CY1115408T1 - ENHANCEMENT OF EXPRESSION OF A MONOCLONE, NUCLEOTOLOGICAL SUCCESS STRUCTURE FROM RECOMMENDED VIRUSES VISUALLY DESIGNED - Google Patents

ENHANCEMENT OF EXPRESSION OF A MONOCLONE, NUCLEOTOLOGICAL SUCCESS STRUCTURE FROM RECOMMENDED VIRUSES VISUALLY DESIGNED

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Publication number
CY1115408T1
CY1115408T1 CY20141100552T CY141100552T CY1115408T1 CY 1115408 T1 CY1115408 T1 CY 1115408T1 CY 20141100552 T CY20141100552 T CY 20141100552T CY 141100552 T CY141100552 T CY 141100552T CY 1115408 T1 CY1115408 T1 CY 1115408T1
Authority
CY
Cyprus
Prior art keywords
raav vector
expression
heterologous sequence
viruses
heterologous
Prior art date
Application number
CY20141100552T
Other languages
Greek (el)
Inventor
Barrie Carter
Original Assignee
Genzyme Corporation
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
Priority claimed from EP07022775.6A external-priority patent/EP1916258B1/en
Application filed by Genzyme Corporation filed Critical Genzyme Corporation
Publication of CY1115408T1 publication Critical patent/CY1115408T1/en

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Abstract

Η παρούσα εφεύρεση παρέχει ένα ιικό παρασκεύασμα ανασυνδυασμένου αδενο-σχετιζόμενου ιού (rΑΑV), το οποίο rΑΑv ιικό παρασκεύασμα είναι ουσιαστικά απαλλαγμένο από άλλους ιούς, περιλαμβάνοντας έναν rΑΑV φορέα, όπου ο rΑΑV φορέας περιλαμβάνει μια μονόκλωνη ετερόλογη νουκλεοτιδική αλληλουχία η οποία περιλαμβάνει μια κωδικοποιούσα περιοχή η οποία σχηματίζει ενδοκλωνικά ζεύγη βάσεων έτσι ώστε έκφραση της κωδικoποιούσας περιοχής της ετερόλογης αλληλουχίας ενισχύεται σε σχέση με έναν rΑΑV φορέα ο οποίος στερείται επαρκούς ενδοκλωνικής σύζευξης βάσεων για ενίσχυση της εν λόγω έκφρασης, όπου ο rΑΑV φορέας περιλαμβάνει μία ή περισσότερες αλληλουχίες ανεστραμμένης τελικής επανάληψης (ΙΤR) οι οποίες πλευρίζουν την εν λόγω ετερόλογη αλληλουχία, όπου ο rΑΑV φορέας είναι μήκους μικρότερου από 2.5 kilobases, όπου η συνολική ποσότητα μοναδικής αλληλουχίας που υπάρχει στην ετερόλογη αλληλουχία είναι περίπου το ήμισυ της ετερόλογης αλληλουχίας και όπου η ετερόλογη αλληλουχία σχηματίζει ενδοκλωνικά ζεύγη βάσεων στο μεγαλύτερο μέρος ή σε ολόκληρο το μήκος της, για χρήση σε μία μέθοδο αγωγής του ανθρώπινου ή ζωικού σώματος μέσω θεραπείας.The present invention provides a recombinant adeno-associated virus (rAV) viral preparation, which rAAV viral preparation is substantially free of other viruses, including a rAAV vector, wherein the rAAV vector comprises a single-stranded heterologous coding region which forms endoclonal base pairs such that expression of the coding region of the heterologous sequence is enhanced relative to a rAAV vector lacking sufficient intraclonal base pairing for amplification of said expression, wherein the rAAV vector comprises one or more inverted end-repeat (ITR) sequences that flank said heterologous sequence, wherein the rAAV vector is less than 2.5 kilobases, wherein the total amount of unique sequence present in the heterologous sequence is about half of the heterologous sequence and wherein the heterologous sequence forms endoclonal base pairs over most or all of its length, for use in a method of human treatment. mind or animal body by therapy.

CY20141100552T 1999-08-09 2014-07-22 ENHANCEMENT OF EXPRESSION OF A MONOCLONE, NUCLEOTOLOGICAL SUCCESS STRUCTURE FROM RECOMMENDED VIRUSES VISUALLY DESIGNED CY1115408T1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US16008099A 1999-08-09 1999-08-09
EP07022775.6A EP1916258B1 (en) 1999-08-09 2000-08-08 Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs

Publications (1)

Publication Number Publication Date
CY1115408T1 true CY1115408T1 (en) 2017-01-04

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Family Applications (1)

Application Number Title Priority Date Filing Date
CY20141100552T CY1115408T1 (en) 1999-08-09 2014-07-22 ENHANCEMENT OF EXPRESSION OF A MONOCLONE, NUCLEOTOLOGICAL SUCCESS STRUCTURE FROM RECOMMENDED VIRUSES VISUALLY DESIGNED

Country Status (1)

Country Link
CY (1) CY1115408T1 (en)

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