Inducible cell-based model for the study of Friedreich's Ataxia
AssigneeIndiana University Research and Technology Corporation
InventorsRonald Mark Payne, Clifford M. Babbey
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Abstract
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.
Claims18
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.
An isolated transduced cell comprising an expression vector comprising a nucleic acid sequence 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, and a heterologous expression control sequence.
A method of screening for a candidate therapeutic agent for treating Friedreich's Ataxia, the method comprising:
- culturing an isolated C2C12 myoblast transduced cell comprising an expression vector comprising a nucleic acid sequence 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:
- 6, and a heterologous expression control sequence;
- inducing the isolated transduced cell to express the shRNA, wherein the shRNA reduces frataxin mRNA transcripts;
- contacting the isolated transduced cell with a candidate therapeutic agent;
- and
- analyzing the isolated transduced cell contacted with the candidate therapeutic agent.
Dependencies are inferred from the claim text.