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Inducible cell-based model for the study of Friedreich's Ataxia
US9316634B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-9316634-B2 |
| Application number | US-201414276348-A |
| Country | US |
| Kind code | B2 |
| Filing date | May 13, 2014 |
| Priority date | May 13, 2013 |
| Publication date | Apr 19, 2016 |
| Grant date | Apr 19, 2016 |
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Abstract
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Isolated transduced cells exhibiting FRDA characteristics in an inducible fashion are disclosed. Isolated transduced cells comprise an expression vector having a nucleic acid sequence encoding an shRNA for frataxin protein knockdown and a heterologous expression control sequence. Additionally, methods of screening for a candidate therapeutic agent for treating Friedreich's Ataxia using isolated transduced cells are disclosed. Further, a recombinant nucleic acid construct for frataxin knockdown is disclosed that comprises a nucleic acid encoding an shRNA operably linked to a heterologous expression control sequence and expressing an shRNA molecule in a dose-responsive fashion.
First claim
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What is claimed is: 1. A recombinant nucleic acid construct for frataxin knockdown comprising a nucleic acid encoding an shRNA, wherein the shRNA comprises a sequence selected from the group consisting of SEQ ID NO: 1, SEQ ID NO: 2 and SEQ ID NO: 3, wherein the shRNA is operably linked to a heterologous expression control sequence. 2. The recombinant nucleic acid construct of claim 1 wherein the heterologous expression control sequence is selected from the gro…
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Chemistry & Metallurgy · mapped topic
- G01N33/5061Primary
Physics · mapped topic
Physics · mapped topic
Chemistry & Metallurgy · mapped topic
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- What does patent US9316634B2 cover?
- Isolated transduced cells exhibiting FRDA characteristics in an inducible fashion are disclosed. Isolated transduced cells comprise an expression vector having a nucleic acid sequence encoding an shRNA for frataxin protein knockdown and a heterologous expression control sequence. Additionally, methods of screening for a candidate therapeutic agent for treating Friedreich's Ataxia using isolated…
- Who is the assignee on this patent?
- Univ Indiana Res & Tech Corp, Univ Indiana Res & Tech Corp
- What technology area does this patent fall under?
- Primary CPC classification G01N33/5061. Mapped technology areas include Physics.
- When was this patent published?
- Publication date Tue Apr 19 2016 00:00:00 GMT+0000 (Coordinated Universal Time) (B2). Legal status and post-grant events are not shown on this page.
- What related patents are in patentsdb?
- We list 8 related publications on this page (citations in our corpus or others sharing the same primary CPC).