Inhibition of the synthesis of beta-app or of the activity of the a-beta peptide in the choroid plexus

Patent metadata

Field	Value
Publication number	US-2016159889-A1
Application number	US-201414901655-A
Country	US
Kind code	A1
Filing date	Jul 4, 2014
Priority date	Jul 4, 2013
Publication date	Jun 9, 2016
Grant date	—

Abstract

The invention relates to the treatment of neurodegenerative diseases, in particular Alzheimer's disease, by inhibition of the synthesis of βAPP or of the activity of the Aβ peptide in the choroid plexus.

First claim

1 . A method of treating a neurodegenerative disease in an individual in need thereof, comprising targeting an inhibitor of the synthesis of the PAPP protein or of the activity of the AP peptide or an expression vector coding for said inhibitor in choroid plexuses of said individual for decreasing the production or the activity of said Aβ peptide in said plexus choroids of the individual.

2 . The method according to claim 1 , wherein said inhibitor is selected from antisense oligonucleotides and interfering RNAs directed against the gene coding for said βAPP protein.

3 . The method according to claim 1 , wherein said inhibitor is an antibody or an antibody fragment selectively directed against said Aβ peptide.

4 . The method according to claim 1 , wherein said vector is a viral vector allowing specific expression of said inhibitor in choroid plexus cells.

5 . The method according to claim 4 , wherein the vector is derived from an adeno-associated virus of serotype 2, 4, or 5.

6 . The method according to claim 4 wherein said inhibitor is under control of a specific promoter of cells of the choroid plexus.

7 . The method according to claim 1 , wherein said inhibitor or vector is administered by injection into the retro-orbital sinus.

8 . The method according to claim 1 , wherein said inhibitor or vector is combined with a soluble functional form of the βAPP protein.

9 . The method according to claim 1 , wherein said neurodegenerative disease is a sporadic or family form of Alzheimer's disease.

Assignees

• Centre Nat Rech Scient
• College France
• Inst Nat Sante Rech Med
• Collège De France
• Inst Nat Santé Et De La Rech Médicale Inserm

Inventors

• Di Nardo Ariel
• Moya Kenneth Lee
• Arnaud Karen
• Prochiantz Alain

Classifications

• C07K16/18 Primary

  against material from animals or humans · CPC title

• A61P25/28

  for 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 · CPC title

• C07K2317/622

  Single chain antibody (scFv) · CPC title

• A61K39/0007

  Nervous system antigens; Prions · CPC title

• A61K2039/54

  characterised by the route of administration · CPC title