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Homocystinuria (HCU) is a rare but potentially serious inherited condition.
Human enzyme-mediated homocysteine depletion for the treatment of hyperhomocysteinemia and homocystinuria patients
Active
CN110603324B
Hyperhomocystinemia
Amino acid substitution
Methods and compositions relating to the
engineering
of modified proteins having
homocysteine
activity are described. For example, a modified cystathionine-γ-
lyase
(CGL) comprising one or more
amino acid
substitutions and capable of degrading
homocysteine
is disclosed. Furthermore, compositions and methods for treating
homocysteine
depletion or
hyperhomocysteinemia
using the disclosed enzymes or nucleic acids are provided.
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Owner:
BOARD OF RGT THE UNIV OF TEXAS SYST
Compositions and methods for treatment of homocystinuria
Active
US12661391B2
Organic active ingredients
Peptide/protein ingredients
Pharmacology
Substitution therapy
Provided herein are improved compositions and methods for
enzyme
replacement therapy using modified human cystathionine beta synthase (CBS) in the treatment of homocystinuria and related diseases and disorders.
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Owner:
THE REGENTS OF THE UNIVERSITY OF COLORADO
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