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1. WO2006047250 - USE OF APOPTOSIS INHIBITING COMPOUNDS IN DEGENERATIVE NEUROLOGICAL DISORDERS

Publication Number WO/2006/047250
Publication Date 04.05.2006
International Application No. PCT/US2005/037822
International Filing Date 21.10.2005
IPC
A61P 25/28 2006.1
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
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
C07K 19/00 2006.1
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
19Hybrid peptides
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
A61P 25/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
25Drugs for disorders of the nervous system
A61P 25/16
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
25Drugs for disorders of the nervous system
14for treating abnormal movements, e.g. chorea, dyskinesia
16Anti-Parkinson drugs
A61P 25/28
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
C07K 14/4747
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
46from vertebrates
47from mammals
4701not used
4747Apoptosis related proteins
C07K 2319/02
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
2319Fusion polypeptide
01containing a localisation/targetting motif
02containing a signal sequence
Applicants
  • NEUROLOGIX, INC. [US]/[US] (AllExceptUS)
  • KAPLITT, Michael [US]/[US] (UsOnly)
  • MOUSSATOV, Serguei [RU]/[US] (UsOnly)
Inventors
  • KAPLITT, Michael
  • MOUSSATOV, Serguei
Agents
  • XIXIS, George, A.
Priority Data
60/621,30722.10.2004US
60/686,58802.06.2005US
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) USE OF APOPTOSIS INHIBITING COMPOUNDS IN DEGENERATIVE NEUROLOGICAL DISORDERS
(FR) UTILISATION DE COMPOSES INHIBANT L'APOPTOSE DANS LE TRAITEMENT DES TROUBLES NEUROLOGIQUES DEGENERATIFS
Abstract
(EN) The invention provides methods and compositions for localized delivery of a vector comprising a therapeutic agent to a specific region of the brain associated with a neurodegenerative diseases that is characterized by an excess buildup of intracellular protein aggregates. In particular, the invention provides methods and compositions used to deliver an adeno-associated virus vector (AAV) comprising a nucleotide sequence encoding an inhibitor of apoptosis protein (IAP) to cells in the region.
(FR) L'invention concerne des méthodes et des compositions permettant l'administration localisée d'un vecteur contenant un agent thérapeutique à une zone spécifique du cerveau, associée aux maladies neurodégénératives, et qui se caractérise par une accumulation excessive d'agrégats de protéines intracellulaires. L'invention concerne en particulier des méthodes et des compositions destinées à administrer à des cellules dans cette zone du cerveau un vecteur de virus associé aux adénovirus (VAA) contenant une séquence nucléotidique codant un inhibiteur de la protéine de l'apoptose (IAP).
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