PL3265563T3 - Regulacja ekspresji genów poprzez modulację alternatywnego splicingu za pośrednictwem aptamerów - Google Patents

Regulacja ekspresji genów poprzez modulację alternatywnego splicingu za pośrednictwem aptamerów

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Publication number
PL3265563T3
PL3265563T3 PL16747144T PL16747144T PL3265563T3 PL 3265563 T3 PL3265563 T3 PL 3265563T3 PL 16747144 T PL16747144 T PL 16747144T PL 16747144 T PL16747144 T PL 16747144T PL 3265563 T3 PL3265563 T3 PL 3265563T3
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PL
Poland
Prior art keywords
aptamer
regulation
gene expression
alternative splicing
mediated modulation
Prior art date
Application number
PL16747144T
Other languages
English (en)
Inventor
Alex R. Boyne
F. Olivier Danos
J. Michael Volles
Xuecui GUO
Original Assignee
Meiragtx Uk Ii Limited
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 Meiragtx Uk Ii Limited filed Critical Meiragtx Uk Ii Limited
Publication of PL3265563T3 publication Critical patent/PL3265563T3/pl

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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/44—Oxidoreductases (1)
    • 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
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/115—Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0012—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7)
    • C12N9/0026—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7) acting on CH-NH groups of donors (1.5)
    • C12N9/0028—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7) acting on CH-NH groups of donors (1.5) with NAD or NADP as acceptor (1.5.1)
    • C12N9/003—Dihydrofolate reductase [DHFR] (1.5.1.3)
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    • C12Y105/00—Oxidoreductases acting on the CH-NH group of donors (1.5)
    • C12Y105/01—Oxidoreductases acting on the CH-NH group of donors (1.5) with NAD+ or NADP+ as acceptor (1.5.1)
    • C12Y105/01003—Dihydrofolate reductase (1.5.1.3)
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2320/33—Alteration of splicing
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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PL16747144T 2015-02-02 2016-02-02 Regulacja ekspresji genów poprzez modulację alternatywnego splicingu za pośrednictwem aptamerów PL3265563T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562110919P 2015-02-02 2015-02-02
EP16747144.0A EP3265563B1 (en) 2015-02-02 2016-02-02 Regulation of gene expression by aptamer-mediated modulation of alternative splicing
PCT/US2016/016234 WO2016126747A1 (en) 2015-02-02 2016-02-02 Regulation of gene expression by aptamer-mediated modulation of alternative splicing

Publications (1)

Publication Number Publication Date
PL3265563T3 true PL3265563T3 (pl) 2021-09-13

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PL16747144T PL3265563T3 (pl) 2015-02-02 2016-02-02 Regulacja ekspresji genów poprzez modulację alternatywnego splicingu za pośrednictwem aptamerów

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Country Link
US (3) US10494646B2 (pl)
EP (2) EP3265563B1 (pl)
JP (3) JP6871174B2 (pl)
KR (2) KR20240133774A (pl)
CN (2) CN107849563B (pl)
AU (2) AU2016215454B2 (pl)
BR (1) BR112017016639A2 (pl)
CA (1) CA2975735A1 (pl)
CY (1) CY1124189T1 (pl)
DK (1) DK3265563T3 (pl)
EA (2) EA037120B1 (pl)
ES (1) ES2873400T3 (pl)
HK (1) HK1248760A1 (pl)
HR (1) HRP20210854T1 (pl)
HU (1) HUE054624T2 (pl)
IL (2) IL253786B (pl)
LT (1) LT3265563T (pl)
MX (2) MX2017009953A (pl)
MY (1) MY186203A (pl)
NZ (1) NZ735054A (pl)
PH (1) PH12017501389A1 (pl)
PL (1) PL3265563T3 (pl)
PT (1) PT3265563T (pl)
RS (1) RS61924B1 (pl)
SG (1) SG11201706297RA (pl)
SI (1) SI3265563T1 (pl)
WO (1) WO2016126747A1 (pl)
ZA (2) ZA202301731B (pl)

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