CY1123599T1 - Συνθεσεις και μεθοδοι για τη διορθωση ζωνιαιας μυικης δυστροφιας τυπου 2c με τη χρησιμοποιηση παραλειψης εξονιων - Google Patents
Συνθεσεις και μεθοδοι για τη διορθωση ζωνιαιας μυικης δυστροφιας τυπου 2c με τη χρησιμοποιηση παραλειψης εξονιωνInfo
- Publication number
- CY1123599T1 CY1123599T1 CY20201101145T CY201101145T CY1123599T1 CY 1123599 T1 CY1123599 T1 CY 1123599T1 CY 20201101145 T CY20201101145 T CY 20201101145T CY 201101145 T CY201101145 T CY 201101145T CY 1123599 T1 CY1123599 T1 CY 1123599T1
- Authority
- CY
- Cyprus
- Prior art keywords
- exon skipping
- muscular dystrophy
- methods
- striped
- correcting
- Prior art date
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- C12N2310/31—Chemical structure of the backbone
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
- C12N2310/3181—Peptide nucleic acid, PNA
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3233—Morpholino-type ring
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3513—Protein; Peptide
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Physics & Mathematics (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Footwear And Its Accessory, Manufacturing Method And Apparatuses (AREA)
Abstract
Η εφεύρεση στρέφεται προς ένα ή περισσότερα αντινοηματικά πολυνουκλεοτίδια και στη χρήση τους σε φαρμακευτικές συνθέσεις σε μια στρατηγική για την επαγωγή παράλειψης εξονίων στο γονίδιο γ-σαρκογλυκάνης σε ασθενείς που πάσχουν από Ζωνιαία Μυϊκή Δυστροφία-2C (LGMD2C) ή σε ασθενείς που κινδυνεύουν από μια τέτοια νόσο. Η εφεύρεση παρέχει επίσης μεθόδους πρόληψης ή θεραπείας μυϊκής δυστροφίας, π.χ., της LGMD2C, με παράλειψη εξονίων στο γονίδιο γάμμα σαρκογλυκάνης με τη χρησιμοποίηση αντινοηματικών πολυνουκλεοτιδίων. Αναλόγως, σε μερικές απόψεις η εφεύρεση παρέχει ένα απομονωμένο αντινοηματικό ολιγονουκλεοτίδιο, όπου το ολιγονουκλεοτίδιο υβριδοποιείται ειδικώς σε μια περιοχή εξονίου-στόχου ενός RNA γ-σαρκογλυκάνης. Σε μια άλλη άποψη, η εφεύρεση παρέχει μια μέθοδο επαγωγής παράλειψης εξονίων ενός RNA γάμμα σαρκογλυκάνης, περιλαμβάνουσα τη χορήγηση ενός αντινοηματικού ολιγονουκλεοτιδίου ή μιας σύνθεσης σε ένα κύτταρο.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201562144712P | 2015-04-08 | 2015-04-08 | |
PCT/US2016/026477 WO2016164602A1 (en) | 2015-04-08 | 2016-04-07 | Compositions and methods for correcting limb girdle muscular dystrophy type 2c using exon skipping |
Publications (1)
Publication Number | Publication Date |
---|---|
CY1123599T1 true CY1123599T1 (el) | 2022-03-24 |
Family
ID=57073329
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CY20201101145T CY1123599T1 (el) | 2015-04-08 | 2020-12-04 | Συνθεσεις και μεθοδοι για τη διορθωση ζωνιαιας μυικης δυστροφιας τυπου 2c με τη χρησιμοποιηση παραλειψης εξονιων |
Country Status (17)
Country | Link |
---|---|
US (5) | US10273483B2 (el) |
EP (1) | EP3283500B1 (el) |
BR (1) | BR112017021485A2 (el) |
CA (1) | CA2981960C (el) |
CY (1) | CY1123599T1 (el) |
DK (1) | DK3283500T3 (el) |
ES (1) | ES2846902T3 (el) |
HK (1) | HK1250720A1 (el) |
HR (1) | HRP20210139T1 (el) |
HU (1) | HUE052604T2 (el) |
LT (1) | LT3283500T (el) |
PL (1) | PL3283500T3 (el) |
PT (1) | PT3283500T (el) |
RS (1) | RS61349B1 (el) |
SI (1) | SI3283500T1 (el) |
TN (1) | TN2017000427A1 (el) |
WO (1) | WO2016164602A1 (el) |
Families Citing this family (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
RS61985B1 (sr) * | 2012-09-06 | 2021-07-30 | Univ Chicago | Antisens polinukleotidi za indukovanje preskakanja egzona i postupci lečenja distrofija |
EP3283500B1 (en) * | 2015-04-08 | 2020-11-11 | The University of Chicago | Compositions and methods for correcting limb girdle muscular dystrophy type 2c using exon skipping |
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EP3283500B1 (en) * | 2015-04-08 | 2020-11-11 | The University of Chicago | Compositions and methods for correcting limb girdle muscular dystrophy type 2c using exon skipping |
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2016
- 2016-04-07 EP EP16777291.2A patent/EP3283500B1/en active Active
- 2016-04-07 LT LTEP16777291.2T patent/LT3283500T/lt unknown
- 2016-04-07 ES ES16777291T patent/ES2846902T3/es active Active
- 2016-04-07 TN TNP/2017/000427A patent/TN2017000427A1/en unknown
- 2016-04-07 PT PT167772912T patent/PT3283500T/pt unknown
- 2016-04-07 SI SI201630981T patent/SI3283500T1/sl unknown
- 2016-04-07 CA CA2981960A patent/CA2981960C/en active Active
- 2016-04-07 BR BR112017021485A patent/BR112017021485A2/pt active Search and Examination
- 2016-04-07 DK DK16777291.2T patent/DK3283500T3/da active
- 2016-04-07 WO PCT/US2016/026477 patent/WO2016164602A1/en active Application Filing
- 2016-04-07 PL PL16777291T patent/PL3283500T3/pl unknown
- 2016-04-07 RS RS20210068A patent/RS61349B1/sr unknown
- 2016-04-07 US US15/564,681 patent/US10273483B2/en active Active
- 2016-04-07 HU HUE16777291A patent/HUE052604T2/hu unknown
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2018
- 2018-08-07 HK HK18110160.3A patent/HK1250720A1/zh unknown
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2019
- 2019-04-26 US US16/395,741 patent/US10801029B2/en active Active
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2020
- 2020-09-14 US US17/020,328 patent/US20210032631A1/en not_active Abandoned
- 2020-12-04 CY CY20201101145T patent/CY1123599T1/el unknown
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2021
- 2021-01-26 HR HRP20210139TT patent/HRP20210139T1/hr unknown
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2022
- 2022-01-07 US US17/571,302 patent/US20220119820A1/en not_active Abandoned
- 2022-08-11 US US17/819,291 patent/US20230167452A1/en not_active Abandoned
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US10273483B2 (en) | 2019-04-30 |
US10801029B2 (en) | 2020-10-13 |
DK3283500T3 (en) | 2020-11-16 |
US20180080030A1 (en) | 2018-03-22 |
LT3283500T (lt) | 2020-12-10 |
CA2981960A1 (en) | 2016-10-13 |
HUE052604T2 (hu) | 2021-05-28 |
TN2017000427A1 (en) | 2019-04-12 |
PL3283500T3 (pl) | 2021-05-31 |
US20210032631A1 (en) | 2021-02-04 |
EP3283500A1 (en) | 2018-02-21 |
US20230167452A1 (en) | 2023-06-01 |
RS61349B1 (sr) | 2021-02-26 |
HK1250720A1 (zh) | 2019-01-11 |
SI3283500T1 (sl) | 2020-12-31 |
ES2846902T3 (es) | 2021-07-30 |
BR112017021485A2 (pt) | 2018-07-03 |
EP3283500B1 (en) | 2020-11-11 |
US20220119820A1 (en) | 2022-04-21 |
CA2981960C (en) | 2023-09-19 |
US20190249180A1 (en) | 2019-08-15 |
WO2016164602A1 (en) | 2016-10-13 |
PT3283500T (pt) | 2021-01-28 |
HRP20210139T1 (hr) | 2021-03-19 |
EP3283500A4 (en) | 2019-01-23 |
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