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 εξονιων

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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
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Cyprus
Prior art keywords
exon skipping
muscular dystrophy
methods
striped
correcting
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Application number
CY20201101145T
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English (en)
Inventor
Elizabeth MCNALLY
Eugene WYATT
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The University Of Chicago
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by The University Of Chicago filed Critical The University Of Chicago
Publication of CY1123599T1 publication Critical patent/CY1123599T1/el

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    • 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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    • C12N15/113—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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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.
CY20201101145T 2015-04-08 2020-12-04 Συνθεσεις και μεθοδοι για τη διορθωση ζωνιαιας μυικης δυστροφιας τυπου 2c με τη χρησιμοποιηση παραλειψης εξονιων CY1123599T1 (el)

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

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CY1123599T1 true CY1123599T1 (el) 2022-03-24

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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)

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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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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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