL2018003079A1 (es) 2019-03-29
MX2018013205A (es) 2019-06-24
IL262583B2 (en) 2023-10-01

Similar Documents

Publication Publication Date Title
WO2015195769A3 (en) Optogenetic probes for measuring membrane potential
AU2018397910A1 (en) Nucleic acid-containing lipid nano-particle and use thereof
MX2020004145A (es) Variantes de desoxirribonucleasa (dnasa).
PE20190393A1 (es) Ensayo de potencia relativa para un vector viral que codifica isomero hidrolasas
AR122050A2 (es) Proteínas de fusión, bacterias recombinantes y métodos de uso de las bacterias recombinantes
CL2017002820A1 (es) Variantes de alfa-amilasa y polinucleótidos que codifican las mismas
CY1122612T1 (el) Μεθοδος
SA520412669B1 (ar) تصنيع ذيفانات عصبية ناتجة عن معاودة الارتباط الجيني من المطثية الوشيقية
MX2018004366A (es) Aislamiento temprano post-transfeccion de celulas (epic) para la produccion de productos biologicos.
EP3741849A3 (en) Protease variants and polynucleotides encoding same
MX2020013127A (es) P-hidroxifenilpiruvato dioxigenasa mutante, ácido nucleico que la codifica y su uso.
UY37970A (es) Plataforma de presentación, productos y métodos con endosporas basadas en paenibacillus
MX2017002805A (es) Descubrimiento y producción de proteínas biológicas condicionalmente activas en las mismas células hospederas eucariotas de producción.
BR112017018821A2 (pt) métodos para quantificação de níveis de insulina através de espectrometria de massa
BR112017016897A2 (pt) cepas fúngicas e métodos de uso
GB2537573A (en) Single platform doppler geolocation
BR112018006045A2 (pt) análise de marcador de massa para células raras e moléculas isentas de células
MX2019005697A (es) Medicion de longitud de onda secuencial multiple de una muestra liquida.
Nunomura et al. Role of oxidative stress in the pathophysiology of neuropsychiatric disorders
WO2016005985A3 (en) Method for reprogramming cells
MX2017014247A (es) Metodos para valorar la pureza de una preparacion de celulas germinales mesenquimales.
EA201990035A1 (ru) Генно-инженерные бета-глюкозидазы и способы глюкозилирования
MX2019000743A (es) Variantes de serinproteasa y polinucleotidos que las codifican.
BR112018077178A2 (pt) aumento de crescimento e rendimento de planta pelo uso de uma sequência de tiorredoxina
MX2019004013A (es) Aislamiento temprano de celulas despues de la transfeccion (epic) para la produccion de productos biologicos.