EP 1735443 A2 20061227 - RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA)
Title (en)
RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA)
Title (de)
RNA-INTERFERENZ-VERMITTELTE BEHANDLUNG VON POLYGLUTAMIN (POLYQ)-REPEAT-EXPANSIONSKRANKHEITEN UNTER VERWENDUNG VON SINA (SHORT INTEFERING NUCLEIC ACID)
Title (fr)
TRAITEMENT DE MALADIES A EXPANSION DE SEQUENCE REPETEE DE POLYGLUTAMINE (POLYQ) A MEDIATION D'ARN INTERFERENT METTANT EN OEUVRE UN ACIDE NUCLEIQUE COURT INTERFERENT (SINA)
Publication
Application
Priority
- US 2005006661 W 20050301
- US 82403604 A 20040414
- US 82696604 A 20040416
- US 2004013456 W 20040430
- US 2004016390 W 20040524
- US 92353604 A 20040820
- US 2005004270 W 20050209
Abstract (en)
[origin: WO2005105995A2] The present invention concerns compounds, compositions, and methods for the study, diagnosis, and treatment of diseases and conditions associated with polyglutamine repeat (polyQ) allelic variants that respond to the modulation of gene expression and/or activity. The present invention also concerns compounds, compositions, and methods relating to diseases and conditions associated with polyglutamine repeat (polyQ) allelic variants that respond to the modulation of expression and/or activity of genes involved in polyQ repeat gene expression pathways. or other cellular processes that mediate the maintenance or development of polyQ repeat diseases and conditions such as Huntington disease and related conditions such as progressive chorea, rigidity, dementia, and seizures, spinocerebellar ataxia, Spinal and bulbar muscular dystrophy (SBMA), dentatorubropallidoluysian atrophy (DIZPLA), and any other diseases or conditions that are related to or will respond to the levels of a repeat expansion (RE) protein in a cell or tissue, alone or in combination with other therapies. Specifically, the invention relates to small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), douse-stranded RN (dsRNA), micro-RNA (miRNA), and short hairpin RN (shRNA) molecules capable of mediating RN interference (RNAi) against the expression disease related genes or alleles having polyQ repeat sequences.
IPC 8 full level
A61K 31/713 (2006.01); A61P 25/28 (2006.01); C07H 21/00 (2006.01); C12N 15/113 (2010.01); A61K 38/00 (2006.01)
CPC (source: EP)
A61P 25/28 (2017.12); C12N 15/113 (2013.01); A61K 38/00 (2013.01); C12N 2310/111 (2013.01); C12N 2310/14 (2013.01); C12N 2310/315 (2013.01); C12N 2310/317 (2013.01); C12N 2310/318 (2013.01); C12N 2310/321 (2013.01); C12N 2310/322 (2013.01); C12N 2310/332 (2013.01); C12N 2310/53 (2013.01)
Citation (search report)
See references of WO 2005105995A2
Designated contracting state (EPC)
AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HU IE IS IT LI LT LU MC NL PL PT RO SE SI SK TR
DOCDB simple family (publication)
WO 2005105995 A2 20051110; WO 2005105995 A3 20051229; EP 1735443 A2 20061227; GB 0507019 D0 20050511; GB 2415961 A 20060111
DOCDB simple family (application)
US 2005006661 W 20050301; EP 05724244 A 20050301; GB 0507019 A 20050406