X-Ray Crystallography in Structure-Function Characterization of Therapeutic Enzymes

  • Anastassios C. PapageorgiouEmail author
Part of the Advances in Experimental Medicine and Biology book series (AEMB, volume 1148)


Enzymes are key biological macromolecules that support life by accelerating the conversion of target molecules to desired products in many biochemical reactions. Enzymes are characterized by high affinity, specificity and great catalytic efficiency. Owing to their unique characteristics, enzymes have attracted significant attention for use in therapeutic settings as a distinct class of drugs different from other types of medicines. Enzyme-based therapies are currently in use for the treatment of a wide range of diseases, including leukemia, metabolic disorders, inflammation and cardiovascular disease. However, several challenges, such as immunogenicity and stability, remain. X-ray crystallography has provided key structural insights into the understanding of the molecular basis of diseases and development of enzyme-based therapies. Here, the role of X-ray crystallography in the development of therapeutic enzymes is examined and several examples are provided.


Enzyme therapy Enzyme deficiency Enzyme stability Chaperones Structure stabilizers 



Arginine deiminase




Chinese hamster ovary






E. coli ASNase


Erwinia chrysanthemi L-asparaginase


Enzyme replacement therapy


Acid alpha-glucosidase






N-acetylgalactosamine-6-sulfate sulfatase


Acid beta-glucosidase


Gaucher disease
















Plasminogen activator inhibitor-1


Phenylalanine ammonia lyase


Pharmacological chaperone therapy


Protein data bank


Polyethylene glycol




N-sulfoglucosamine sulfohydrolase


Tissue plasminogen activator


Urokinase-type plasminogen activator


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© Springer Nature Singapore Pte Ltd. 2019

Authors and Affiliations

  1. 1.Turku Centre for BiotechnologyUniversity of Turku and Åbo Akademi UniversityTurkuFinland

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