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1. WO2018229254 - EXPRESSION VECTORS COMPRISING ENGINEERED GENES

Publication Number WO/2018/229254
Publication Date 20.12.2018
International Application No. PCT/EP2018/065953
International Filing Date 15.06.2018
IPC
C12N 15/86 2006.1
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
86Viral vectors
C12Q 1/6813 2018.1
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
QMEASURING 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
1Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
68involving nucleic acids
6813Hybridisation assays
A61K 38/17 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
17from animals; from humans
CPC
A61K 38/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
A61K 38/177
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
17from animals; from humans
177Receptors; Cell surface antigens; Cell surface determinants
A61K 48/0058
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
005characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
A61P 25/08
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
25Drugs for disorders of the nervous system
08Antiepileptics; Anticonvulsants
C07K 14/705
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
705Receptors; Cell surface antigens; Cell surface determinants
C12N 15/86
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
86Viral vectors
Applicants
  • UCL BUSINESS LTD [GB]/[GB]
Inventors
  • SCHORGE, Stephanie
  • WALKER, Matthew Charles
  • KULLMANN, Dimitri M
  • SNOWBALL, Albert
  • CHABROL, Elodie
Agents
  • KREMER, Simon
  • ADDISS, John
  • ANDREWS, Robert
  • BAILEY, Sam
  • BREARLEY, Sarah
  • BURNS, Alexander
  • CARLISLE, Julie
  • CASLEY, Christopher
  • CLEGG, Richard
  • CRIPPS, Joanna
  • DALE, Elizabeth
  • DENISON, Christopher
  • DUNNE, Paul
  • FORREST, Graham
  • FURNIVAL, Tom
  • GILL, Stephen
  • GRAHAM, Emma
  • GREEN, Katherine
  • GREGORY, Adam
  • HARRISON, Susan
  • HAYES, Emily
  • HODSDON, Stephen
  • JOHNSON, Richard
  • JONES, Rachel
  • KEIRSTEAD, Tanis
  • KIDDLE, Simon
  • LEACH, James
  • LENTHALL, Joseph
  • LYONS, June
  • MACIVER, Eleanor
  • MCGUINN, Callum
  • MOELLER, Christoph
  • MOORE, Graeme
  • NAYLOR, Matthew
  • OXLEY, Rachel
  • PARRY, Simon
  • SALISBURY, Frances
  • SMITH, Matthew
  • STONER, Patrick
  • SUTCLIFFE, Nicholas
  • THORNTON, Dan
  • TOLLERVEY, Rebecca
  • WALTON, Seán
  • WARD, Eliot
  • WATSON, Robert
  • WEBSTER, Jeremy
  • WESCOTT, Rhiannon
  • WILLS, Jonathan
  • WYTENBURG, Wilhelmus
Priority Data
1709551.415.06.2017GB
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) EXPRESSION VECTORS COMPRISING ENGINEERED GENES
(FR) VECTEURS D'EXPRESSION COMPRENANT DES GÈNES MODIFIÉS
Abstract
(EN)
The invention provides expression vectors, nucleic acids, vector particles and methods of treatment involving these vector particles, comprising an engineered KCNA 1 gene encoding an edited Kv1.1 potassium channel, as well as methods of confirming the presence of engineered KCNA 1 mRNA in a cell. The features of the engineered KCNA 1 gene combine to advantageously enhance the translation and activity of the Kv1.1 protein and improve detection of KCNA 1 gene expression in a cell and can be used for example in the treatment of epilepsy and similar neurological disorders.
(FR)
L'invention concerne des vecteurs d'expression, des acides nucléiques, des particules de vecteur et des méthodes de traitement impliquant ces particules de vecteur, dont un gène KCNA 1 modifié codant pour un canal potassique Kv1.1 édité, ainsi que des procédés de confirmation de la présence d'ARNm KCNA 1 modifié dans une cellule. Les caractéristiques du gène KCNA 1 modifié se combinent pour améliorer avantageusement la traduction et l'activité de la protéine Kv1.1 et pour améliorer la détection de l'expression du gène KCNA 1 dans une cellule, et peuvent être utilisées, par exemple, dans le traitement de l'épilepsie et de troubles neurologiques similaires.
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