A Potential New Therapeutic Approach for Friedreich Ataxia: Induction of Frataxin Expression With TALE Proteins
TALEs targeting a promoter sequence and fused with a transcription activation domain (TAD) may be used to specifically induce the expression of a gene as a potential treatment for haploinsufficiency. This potential therapeutic approach was applied to increase the expression of frataxin in fibroblast...
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| Main Authors: | , , , , |
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| Format: | Book |
| Published: |
Elsevier,
2013-01-01T00:00:00Z.
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| Online Access: | Connect to this object online. |
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