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1. WO2020117703 - METHODS FOR THE TREATMENT OF TRINUCLEOTIDE REPEAT EXPANSION DISORDERS ASSOCIATED WITH MSH3 ACTIVITY

Publication Number WO/2020/117703
Publication Date 11.06.2020
International Application No. PCT/US2019/064055
International Filing Date 02.12.2019
IPC
C12N 15/10 2006.01
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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
10Processes for the isolation, preparation or purification of DNA or RNA
C12N 15/11 2006.01
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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
11DNA or RNA fragments; Modified forms thereof
C12N 15/113 2010.01
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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
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C12N 15/63 2006.01
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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
A61P 25/28 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
25Drugs for disorders of the nervous system
28for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
C12Q 1/68 2018.01
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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
Applicants
  • TRIPLET THERAPEUTICS, INC. [US]/[US]
Inventors
  • BERMINGHAM, Nessan Anthony
  • BETTENCOURT, Brian R.
  • BIALEK, Peter Edward
Agents
  • ROSE GILLENTINE, Marsha
  • BANOWIT, Donald R.
  • BEMBEN, Richard M.
  • BEZOS, Salvador M.
  • BLOCK, Daniel S.
  • BODENSTEIN, Matthew
  • CALVO, Paul A.
  • CAMARCE, Christian A.
  • CIMBALA, Michele A.
  • COLLER III, Richard D.
  • CONKLIN, Kyle
  • COVERT, John M.
  • DESAI, Nirav N.
  • DURKIN, Tracy-Gene G.
  • EISENBERG, Jason D.
  • ELLISON, Eldora L.
  • ESMOND, Robert W.
  • FEATHERSTONE, Donald J.
  • FITZSIMMONS, Jason A.
  • FRUEAUF, Jeremiah B.
  • GAJEWSKI, Daniel
  • GOLDSTEIN, Jorge A.
  • HELVEY, Jeffrey T.
  • HICKS, Ross
  • HOLOUBEK, Michelle K.
  • JACKMAN, Peter A.
  • KENTON, JR., Lestin L.
  • KIM, Ji-Eun
  • LAROCK, Adam C.
  • LEE, Michael Q.
  • LONGSWORTH, Gaby L.
  • MILLONIG, Robert C.
  • MUTSCHELKNAUS, Joseph
  • NANNENGA-COMBS, Bonnie
  • PICKARD, Byron L.
  • POWERS III, R. Wilson
  • RAY, Michael B.
  • RICHARDSON, Ryan C.
  • RYGIEL, Mark W.
  • SHEA, JR., Timothy J.
  • SOKOHL, Robert E.
  • SPECHT, Michael D.
  • STEFFE, Eric K.
  • STERLING, Deborah A.
  • STERNE, Robert Greene
  • TUMINARO, Jonathan
  • VARUGHESE, Dennies
  • VIRA, Chandrika
  • WRIGHT, Jon E.
  • YONAN, Daniel E.
Priority Data
62/774,79103.12.2018US
62/877,14222.07.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) METHODS FOR THE TREATMENT OF TRINUCLEOTIDE REPEAT EXPANSION DISORDERS ASSOCIATED WITH MSH3 ACTIVITY
(FR) PROCÉDÉS POUR LE TRAITEMENT DE TROUBLES D'EXPANSION DE RÉPÉTITION TRINUCLÉOTIDIQUE ASSOCIÉS À UNE ACTIVITÉ DE MSH3
Abstract
(EN)
The present disclosure features useful compositions and methods to treat trinucleotide repeat expansion disorders, e.g., in a subject in need thereof. In some aspects, the compositions and methods described herein are useful in the treatment of disorders associated with MSH3 activity.
(FR)
La présente invention concerne des compositions et des procédés utiles pour traiter les troubles d'expansion de répétition trinucléotidique chez un sujet en ayant besoin. Dans certains modes de réalisation, les compositions et les procédés de la présente invention sont utiles dans le traitement des troubles associés à une activité de MSH3.
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