CY1123378T1 - Συνθεσεις χρησιμες στην αγωγη της ανεπαρκειας της τρανσκαρβαμυλασης της ορνιθινης (otc) - Google Patents

Συνθεσεις χρησιμες στην αγωγη της ανεπαρκειας της τρανσκαρβαμυλασης της ορνιθινης (otc)

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Publication number
CY1123378T1
CY1123378T1 CY20201100902T CY201100902T CY1123378T1 CY 1123378 T1 CY1123378 T1 CY 1123378T1 CY 20201100902 T CY20201100902 T CY 20201100902T CY 201100902 T CY201100902 T CY 201100902T CY 1123378 T1 CY1123378 T1 CY 1123378T1
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Cyprus
Prior art keywords
treatment
ornithine transcarbamylase
otc
transcarbamylase deficiency
compositions useful
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CY20201100902T
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English (en)
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Lili Wang
James M. Wilson
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The Trustees Of The University Of Pennsylvania
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Publication of CY1123378T1 publication Critical patent/CY1123378T1/el

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Abstract

Παρέχονται ιικοί φορείς που περιλαμβάνουν μηχανικά τροποποιημένες αλληλουχίες hOTC DNA και RNA που όταν διανέμονται σε ένα άτομο σε ανάγκη αυτών είναι χρήσιμοι για την αγωγή της υπεραμμωνιαιμίας, της ανεπάρκειας της τρανσκαρβαμυλάσης της ορνιθίνης και των συμπτωμάτων που σχετίζονται με αυτές. Επίσης παρέχονται μέθοδοι χρήσης της hOTC για την αγωγή της ηπατικής ίνωσης κίρρωσης σε ασθενείς με OTCD μέσω χορήγησης hOTC.
CY20201100902T 2014-03-09 2020-09-24 Συνθεσεις χρησιμες στην αγωγη της ανεπαρκειας της τρανσκαρβαμυλασης της ορνιθινης (otc) CY1123378T1 (el)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201461950157P 2014-03-09 2014-03-09
PCT/US2015/019513 WO2015138348A1 (en) 2014-03-09 2015-03-09 Compositions useful in treatment of ornithine transcarbamylase (otc) deficiency

Publications (1)

Publication Number Publication Date
CY1123378T1 true CY1123378T1 (el) 2021-12-31

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US (6) US9890365B2 (el)
EP (2) EP3116900B1 (el)
JP (2) JP6822841B2 (el)
KR (1) KR102390075B1 (el)
AU (2) AU2015229750A1 (el)
BR (2) BR122023023004A2 (el)
CA (1) CA2939950C (el)
CL (2) CL2016002235A1 (el)
CY (1) CY1123378T1 (el)
DK (1) DK3116900T3 (el)
ES (1) ES2821938T3 (el)
HR (1) HRP20201544T1 (el)
HU (1) HUE051311T2 (el)
IL (2) IL247329B (el)
LT (1) LT3116900T (el)
MX (1) MX2016011686A (el)
PL (1) PL3116900T3 (el)
PT (1) PT3116900T (el)
RS (1) RS60902B1 (el)
SI (1) SI3116900T1 (el)
WO (2) WO2015138348A1 (el)

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