EA202192819A1 - METHODS AND COMPOSITIONS FOR TRANSGENE EXPRESSION - Google Patents
METHODS AND COMPOSITIONS FOR TRANSGENE EXPRESSIONInfo
- 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
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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.
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 |
Family
ID=80631338
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 |
Country Status (1)
Country | Link |
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EA (1) | EA202192819A1 (en) |
Cited By (2)
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 |
-
2020
- 2020-04-15 EA EA202192819A patent/EA202192819A1/en unknown
Cited By (2)
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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