CA3048038C - Therapie genique pour le traitement de la phenylcetonurie - Google Patents

Therapie genique pour le traitement de la phenylcetonurie

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Publication number
CA3048038C
CA3048038C CA3048038A CA3048038A CA3048038C CA 3048038 C CA3048038 C CA 3048038C CA 3048038 A CA3048038 A CA 3048038A CA 3048038 A CA3048038 A CA 3048038A CA 3048038 C CA3048038 C CA 3048038C
Authority
CA
Canada
Prior art keywords
raav
pah
aav
promoter
sequence
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
CA3048038A
Other languages
English (en)
Other versions
CA3048038A1 (fr
Inventor
James M. Wilson
Jenny Agnes Sidrane
Scott Ashley
Original Assignee
University of Pennsylvania Penn
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 University of Pennsylvania Penn filed Critical University of Pennsylvania Penn
Priority claimed from PCT/US2017/068897 external-priority patent/WO2018126112A1/fr
Publication of CA3048038A1 publication Critical patent/CA3048038A1/fr
Application granted granted Critical
Publication of CA3048038C publication Critical patent/CA3048038C/fr
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Abstract

L'invention concerne des compositions et des posologies utiles dans le traitement de la phénylcétonurie. Lesdites compositions comprennent un virus adéno-associé recombinant (VAAr) avec un activateur et un promoteur de la transthyrétine entraînant l'expression d'une phénylalanine hydroxylase humaine.
CA3048038A 2016-12-30 2017-12-29 Therapie genique pour le traitement de la phenylcetonurie Active CA3048038C (fr)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201662440651P 2016-12-30 2016-12-30
US62/440,651 2016-12-30
US201762469898P 2017-03-10 2017-03-10
US62/469,898 2017-03-10
US201762505373P 2017-05-12 2017-05-12
US62/505,373 2017-05-12
PCT/US2017/068897 WO2018126112A1 (fr) 2016-12-30 2017-12-29 Thérapie génique pour le traitement de la phénylcétonurie

Publications (2)

Publication Number Publication Date
CA3048038A1 CA3048038A1 (fr) 2018-07-05
CA3048038C true CA3048038C (fr) 2025-09-23

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