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1. WO2011015573 - MOLECULAR TARGETS AND COMPOUNDS, AND METHODS TO IDENTIFY THE SAME, USEFUL IN THE TREATMENT OF NEURODEGENERATIVE DISEASES

Publication Number WO/2011/015573
Publication Date 10.02.2011
International Application No. PCT/EP2010/061274
International Filing Date 03.08.2010
IPC
G01N 33/50 2006.1
GPHYSICS
01MEASURING; TESTING
NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
33Investigating or analysing materials by specific methods not covered by groups G01N1/-G01N31/131
48Biological material, e.g. blood, urine; Haemocytometers
50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
G01N 33/68 2006.1
GPHYSICS
01MEASURING; TESTING
NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
33Investigating or analysing materials by specific methods not covered by groups G01N1/-G01N31/131
48Biological material, e.g. blood, urine; Haemocytometers
50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
68involving proteins, peptides or amino acids
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
CPC
C12Q 1/6883
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS
1Measuring or testing processes involving enzymes, nucleic acids or microorganisms
68involving nucleic acids
6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
6883for diseases caused by alterations of genetic material
C12Q 2600/136
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS
2600Oligonucleotides characterized by their use
136Screening for pharmacological compounds
C12Q 2600/156
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS
2600Oligonucleotides characterized by their use
156Polymorphic or mutational markers
C12Q 2600/158
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS
2600Oligonucleotides characterized by their use
158Expression markers
G01N 2500/04
GPHYSICS
01MEASURING; TESTING
NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
2500Screening for compounds of potential therapeutic value
04Screening involving studying the effect of compounds C directly on molecule A (e.g. C are potential ligands for a receptor A, or potential substrates for an enzyme A)
G01N 2500/10
GPHYSICS
01MEASURING; TESTING
NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
2500Screening for compounds of potential therapeutic value
10involving cells
Applicants
  • GALAPAGOS NV [BE]/[BE] (AllExceptUS)
  • FISCHER, David, Frederik [NL]/[NL] (UsOnly)
  • DE PRIL, Remko, [NL]/[NL] (UsOnly)
  • VAN STEENHOVEN, Desiré, Maria, Petronella, Catharina [NL]/[NL] (UsOnly)
  • JANSSEN, Richard, Antonius, Jozef [NL]/[CH] (UsOnly)
  • KWAK, Seung [US]/[US] (UsOnly)
  • HOWLAND, David [US]/[US] (UsOnly)
  • SIGNER, Ethan [US]/[US] (UsOnly)
Inventors
  • FISCHER, David, Frederik
  • DE PRIL, Remko,
  • VAN STEENHOVEN, Desiré, Maria, Petronella, Catharina
  • JANSSEN, Richard, Antonius, Jozef
  • KWAK, Seung
  • HOWLAND, David
  • SIGNER, Ethan
Agents
  • TEUTEN, Andrew
Priority Data
61/273,30603.08.2009US
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) MOLECULAR TARGETS AND COMPOUNDS, AND METHODS TO IDENTIFY THE SAME, USEFUL IN THE TREATMENT OF NEURODEGENERATIVE DISEASES
(FR) CIBLES ET COMPOSÉS MOLÉCULAIRES, ET PROCÉDÉS POUR LES IDENTIFIER, UTILES DANS LE TRAITEMENT DE MALADIES NEURODÉGÉNÉRATIVES
Abstract
(EN) The present invention relates to methods and assays for identifying agents capable of inhibiting the mutant huntingtin protein, inhibiting or reducing cell death, in particular cell death associated with polyglutamine-induced protein aggregation, which inhibition is useful in the prevention, amelioration and/or treatment of neurodegenerative diseases, and Huntington's disease more generally. In particular, the present invention provides methods and assays for identifying agents for use in the prevention and/or treatment of Huntington's disease. The invention provides polypeptide and nucleic acid TARGETs and siRNA sequences based on these TARGETs.
(FR) La présente invention a pour objet des procédés et des analyses pour l'identification d'agents capables d'inhiber la protéine huntingtine mutante, d'inhiber ou de réduire la mort cellulaire, en particulier la mort cellulaire associée à une agrégation de protéine induite par la polyglutamine, laquelle inhibition est utile dans la prévention, l'amélioration et/ou le traitement de maladies neurodégénératives, et de la maladie de Huntington de manière plus générale. En particulier, la présente invention concerne des procédés et des analyses pour l'identification d'agents destinés à être utilisés dans la prévention et/ou le traitement de la maladie de Huntington. La présente invention concerne des CIBLES polypeptide et acide nucléique et des séquences d'ARNsi basées sur ces CIBLES.
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