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1. WO2022119826 - PRODUCTS AND METHODS FOR INHIBITION OF EXPRESSION OF PERIPHERAL MYELIN PROTEIN-22

Publication Number WO/2022/119826
Publication Date 09.06.2022
International Application No. PCT/US2021/061177
International Filing Date 30.11.2021
IPC
C12P 19/34 2006.1
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
PFERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
19Preparation of compounds containing saccharide radicals
26Preparation of nitrogen-containing carbohydrates
28N-glycosides
30Nucleotides
34Polynucleotides, e.g. nucleic acids, oligoribonucleotides
A61K 48/00 2006.1
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
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
CPC
A61K 48/00
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
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
C12P 19/34
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
PFERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
19Preparation of compounds containing saccharide radicals
26Preparation of nitrogen-containing carbohydrates
28N-glycosides
30Nucleotides
34Polynucleotides, e.g. nucleic acids, oligoribonucleotides
Applicants
  • RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL [US]/[US]
  • THE CYPRUS INSTITUTE OF NEUROLOGY AND GENETICS [CY]/[CY]
Inventors
  • HARPER, Scott Quenton
  • KLEOPA, Kleopas
  • STAVROU, Marina
Agents
  • NOLAND, Greta, E.
Priority Data
63/120,19001.12.2020US
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) PRODUCTS AND METHODS FOR INHIBITION OF EXPRESSION OF PERIPHERAL MYELIN PROTEIN-22
(FR) COMPOSITIONS ET PROCÉDÉS POUR L'INHIBITION DE L'EXPRESSION DE LA PROTÉINE 22 DE LA MYÉLINE PÉRIPHÉRIQUE
Abstract
(EN) RNA interference-based methods and products for inhibiting the expression of a peripheral myelin protein-22 gene are provided. RNAs that inhibit the peripheral myelin protein-22 gene are provided as well as DMAs encoding the RNAs. Delivery vehicles such as recombinant adeno-associated viruses deliver DMAs encoding RNAs that inhibit the peripheral myelin protein-22 gene. The methods treat Charcot-Marie-Tooth Disease such as Charcot-Marie-Tooth Disease Type 1 A (CMT1A).
(FR) Procédés et produits basés sur l'interférence ARN pour inhiber l'expression d'un gène de la protéine 22 de la myéline périphérique. La présente invention concerne également des ARN inhibant le gène de la protéine 22 de la myéline périphérique ainsi que des DMA codant pour ces ARN. Des vecteurs d'administration tels que les virus adéno-associés recombinés délivrent des DMA codant pour des ARN inhibant le gène de la protéine 22 de la myéline périphérique. Les procédés traitent la maladie de Charcot-Marie-Tooth telle que la maladie de Charcot-Marie-Tooth de type 1 A (CMT1A).
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