HRP20210273T1 - Prenilacijski test - Google Patents
Prenilacijski test Download PDFInfo
- Publication number
- HRP20210273T1 HRP20210273T1 HRP20210273TT HRP20210273T HRP20210273T1 HR P20210273 T1 HRP20210273 T1 HR P20210273T1 HR P20210273T T HRP20210273T T HR P20210273TT HR P20210273 T HRP20210273 T HR P20210273T HR P20210273 T1 HRP20210273 T1 HR P20210273T1
- Authority
- HR
- Croatia
- Prior art keywords
- rep1
- rab6a
- encoding
- gene therapy
- vector
- Prior art date
Links
- 238000003556 assay Methods 0.000 title 1
- 230000013823 prenylation Effects 0.000 title 1
- 101710088575 Rab escort protein 1 Proteins 0.000 claims 32
- 101710108890 Rab proteins geranylgeranyltransferase component A 1 Proteins 0.000 claims 32
- 102100022881 Rab proteins geranylgeranyltransferase component A 1 Human genes 0.000 claims 32
- 101150117360 RAB6A gene Proteins 0.000 claims 14
- 238000001415 gene therapy Methods 0.000 claims 13
- 239000013598 vector Substances 0.000 claims 13
- 238000000034 method Methods 0.000 claims 12
- 239000013603 viral vector Substances 0.000 claims 7
- 208000033810 Choroidal dystrophy Diseases 0.000 claims 4
- 208000003571 choroideremia Diseases 0.000 claims 4
- 239000002773 nucleotide Substances 0.000 claims 4
- 125000003729 nucleotide group Chemical group 0.000 claims 4
- 238000001514 detection method Methods 0.000 claims 3
- 150000002632 lipids Chemical class 0.000 claims 3
- 239000000758 substrate Substances 0.000 claims 3
- OINNEUNVOZHBOX-QIRCYJPOSA-K 2-trans,6-trans,10-trans-geranylgeranyl diphosphate(3-) Chemical group CC(C)=CCC\C(C)=C\CC\C(C)=C\CC\C(C)=C\COP([O-])(=O)OP([O-])([O-])=O OINNEUNVOZHBOX-QIRCYJPOSA-K 0.000 claims 2
- 238000002965 ELISA Methods 0.000 claims 2
- OINNEUNVOZHBOX-XBQSVVNOSA-N Geranylgeranyl diphosphate Natural products [P@](=O)(OP(=O)(O)O)(OC/C=C(\CC/C=C(\CC/C=C(\CC/C=C(\C)/C)/C)/C)/C)O OINNEUNVOZHBOX-XBQSVVNOSA-N 0.000 claims 2
- 108010055078 Rab geranylgeranyltransferase Proteins 0.000 claims 2
- 125000003275 alpha amino acid group Chemical group 0.000 claims 2
- 238000004458 analytical method Methods 0.000 claims 2
- 230000000694 effects Effects 0.000 claims 2
- 238000000338 in vitro Methods 0.000 claims 2
- 239000006166 lysate Substances 0.000 claims 2
- 238000003908 quality control method Methods 0.000 claims 2
- 238000011002 quantification Methods 0.000 claims 2
- BSODMXAKARYELM-ILVJLRRUSA-N triazanium;[[(2e,6e)-8-[5-[(3as,4s,6ar)-2-oxo-1,3,3a,4,6,6a-hexahydrothieno[3,4-d]imidazol-4-yl]pentanoylamino]-3,7-dimethylocta-2,6-dienoxy]-oxidophosphoryl] phosphate Chemical compound [NH4+].[NH4+].[NH4+].N1C(=O)N[C@@H]2[C@H](CCCCC(=O)NCC(/C)=C/CCC(/C)=C/COP([O-])(=O)OP([O-])([O-])=O)SC[C@@H]21 BSODMXAKARYELM-ILVJLRRUSA-N 0.000 claims 2
- 238000000376 autoradiography Methods 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 239000007790 solid phase Substances 0.000 claims 1
- 238000001262 western blot Methods 0.000 claims 1
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
- C12Q1/48—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving transferase
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
- G01N33/6893—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y205/00—Transferases transferring alkyl or aryl groups, other than methyl groups (2.5)
- C12Y205/01—Transferases transferring alkyl or aryl groups, other than methyl groups (2.5) transferring alkyl or aryl groups, other than methyl groups (2.5.1)
- C12Y205/0106—Protein geranylgeranyltransferase type II (2.5.1.60)
-
- 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
- G01N2333/91—Transferases (2.)
- G01N2333/9116—Transferases (2.) transferring alkyl or aryl groups other than methyl groups (2.5)
- G01N2333/91165—Transferases (2.) transferring alkyl or aryl groups other than methyl groups (2.5) general (2.5.1)
- G01N2333/91171—Transferases (2.) transferring alkyl or aryl groups other than methyl groups (2.5) general (2.5.1) with definite EC number (2.5.1.-)
Landscapes
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- Immunology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Biotechnology (AREA)
- Physics & Mathematics (AREA)
- Analytical Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- General Engineering & Computer Science (AREA)
- Biophysics (AREA)
- Urology & Nephrology (AREA)
- Biomedical Technology (AREA)
- Hematology (AREA)
- Medicinal Chemistry (AREA)
- Food Science & Technology (AREA)
- Cell Biology (AREA)
- General Physics & Mathematics (AREA)
- Pathology (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Steroid Compounds (AREA)
Claims (15)
1. Postupak određivanja aktivnosti vektora za gensku terapiju koji kodira Rab-eskort-protein 1 (REP1), naznačen time, da je za uporabu u liječenju koroideremije, gdje postupak obuhvaća sljedeće korake:
(a) stavljanje u doticaj uzorka koji sadrži REP1 s Rab6a, Rab-geranilgeraniltransferazom (Rab GGTaza) i supstratom lipidnog donatora, pri čemu uzorak koji sadrži REP1, potječe iz stanice koja je modificirana genetskim inženjerstvom tako da eksprimira REP1 uporabom vektora za gensku terapiju koji kodira REP1; i
(b) detektiranje lipidiranog proizvoda Rab6a.
2. Postupak za kontrolnu analizu kvalitete vektora za gensku terapiju koji kodira Rab-eskort-protein 1 (REP1), naznačen time, da je za uporabu u liječenju koroideremije, gdje postupak obuhvaća sljedeće korake:
(a) stavljanje u doticaj uzorka koji sadrži REP1 s Rab6a, Rab-geranilgeraniltransferazom (Rab GGTaza) i supstratom lipidnog donatora, pri čemu uzorak koji sadrži REP1, potječe iz stanice koja je modificirana genetskim inženjerstvom tako da eksprimira REP1 uporabom vektora za gensku terapiju koji kodira REP1; i
(b) detektiranje lipidiranog proizvoda Rab6a.
3. Postupak prema patentnom zahtjevu 1 ili 2, naznačen time, da uzorak koji sadrži REP1 jest lizat od stanice koja je genetski modificirana tako da eksprimira REP1 uporabom vektora za gensku terapiju koji kodira REP1.
4. Postupak prema bilo kojem od patentnih zahtjeva 1 do 3, naznačen time, da vektor za gensku terapiju koji kodira REP1 jest virusni vektor koji sadrži REP1-kodirajući nukleotidni slijed, poželjno s time, da je virusni vektor adeno-pridruženi virusni vektor (AAV), poželjno gdje je virusni vektor AAV serotipa 2 (AAV2).
5. Postupak prema bilo kojem od prethodnih patentnih zahtjeva, naznačen time, da nukleotidni slijed koji kodira REP1 vektora za gensku terapiju koji kodira REP1, sadrži nukleotidni slijed koji kodira aminokiselinski slijed koji je najmanje 80% istovjetan prema SEQ ID NO: 5, poželjno s time, da aminokiselinski slijed uglavnom zadržava prirodnu funkciju proteina predstavljenog sa SEQ ID NO: 5.
6. Postupak prema bilo kojem od prethodnih patentnih zahtjeva, naznačen time, da Rab6a i/ili Rab GGTaza su u osnovi čisti.
7. Postupak prema bilo kojem od prethodnih patentnih zahtjeva, naznačen time, da molarni omjer Rab6a:Rab GGTaza iznosi 1:2,3 ili 1:2,5.
8. Postupak prema bilo kojem od prethodnih patentnih zahtjeva, naznačen time, da supstrat lipidnog donatora jest geranilgeranilpirofosfat (GGPP) ili njegov analog, poželjno biotin-geranilpirofosfat (BGPP).
9. Postupak prema bilo kojem od prethodnih patentnih zahtjeva, naznačen time, da se lipidirani Rab6a proizvod detektira uporabom enzimskog imunotesta na čvrstoj fazi (ELISA), Western blot testa ili autoradiografije.
10. Postupak prema bilo kojem od prethodnih patentnih zahtjeva, naznačen time, da detektiranje lipidiranog Rab6a proizvoda obuhvaća kvantificiranje količine lipdidiranog Rab6a proizvoda, poželjno kvantificiranje količine u odnosu na kontrolnu ili referentnu razinu.
11. Uporaba Rab6a, naznačena time, da je za in vitro određivanje aktivnosti vektora za gensku terapiju koji kodira Rab-eskort-protein 1 (REP1), koji je za uporabu u liječenju koroideremije, pri čemu se Rab6a stavlja u doticaj s REP1 iz stanice koja je modificirana genetskim inženjerstvom tako da eksprimira REP1 uporabom vektora za gensku terapiju koji kodira REP1.
12. Uporaba Rab6a, naznačena time, da je za in vitro kontrolnu analizu kvalitete vektora za gensku terapiju koji kodira Rab-eskort-protein 1 (REP1), koji je za uporabu u liječenju koroideremije, pri čemu se Rab6a stavlja u doticaj s REP1 iz stanice koja je modificirana genetskim inženjerstvom tako da eksprimira REP1 uporabom vektora za gensku terapiju koji kodira REP1.
13. Uporaba prema patentnom zahtjevu 11 ili 12, naznačena time, da REP1 je lizat iz stanice koja je modificirana genetskim inženjerstvom tako da eksprimira REP1 uporabom vektora za gensku terapiju koji kodira REP1.
14. Uporaba prema bilo kojem od patentnih zahtjeva 11 do 13, naznačena time, da vektor za gensku terapiju koji kodira REP1 jest virusni vektor koji sadrži REP1-kodirajući nukleotidni slijed, poželjno gdje virusni vektor predstavlja adeno-pridruženi virusni vektor (AAV).
15. Uporaba prema bilo kojem od patentnih zahtjeva 11 do 14, naznačena time, da je Rab6a u osnovi čist.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GBGB1604146.9A GB201604146D0 (en) | 2016-03-10 | 2016-03-10 | Prenylation assay |
| PCT/GB2017/050659 WO2017153774A1 (en) | 2016-03-10 | 2017-03-10 | Prenylation assay |
| EP17711290.1A EP3426795B1 (en) | 2016-03-10 | 2017-03-10 | Prenylation assay |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20210273T1 true HRP20210273T1 (hr) | 2021-05-28 |
Family
ID=55952158
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20210273TT HRP20210273T1 (hr) | 2016-03-10 | 2021-02-17 | Prenilacijski test |
Country Status (21)
| Country | Link |
|---|---|
| US (2) | US20190017096A1 (hr) |
| EP (2) | EP3426795B1 (hr) |
| JP (1) | JP7021122B2 (hr) |
| KR (2) | KR20230093528A (hr) |
| CN (1) | CN109072281B (hr) |
| AU (1) | AU2017230952B2 (hr) |
| CA (1) | CA3016492A1 (hr) |
| CY (1) | CY1124274T1 (hr) |
| DK (1) | DK3426795T3 (hr) |
| ES (1) | ES2862773T3 (hr) |
| GB (1) | GB201604146D0 (hr) |
| HK (1) | HK1258918A1 (hr) |
| HR (1) | HRP20210273T1 (hr) |
| HU (1) | HUE054217T2 (hr) |
| LT (1) | LT3426795T (hr) |
| PL (1) | PL3426795T3 (hr) |
| PT (1) | PT3426795T (hr) |
| RS (1) | RS62032B1 (hr) |
| SG (1) | SG11201807766YA (hr) |
| SI (1) | SI3426795T1 (hr) |
| WO (1) | WO2017153774A1 (hr) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10633689B2 (en) * | 2016-11-07 | 2020-04-28 | Spark Therapeutics, Inc. | Rab Escort Protein potency assay |
| CA3083898A1 (en) * | 2017-10-17 | 2019-04-25 | Nightstarx Limited | Prenylation assay |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB9803351D0 (en) | 1998-02-17 | 1998-04-15 | Oxford Biomedica Ltd | Anti-viral vectors |
| GB0009760D0 (en) | 2000-04-19 | 2000-06-07 | Oxford Biomedica Ltd | Method |
| GB201103062D0 (en) * | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
| SE540689C2 (en) * | 2013-11-25 | 2018-10-09 | Esbe Ab | An valve for an indoor temperature regulating system and an air temperature conditioning unit including such a valve |
| WO2015082690A1 (en) * | 2013-12-06 | 2015-06-11 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods and pharmaceutical compositions for expressing a polynucleotide of interest in the retinal pigment epithelium of a subject |
-
2016
- 2016-03-10 GB GBGB1604146.9A patent/GB201604146D0/en not_active Ceased
- 2016-03-10 US US16/080,186 patent/US20190017096A1/en not_active Abandoned
-
2017
- 2017-03-10 ES ES17711290T patent/ES2862773T3/es active Active
- 2017-03-10 CN CN201780023762.5A patent/CN109072281B/zh active Active
- 2017-03-10 SI SI201730629T patent/SI3426795T1/sl unknown
- 2017-03-10 EP EP17711290.1A patent/EP3426795B1/en active Active
- 2017-03-10 KR KR1020237020248A patent/KR20230093528A/ko not_active Application Discontinuation
- 2017-03-10 JP JP2018566650A patent/JP7021122B2/ja active Active
- 2017-03-10 HU HUE17711290A patent/HUE054217T2/hu unknown
- 2017-03-10 WO PCT/GB2017/050659 patent/WO2017153774A1/en active Application Filing
- 2017-03-10 KR KR1020187029109A patent/KR20180120751A/ko not_active IP Right Cessation
- 2017-03-10 CA CA3016492A patent/CA3016492A1/en active Pending
- 2017-03-10 SG SG11201807766YA patent/SG11201807766YA/en unknown
- 2017-03-10 RS RS20210480A patent/RS62032B1/sr unknown
- 2017-03-10 LT LTEP17711290.1T patent/LT3426795T/lt unknown
- 2017-03-10 DK DK17711290.1T patent/DK3426795T3/da active
- 2017-03-10 PL PL17711290T patent/PL3426795T3/pl unknown
- 2017-03-10 PT PT177112901T patent/PT3426795T/pt unknown
- 2017-03-10 EP EP21152951.6A patent/EP3868892A1/en not_active Withdrawn
- 2017-03-10 AU AU2017230952A patent/AU2017230952B2/en active Active
-
2019
- 2019-01-28 HK HK19101395.8A patent/HK1258918A1/zh unknown
-
2021
- 2021-02-17 HR HRP20210273TT patent/HRP20210273T1/hr unknown
- 2021-04-13 CY CY20211100321T patent/CY1124274T1/el unknown
- 2021-12-22 US US17/559,713 patent/US20220380831A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| GB201604146D0 (en) | 2016-04-27 |
| PL3426795T3 (pl) | 2021-12-06 |
| JP7021122B2 (ja) | 2022-02-16 |
| SI3426795T1 (sl) | 2021-09-30 |
| DK3426795T3 (da) | 2021-03-01 |
| ES2862773T3 (es) | 2021-10-07 |
| JP2019509762A (ja) | 2019-04-11 |
| PT3426795T (pt) | 2021-04-07 |
| CY1124274T1 (el) | 2022-07-22 |
| US20220380831A1 (en) | 2022-12-01 |
| KR20180120751A (ko) | 2018-11-06 |
| AU2017230952B2 (en) | 2022-03-10 |
| CN109072281A (zh) | 2018-12-21 |
| EP3426795A1 (en) | 2019-01-16 |
| WO2017153774A1 (en) | 2017-09-14 |
| CN109072281B (zh) | 2023-07-25 |
| KR20230093528A (ko) | 2023-06-27 |
| AU2017230952A1 (en) | 2018-09-27 |
| HUE054217T2 (hu) | 2021-08-30 |
| EP3426795B1 (en) | 2021-01-27 |
| SG11201807766YA (en) | 2018-10-30 |
| LT3426795T (lt) | 2021-07-12 |
| RS62032B1 (sr) | 2021-07-30 |
| US20190017096A1 (en) | 2019-01-17 |
| EP3868892A1 (en) | 2021-08-25 |
| HK1258918A1 (zh) | 2019-11-22 |
| CA3016492A1 (en) | 2017-09-14 |
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