CY1117852T1 - Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα - Google Patents
Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταραInfo
- Publication number
- CY1117852T1 CY1117852T1 CY20161100724T CY161100724T CY1117852T1 CY 1117852 T1 CY1117852 T1 CY 1117852T1 CY 20161100724 T CY20161100724 T CY 20161100724T CY 161100724 T CY161100724 T CY 161100724T CY 1117852 T1 CY1117852 T1 CY 1117852T1
- Authority
- CY
- Cyprus
- Prior art keywords
- exon
- cell
- skipping
- production
- instance
- Prior art date
Links
- 238000004519 manufacturing process Methods 0.000 title abstract 2
- 108020004707 nucleic acids Proteins 0.000 abstract 2
- 102000039446 nucleic acids Human genes 0.000 abstract 2
- 150000007523 nucleic acids Chemical class 0.000 abstract 2
- 108090000623 proteins and genes Proteins 0.000 abstract 2
- 102000004169 proteins and genes Human genes 0.000 abstract 2
- 108700024394 Exon Proteins 0.000 abstract 1
- 208000028782 Hereditary disease Diseases 0.000 abstract 1
- 108091027974 Mature messenger RNA Proteins 0.000 abstract 1
- 208000024556 Mendelian disease Diseases 0.000 abstract 1
- 230000001594 aberrant effect Effects 0.000 abstract 1
- 150000001413 amino acids Chemical class 0.000 abstract 1
- 239000003795 chemical substances by application Substances 0.000 abstract 1
- 230000003247 decreasing effect Effects 0.000 abstract 1
- 239000003814 drug Substances 0.000 abstract 1
- 230000001939 inductive effect Effects 0.000 abstract 1
- 230000002401 inhibitory effect Effects 0.000 abstract 1
- 108020004999 messenger RNA Proteins 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
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- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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Abstract
Η παρούσα εφεύρεση παρέχει μία μέθοδο για μείωση τουλάχιστον εν μέρει της παραγωγής μιας παρεκκλίνουσας πρωτεΐνης σε ένα κύτταρο και το εν λόγω κύτταρο περιέχει pre-mRNA το οποίον περιλαμβάνει εξώνια που κωδικεύουν αυτή την πρωτεΐνη, με επαγωγή της λεγομένης παράκαμψης εξωνίου στο εν λόγω κύτταρο. Η παράκαμψη εξωνίου έχει σαν αποτέλεσμα ώριμο mRNA που δεν περιέχει το παρακαμφθέν εξώνιο το οποίον οδηγεί σε ένα αλλαγμένο προϊόν εάν το εν λόγω εξώνιο κωδικεύει αμινοξέα. Η παράκαμψη εξωνίου διεξάγεται με εφοδιασμό ενός κυττάρου με έναν παράγοντα ικανό για ειδική αναστολή σήματος συμπερίληψης εξωνίου, για παράδειγμα μία αλληλουχία αναγνώρισης εξωνίου του εν λόγω εξωνίου. Το εν λόγω σήμα έγκλεισης εξωνίου μπορεί να παρέμβει σε ένα νουκλεϊνικό οξύ που περιλαμβάνει συμπληρωματικότητα για ένα μέρος αυτού του εξωνίου. Το εν λόγω νουκλεϊνικό οξύ το οποίον επίσης παρέχεται στο παρόν έγγραφο, μπορεί να χρησιμοποιηθεί για την παρασκευή ενός φαρμάκου, για παράδειγμα για την θεραπευτική αγωγή μιας κληρονομικής ασθένειας.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
EP00203283A EP1191097A1 (en) | 2000-09-21 | 2000-09-21 | Induction of exon skipping in eukaryotic cells |
EP01979073A EP1320597A2 (en) | 2000-09-21 | 2001-09-21 | Induction of exon skipping in eukaryotic cells |
EP13170245.8A EP2636741B1 (en) | 2000-09-21 | 2001-09-21 | Induction of exon skipping in eukaryotic cells |
Publications (1)
Publication Number | Publication Date |
---|---|
CY1117852T1 true CY1117852T1 (el) | 2017-05-17 |
Family
ID=8172043
Family Applications (10)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CY20081101494T CY1109601T1 (el) | 2000-09-21 | 2008-12-23 | Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα |
CY20161100207T CY1117521T1 (el) | 2000-09-21 | 2016-03-10 | Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα |
CY20161100215T CY1117522T1 (el) | 2000-09-21 | 2016-03-11 | Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα |
CY20161100216T CY1117318T1 (el) | 2000-09-21 | 2016-03-11 | Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα |
CY20161100443T CY1117887T1 (el) | 2000-09-21 | 2016-05-23 | Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα |
CY20161100724T CY1117852T1 (el) | 2000-09-21 | 2016-07-22 | Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα |
CY20171100302T CY1118972T1 (el) | 2000-09-21 | 2017-03-09 | Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα |
CY20171100691T CY1119026T1 (el) | 2000-09-21 | 2017-06-29 | Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα |
CY20171100750T CY1119088T1 (el) | 2000-09-21 | 2017-07-14 | Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα |
CY20181100969T CY1121139T1 (el) | 2000-09-21 | 2018-09-19 | Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα |
Family Applications Before (5)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CY20081101494T CY1109601T1 (el) | 2000-09-21 | 2008-12-23 | Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα |
CY20161100207T CY1117521T1 (el) | 2000-09-21 | 2016-03-10 | Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα |
CY20161100215T CY1117522T1 (el) | 2000-09-21 | 2016-03-11 | Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα |
CY20161100216T CY1117318T1 (el) | 2000-09-21 | 2016-03-11 | Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα |
CY20161100443T CY1117887T1 (el) | 2000-09-21 | 2016-05-23 | Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα |
Family Applications After (4)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CY20171100302T CY1118972T1 (el) | 2000-09-21 | 2017-03-09 | Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα |
CY20171100691T CY1119026T1 (el) | 2000-09-21 | 2017-06-29 | Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα |
CY20171100750T CY1119088T1 (el) | 2000-09-21 | 2017-07-14 | Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα |
CY20181100969T CY1121139T1 (el) | 2000-09-21 | 2018-09-19 | Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα |
Country Status (17)
Country | Link |
---|---|
US (6) | US7973015B2 (el) |
EP (14) | EP1191097A1 (el) |
JP (7) | JP4846965B2 (el) |
AT (1) | ATE409224T2 (el) |
AU (4) | AU1106202A (el) |
CA (1) | CA2423044C (el) |
CY (10) | CY1109601T1 (el) |
DE (1) | DE60135936D1 (el) |
DK (10) | DK2602322T3 (el) |
ES (11) | ES2581285T3 (el) |
HK (8) | HK1184821A1 (el) |
LT (3) | LT2940139T (el) |
NZ (1) | NZ524853A (el) |
PT (7) | PT2636741E (el) |
SI (1) | SI2801618T1 (el) |
TR (1) | TR201810606T4 (el) |
WO (1) | WO2002024906A1 (el) |
Families Citing this family (127)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP1282699B1 (en) | 2000-05-04 | 2012-11-21 | Sarepta Therapeutics, Inc. | Splice-region antisense composition and method |
EP1191097A1 (en) | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induction of exon skipping in eukaryotic cells |
ITRM20020253A1 (it) * | 2002-05-08 | 2003-11-10 | Univ Roma | Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche. |
ES2566628T3 (es) * | 2002-11-25 | 2016-04-14 | Masafumi Matsuo | Fármacos de ácido nucleico ENA que modifican el corte y empalme en precursores de ARNm |
WO2004083432A1 (en) * | 2003-03-21 | 2004-09-30 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure |
DK2351844T3 (da) | 2003-04-29 | 2014-09-22 | Sarepta Therapeutics Inc | Præparater til forøgelse af transport- og antisense-effektivitet af nukleinsyreanalog i celler |
US20050288246A1 (en) * | 2004-05-24 | 2005-12-29 | Iversen Patrick L | Peptide conjugated, inosine-substituted antisense oligomer compound and method |
USRE48960E1 (en) | 2004-06-28 | 2022-03-08 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
EP4047096A1 (en) | 2004-06-28 | 2022-08-24 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
FR2874384B1 (fr) * | 2004-08-17 | 2010-07-30 | Genethon | Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables |
EP1855694B1 (en) | 2005-02-09 | 2020-12-02 | Sarepta Therapeutics, Inc. | Antisense composition for treating muscle atrophy |
CN101184841A (zh) * | 2005-04-22 | 2008-05-21 | 莱顿教学医院 | 通过干扰SR蛋白的结合以及干扰RNA二级结构调节前mRNA中的外显子识别 |
US8067571B2 (en) | 2005-07-13 | 2011-11-29 | Avi Biopharma, Inc. | Antibacterial antisense oligonucleotide and method |
AU2006315758A1 (en) * | 2005-11-10 | 2007-05-24 | Ercole Biotech, Inc. | Splice switching oligomers for TNF superfamily receptors and their use in treatment of disease |
US7785834B2 (en) * | 2005-11-10 | 2010-08-31 | Ercole Biotech, Inc. | Soluble TNF receptors and their use in treatment of disease |
WO2007123391A1 (en) | 2006-04-20 | 2007-11-01 | Academisch Ziekenhuis Leiden | Therapeutic intervention in a genetic disease in an individual by modifying expression of an aberrantly expressed gene. |
EP1857548A1 (en) | 2006-05-19 | 2007-11-21 | Academisch Ziekenhuis Leiden | Means and method for inducing exon-skipping |
CA2660523C (en) | 2006-08-11 | 2019-03-19 | Prosensa Technologies B.V. | Methods and means for treating dna repeat instability associated genetic disorders |
US20090264353A1 (en) * | 2007-10-19 | 2009-10-22 | Santaris Pharma A/S | Splice Switching Oligomers for TNF Superfamily Receptors and their Use in Treatment of Disease |
ES2694726T3 (es) * | 2007-06-29 | 2018-12-26 | Sarepta Therapeutics, Inc. | Conjugados peptídicos específicos de tejido y métodos |
US20100016215A1 (en) | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
AU2008273096B2 (en) | 2007-07-12 | 2013-05-02 | Academisch Ziekenhuis Leiden | Molecules for targeting compounds to various selected organs or tissues |
CN101790385A (zh) | 2007-07-12 | 2010-07-28 | 普罗森那技术公司 | 用于使化合物靶向多种选定器官、组织或肿瘤细胞的分子 |
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