Human-enzyme mediated depletion of cystine for treating patients with cystinuria
AssigneeBoard of Regents, The University of Texas System
InventorEverett STONE
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Abstract
Methods and compositions related to the engineering of a protein with L-cyst(e)ine degrading enzyme activity are described. For example, in certain aspects there may be disclosed a modified cystathionine-γ-lyase comprising one or more amino acid substitutions and capable of degrading L-cyst(e)ine. Furthermore, certain aspects of the invention provide compositions and methods for the treatment of cancer with L-cyst(e)ine using the disclosed proteins or nucleic acids.
Claims14
A method of treating a subject having or at risk of developing cystinuria comprising administering to the subject a therapeutically effective amount of a formulation comprising an isolated, modified primate cystathionine-γ-lyase (CGL) enzyme having at least two substitutions relative to a native primate CGL amino acid sequence (see SEQ ID NOs:
- 1 and 7-10), said at least two substitutions including a threonine at position 59 and a valine at position 339 of the native primate CGL sequence or a nucleic acid comprising a nucleotide sequence encoding the isolated, modified primate cystathionine-γ-lyase (CGL) enzyme.
Dependencies are inferred from the claim text.