Modified tafazzin proteins and methods of making and using the same
AssigneeUniversity of Washington
InventorsMichael T. Chin, Wei-Ming Chien, Ana Dinca
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Abstract
Fusion proteins comprising a tafazzin peptide and a cellular permeability peptide are disclosed. The tafazzin peptide may be coupled to the permeability peptide through a polypeptide linker. Methods of making and using the fusion proteins are also disclosed. For example, the disclosed fusion proteins may be used to treat a patient having a disorder associated with a tafazzin deficiency or a remodeled cardiolipin deficiency (e.g., Barth syndrome). Additionally, the disclosed fusion proteins may be used in prophylaxis against developing a disorder associated with a tafazzin deficiency or a remodeled cardiolipin deficiency in a patient at risk of developing such a disorder.
Claims30
An isolated and purified fusion protein comprising:
- a tafazzin peptide;
- and
- a cellular permeability peptide.
A method for treating a patient having a disorder associated with a tafazzin deficiency or a remodeled cardiolipin deficiency, comprising:
- administering to the patient an effective amount of a pharmaceutical composition comprising:
- a fusion protein comprising a tafazzin peptide and a cellular permeability peptide;
- and
- a pharmaceutically acceptable carrier
- to reduce a pathological effect or symptom of the disorder associated with the tafazzin deficiency or the remodeled cardiolipin deficiency.
A method of treating a patient having or at risk of developing, Barth syndrome, comprising:
- administering to the patient an effective amount of a pharmaceutical composition comprising:
- a fusion protein comprising a tafazzin peptide and a permeability peptide;
- and
- a pharmaceutically acceptable carrier
- to reduce a pathological effect or symptom of Barth syndrome, or to reduce the risk of developing Barth syndrome.
Dependencies are inferred from the claim text.