EA202192819A1 - METHODS AND COMPOSITIONS FOR TRANSGENE EXPRESSION - Google Patents

METHODS AND COMPOSITIONS FOR TRANSGENE EXPRESSION

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Publication number
EA202192819A1
EA202192819A1 EA202192819A EA202192819A EA202192819A1 EA 202192819 A1 EA202192819 A1 EA 202192819A1 EA 202192819 A EA202192819 A EA 202192819A EA 202192819 A EA202192819 A EA 202192819A EA 202192819 A1 EA202192819 A1 EA 202192819A1
Authority
EA
Eurasian Patent Office
Prior art keywords
methods
transgene
cell
compositions
transgene expression
Prior art date
Application number
EA202192819A
Other languages
Russian (ru)
Inventor
Джон Ф. Энгельхард
Цзыин Янь
Инхуа Тан
Эрик Юэнь
Шэнь Линь
Original Assignee
Юниверсити Оф Айова Рисерч Фаундейшн
Спайровант Сайенсиз, Инк.
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 Юниверсити Оф Айова Рисерч Фаундейшн, Спайровант Сайенсиз, Инк. filed Critical Юниверсити Оф Айова Рисерч Фаундейшн
Priority claimed from PCT/US2020/028269 external-priority patent/WO2020214672A1/en
Publication of EA202192819A1 publication Critical patent/EA202192819A1/en

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

Изобретение относится к способам экспрессии трансгена в клетке, способам лечения нарушений у субъекта, нуждающегося в этом, и фармацевтическим композициям. В частности, способы включают контакт клетки (например, клетки субъекта, страдающего таким заболеванием, как муковисцидоз) с рекомбинантным аденоассоциированным вирусом (rAAV), который содержит, в одном варианте осуществления, капсидный белок AV.TL65 и полинуклеотид, который содержит трансген в комбинации с усилителем трансдукции AAV, тем самым экспрессируя трансген в клетке. Изобретение также относится к фармацевтическим композициям, которые содержат rAAV, который содержит, в одном варианте осуществления, капсидный белок AV.TL65 и полинуклеотид, содержащий трансген в комбинации с одним или более усилителями.The invention relates to methods for expressing a transgene in a cell, methods for treating disorders in a subject in need thereof, and pharmaceutical compositions. In particular, the methods include contacting a cell (e.g., cells of a subject suffering from a disease such as cystic fibrosis) with a recombinant adeno-associated virus (rAAV) that contains, in one embodiment, an AV.TL65 capsid protein and a polynucleotide that contains a transgene in combination with an AAV transduction enhancer, thereby expressing the transgene in the cell. The invention also relates to pharmaceutical compositions that contain rAAV, which contains, in one embodiment, the AV.TL65 capsid protein and a polynucleotide containing a transgene in combination with one or more enhancers.

EA202192819A 2020-01-29 2020-04-15 METHODS AND COMPOSITIONS FOR TRANSGENE EXPRESSION EA202192819A1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202062967219P 2020-01-29 2020-01-29
PCT/US2020/028269 WO2020214672A1 (en) 2019-04-15 2020-04-15 Methods and compositions for transgene expression

Publications (1)

Publication Number Publication Date
EA202192819A1 true EA202192819A1 (en) 2022-02-18

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Family Applications (1)

Application Number Title Priority Date Filing Date
EA202192819A EA202192819A1 (en) 2020-01-29 2020-04-15 METHODS AND COMPOSITIONS FOR TRANSGENE EXPRESSION

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Country Link
EA (1) EA202192819A1 (en)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11684679B2 (en) 2016-03-07 2023-06-27 University Of Iowa Research Foundation AAV-mediated expression using a synthetic promoter and enhancer
US11702672B2 (en) 2016-02-08 2023-07-18 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11702672B2 (en) 2016-02-08 2023-07-18 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
US11684679B2 (en) 2016-03-07 2023-06-27 University Of Iowa Research Foundation AAV-mediated expression using a synthetic promoter and enhancer

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