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1. WO2011022843 - USE OF A HOLOTOXIN TO REDUCE ENDOPLASMIC RETICULUM-ASSOCIATED DEGRADATION OF MISFOLDED PROTEINS

Publication Number WO/2011/022843
Publication Date 03.03.2011
International Application No. PCT/CA2010/001360
International Filing Date 27.08.2010
IPC
A61K 38/16 2006.01
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
A61K 35/74 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
35Medicinal preparations containing materials or reaction products thereof with undetermined constitution
66Microorganisms or materials therefrom
74Bacteria
A61P 3/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
3Drugs for disorders of the metabolism
CPC
A61K 38/164
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
164from bacteria
A61P 19/02
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
19Drugs for skeletal disorders
02for joint disorders, e.g. arthritis, arthrosis
A61P 3/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
3Drugs for disorders of the metabolism
A61P 3/06
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
3Drugs for disorders of the metabolism
06Antihyperlipidemics
A61P 31/18
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
31Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
12Antivirals
14for RNA viruses
18for HIV
A61P 43/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
43Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
Applicants
  • THE HOSPITAL FOR SICK CHILDREN [CA]/[CA] (AllExceptUS)
  • LINGWOOD, Clifford (UsOnly)
Inventors
  • LINGWOOD, Clifford
Agents
  • GOWLING LAFLEUR HENDERSON LLP
Priority Data
61/237,91028.08.2009US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) USE OF A HOLOTOXIN TO REDUCE ENDOPLASMIC RETICULUM-ASSOCIATED DEGRADATION OF MISFOLDED PROTEINS
(FR) UTILISATION D'UNE HOLOTOXINE POUR RÉDUIRE LA DÉGRADATION ASSOCIÉE AU RÉTICULUM ENDOPLASMIQUE DE PROTÉINES MAL PLIÉES
Abstract
(EN)
Provided is a use of a holotoxin to reduce endoplasmic reticulum-associated degradation (ERAD) of misfolded or abnormally folded proteins The holotoxin can thus be used in a method to treat diseases related to ERAD Examples of misfolded proteins that are degraded in the ER include the cystic fibrosis transmembrane conductance regulator (CFTR) delta F508 mutant protein, a misfolded mutant (G268V) of multi-drug resistance 1 (MDR1), and the glucocerebrosidase (GCC) enzyme in Gaucher' s disease cells Examples of suitable holotoxins include ricin, shiga toxin, exotoxin A, plasmid-encoded toxin, cholera toxin, and verotoxin 1 (VT1) VT1 is also known as verotoxin A, shiga-like toxin 1, shiga-like toxin 1, or shiga toxin type 1 A non-toxic inactive VT1 can also be used wherein crucial residues of the A subumt active site are mutated, for example, the mutations Y77S and E167Q
(FR)
L'invention porte sur une utilisation d'une holotoxine pour réduire la dégradation associée au réticulum endoplasmique (ERAD) de protéines mal pliées ou anormalement pliées. L'holotoxine peut ainsi être utilisée dans un procédé de traitement de maladies liées à l'ERAD. Des exemples de protéines mal pliées qui sont dégradées dans l'ER comprennent la protéine mutante delta F508 du régulateur de conductance transmembranaire de la fibrose kystique (CFTR), un mutant mal plié (G268V) de résistance pléïotrope (MDR1) et l'enzyme glucocérébrosidase (GCC) dans les cellules atteintes de la maladie de Gaucher. Des exemples d'holotoxines appropriées comprennent la ricine, la toxine dysentérique, l'exotoxine A, la toxine codée par les plasmides, la toxine cholérique et la vérotoxine 1 (VT1). La VT1 est également connue sous le nom de vérotoxine A, vérocytotoxine 1 ou toxine dysentérique de type 1. Une VT1 inactive non toxique peut également être utilisée lorsque des résidus cruciaux du site actif de la sous-unité A sont mutés, par exemple les mutations Y77S et E167Q.
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