HRP20211697T1 - Zatvorena linearna dupleks dnk za nevirusni prijenos gena - Google Patents
Zatvorena linearna dupleks dnk za nevirusni prijenos gena Download PDFInfo
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- HRP20211697T1 HRP20211697T1 HRP20211697TT HRP20211697T HRP20211697T1 HR P20211697 T1 HRP20211697 T1 HR P20211697T1 HR P20211697T T HRP20211697T T HR P20211697TT HR P20211697 T HRP20211697 T HR P20211697T HR P20211697 T1 HRP20211697 T1 HR P20211697T1
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- 108700005077 Viral Genes Proteins 0.000 title 1
- 108020004707 nucleic acids Proteins 0.000 claims 17
- 102000039446 nucleic acids Human genes 0.000 claims 17
- 150000007523 nucleic acids Chemical class 0.000 claims 17
- 210000004027 cell Anatomy 0.000 claims 13
- 238000000034 method Methods 0.000 claims 7
- 101710150114 Protein rep Proteins 0.000 claims 6
- 101710152114 Replication protein Proteins 0.000 claims 6
- 239000000758 substrate Substances 0.000 claims 6
- 238000005096 rolling process Methods 0.000 claims 5
- 239000002773 nucleotide Substances 0.000 claims 4
- 125000003729 nucleotide group Chemical group 0.000 claims 4
- 108091081548 Palindromic sequence Proteins 0.000 claims 3
- 239000005547 deoxyribonucleotide Substances 0.000 claims 3
- 125000002637 deoxyribonucleotide group Chemical group 0.000 claims 3
- 108010017070 Zinc Finger Nucleases Proteins 0.000 claims 2
- 238000010362 genome editing Methods 0.000 claims 2
- 230000028993 immune response Effects 0.000 claims 2
- 238000000338 in vitro Methods 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- 102000004169 proteins and genes Human genes 0.000 claims 2
- 239000013598 vector Substances 0.000 claims 2
- 230000003612 virological effect Effects 0.000 claims 2
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 1
- 101100524317 Adeno-associated virus 2 (isolate Srivastava/1982) Rep40 gene Proteins 0.000 claims 1
- 101100524321 Adeno-associated virus 2 (isolate Srivastava/1982) Rep68 gene Proteins 0.000 claims 1
- 241001203868 Autographa californica Species 0.000 claims 1
- 108091033409 CRISPR Proteins 0.000 claims 1
- 108090000565 Capsid Proteins Proteins 0.000 claims 1
- 102100023321 Ceruloplasmin Human genes 0.000 claims 1
- 108020004414 DNA Proteins 0.000 claims 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 241000238631 Hexapoda Species 0.000 claims 1
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 1
- VYPSYNLAJGMNEJ-UHFFFAOYSA-N Silicium dioxide Chemical compound O=[Si]=O VYPSYNLAJGMNEJ-UHFFFAOYSA-N 0.000 claims 1
- 108700019146 Transgenes Proteins 0.000 claims 1
- 230000001580 bacterial effect Effects 0.000 claims 1
- 230000036755 cellular response Effects 0.000 claims 1
- 238000012217 deletion Methods 0.000 claims 1
- 230000037430 deletion Effects 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 239000013604 expression vector Substances 0.000 claims 1
- 230000008348 humoral response Effects 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 238000004806 packaging method and process Methods 0.000 claims 1
- 239000002245 particle Substances 0.000 claims 1
- 230000004962 physiological condition Effects 0.000 claims 1
- 238000002360 preparation method Methods 0.000 claims 1
- 238000000746 purification Methods 0.000 claims 1
- 230000003362 replicative effect Effects 0.000 claims 1
- 239000011347 resin Substances 0.000 claims 1
- 229920005989 resin Polymers 0.000 claims 1
- 239000000741 silica gel Substances 0.000 claims 1
- 229910002027 silica gel Inorganic materials 0.000 claims 1
- 241000701447 unidentified baculovirus Species 0.000 claims 1
- 241000701366 unidentified nuclear polyhedrosis viruses Species 0.000 claims 1
- 210000005253 yeast cell Anatomy 0.000 claims 1
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Claims (15)
1. Zatvorena linearna dupleks DNK (ceDNK) koja sadrži umetak nukleinske kiseline koji sadrži transgen, umetak je okružen s najmanje dvije sekvence ponavljanja obrnutog završetka (ITR) s adeno-povezanim virusom (AAV), pri čemu su najmanje dvije ITR sekvence asimetrične i kovalentno povezane jedna s drugom, pri čemu svaka sekvenca ima operativno terminalno mjesto razlučivanja i element za vezanje replikacijskog proteina kotrljajućeg kruga (RBE), pri čemu je prva ITR sekvenca prekinuta pomoću sekvence s unakrsnim krakom koja tvori dvije suprotne, po dužini simetrične stabljike-petlje nastale isprekidanim palindromskim sekvencama B-B' i C-C', pri čemu svaka od suprotnih po dužini simetričnih stabljika-petlji ima dio stabljike u rasponu duljine od 5 do 15 parova baza i dio petlje koji ima 2 do 5 neuparenih deoksiribonukleotida, pri čemu je druga ITR sekvenca prekinuta s odsječenom sekvencom s unakrsnim krakom koja ima jednu ili više delecija od između 11 i 20 nukleotida u području B-B' palindromske sekvence i/ili području C-C' palindromske sekvence.
2. ceDNK prema patentnom zahtjevu 1, naznačena time što
(i) ITR-ovi su u rasponu duljine od 40 do 1000 nukleotida, po izboru duljine 100 do 160 nukleotida;
(ii) sekvenca s unakrsnim krakom ima Gibbsovu slobodnu energiju (ΔG) odmatanja u fiziološkim uvjetima u rasponu od -12 kcal/mol do -30 kcal/mol, po izboru -20 kcal/mol do -25 kcal/mol;
(iii) RBE sadrži sekvencu 5'-GCTCGCTCGCTC-3'; i/ili
(iv) operativno terminalno mjesto razlučivanja sadrži sekvencu 5'-TT-3' i/ili 3' kraj operativnog terminalnog mjesta razlučivanja je 15 do 25 nukleotida od 5' kraja elementa za vezanje replikacijskog proteina kotrljajućeg kruga.
3. ceDNK prema patentnom zahtjevu 1 ili 2, naznačena time što odsječena sekvenca s unakrsnim krakom tvori dvije suprotne, po dužini asimetrične stabljike-petlje, po izboru pri čemu jedna od suprotnih, po dužini asimetrična stabljika-petlja ima dio stabljike u rasponu duljine od 8 do 10 parova baza i dio petlje koji ima 2 do 5 neuparenih deoksiribonukleotida ili jedna po dužini asimetričnu stabljika-petlja ima dio stabljike duljine manje od 8 parova baza i dio petlje koji ima 2 do 5 deoksiribonukleotida.
4. ceDNK prema bilo kojem od patentnih zahtjeva 1 do 3, naznačena time što je umetak nukleinske kiseline konstruiran za ekspresiju proteina ili funkcionalne RNK.
5. ceDNK prema bilo kojem od patentnih zahtjeva 1 do 4, naznačena time što je umetak nukleinske kiseline konstrukt bez promotora kao supstrat za uređivanje gena odabran od supstrata za TALENS, supstrata za nukleaze cinkovih prstiju (ZFN), supstrata za meganukleaze, supstrata za Cas9, te supstrata za drugi protein za uređivanje gena.
6. ceDNK prema bilo kojem od patentnih zahtjeva 1 do 5, naznačena time što je sadržana unutar vektora.
7. Pripravak naznačen time što sadrži mnoštvo ceDNK prema bilo kojem od patentnih zahtjeva 1 do 6, po izboru pri čemu je mnoštvo ceDNK povezano kraj s krajem, te po izboru nadalje sadrži farmaceutski prihvatljiv nosač.
8. Stanica domaćin koja sadrži ceDNK prema bilo kojem od patentnih zahtjeva 1 do 6, po izboru pri čemu stanica domaćin nadalje sadrži replikacijski protein koji se selektivno veže za RBE nukleinske kiseline.
9. In vitro postupak za isporuku heterologne nukleinske kiseline u stanicu, te se postupak sastoji od isporuke u stanicu ceDNK prema bilo kojem od patentnih zahtjeva 1 do 6.
10. ceDNK prema bilo kojem od patentnih zahtjeva 1 do 6 naznačena time što se koristi u postupku isporuke heterologne nukleinske kiseline subjektu, pri čemu isporuka ne rezultira imunološkim odgovorom na nukleinsku kiselinu u subjektu, po izboru pri čemu je imunološki odgovor humoralni odgovor ili stanični odgovor.
11. ceDNK za uporabu prema patentnom zahtjevu 10, naznačena time što se isporuka događa jednom ili više puta.
12. Stanica domaćin prema patentnom zahtjevu 8 za uporabu u postupku isporuke heterologne nukleinske kiseline subjektu, naznačena time što se isporuka stanice domaćina događa jednom ili više puta.
13. Postupak priprave nukleinskih kiselina, te postupak obuhvaća:
(i) uvođenje u pogodnu stanicu in vitro ceDNK nukleinske kiseline prema bilo kojem od zahtjeva 1 do 6, po izboru pri čemu je pogodnu stanicu stanična linija insekata, stanična linija kvasca ili bakterijska stanična linija; i
(ii) održavanje pogodne stanice pod uvjetima u kojima replikacijski protein kotrljajućeg kruga u pogodnoj stanici pokreće proizvodnju višestrukih kopija nukleinske kiseline, pri čemu pogodna stanica ne eksprimira virusne kapsidne proteine sposobne pakirati replikacijske kopije nukleinske kiseline u virusnu česticu, pri čemu je replikacijski protein kotrljajućeg kruga odabran iz skupine koju čine AAV78, AAV52, AAV Rep68 i AAV Rep40; i po izboru,
(iii) pročišćavanje višestrukih kopija nukleinske kiseline, pri čemu se pročišćavanje po izboru sastoji od dovođenja u kontakt nukleinske kiseline sa smolom silikagela.
14. Postupak prema patentnom zahtjevu 13, naznačen time što je replikacijski protein kotrljajućeg kruga kodiran vektorom Autographa californica multiple nuclear polyhedrosis virusa (AcMNPV) ili vektorom ekspresije bakulovirusa (BEV).
15. ceDNK prema bilo kojem od patentnih zahtjeva 1 do 6, naznačena time što ceDNK sadrži monomernu nukleinsku kiselinu koja sadrži jednu podjedinicu ili najmanje jednu multimernu nukleinsku kiselinu koja sadrži dvije ili više podjedinica, pri čemu svaka podjedinica sadrži umetak nukleinske kiseline na boku sa ITR sekvencama.
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662303047P | 2016-03-03 | 2016-03-03 | |
| US201662394720P | 2016-09-14 | 2016-09-14 | |
| US201662406913P | 2016-10-11 | 2016-10-11 | |
| PCT/US2017/020828 WO2017152149A1 (en) | 2016-03-03 | 2017-03-03 | Closed-ended linear duplex dna for non-viral gene transfer |
| EP17760963.3A EP3423110B1 (en) | 2016-03-03 | 2017-03-03 | Closed-ended linear duplex dna for non-viral gene transfer |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20211697T1 true HRP20211697T1 (hr) | 2022-02-04 |
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20211697TT HRP20211697T1 (hr) | 2016-03-03 | 2017-03-03 | Zatvorena linearna dupleks dnk za nevirusni prijenos gena |
Country Status (18)
| Country | Link |
|---|---|
| US (2) | US11066679B2 (hr) |
| EP (2) | EP3957331A1 (hr) |
| JP (3) | JP6994140B2 (hr) |
| KR (1) | KR102336362B1 (hr) |
| CN (2) | CN115287301A (hr) |
| AU (1) | AU2017228413A1 (hr) |
| CA (1) | CA3014683A1 (hr) |
| DK (1) | DK3423110T3 (hr) |
| ES (1) | ES2898337T3 (hr) |
| HR (1) | HRP20211697T1 (hr) |
| IL (2) | IL261524B2 (hr) |
| LT (1) | LT3423110T (hr) |
| MX (1) | MX2018010633A (hr) |
| PT (1) | PT3423110T (hr) |
| RU (1) | RU2752882C2 (hr) |
| SG (2) | SG10201913688TA (hr) |
| WO (1) | WO2017152149A1 (hr) |
| ZA (1) | ZA201806544B (hr) |
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