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US 8,912,147 B2Granted
Mitochondrial proteins constructs and uses thereof
AssigneeBioBlast Pharma Ltd.
InventorHaya Lorberboum-Galski
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Abstract
The invention provides fusion protein constructs comprising a functional mitochondrial protein and methods of treating mitochondrial disorders by the fusion proteins and compositions thereof.
Claims7
A fusion protein comprising a HIV-1 transactivator of transcription (TAT) domain fused to human frataxin and a human mitochondria targeting sequence (MTS) of a human mitochondrial protein being citrate synthase (CS) situated between said TAT domain and said frataxin, wherein said frataxin is C-terminal to said MTS of human citrate synthase.
A fusion protein having the amino acid sequence denoted by SEQ ID NO:
- 28, comprising a HIV-1 transactivator of transcription (TAT) domain having the amino acid sequence denoted by SEQ ID NO:
- 27 fused to human frataxin having the amino acid sequence denoted by SEQ ID NO:
- 26 and a mitochondria targeting sequence (MTS) of human citrate synthase having the amino acid sequence denoted by SEQ ID NO:
- 23, said MTS situated between said TAT domain and said frataxin, and is linked to said TAT domain via a linker having the amino acid sequence denoted by SEQ ID NO:
- 32, and wherein said frataxin is C-terminal to said MTS of human citrate synthase.
2 more elements
Dependencies are inferred from the claim text.