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Nat Commun ; 12(1): 6215, 2021 10 28.
Artigo em Inglês | MEDLINE | ID: mdl-34711827

RESUMO

In phenylketonuria (PKU) patients, a genetic defect in the enzyme phenylalanine hydroxylase (PAH) leads to elevated systemic phenylalanine (Phe), which can result in severe neurological impairment. As a treatment for PKU, Escherichia coli Nissle (EcN) strain SYNB1618 was developed under Synlogic's Synthetic Biotic™ platform to degrade Phe from within the gastrointestinal (GI) tract. This clinical-stage engineered strain expresses the Phe-metabolizing enzyme phenylalanine ammonia lyase (PAL), catalyzing the deamination of Phe to the non-toxic product trans-cinnamate (TCA). In the present work, we generate a more potent EcN-based PKU strain through optimization of whole cell PAL activity, using biosensor-based high-throughput screening of mutant PAL libraries. A lead enzyme candidate from this screen is used in the construction of SYNB1934, a chromosomally integrated strain containing the additional Phe-metabolizing and biosafety features found in SYNB1618. Head-to-head, SYNB1934 demonstrates an approximate two-fold increase in in vivo PAL activity compared to SYNB1618.


Assuntos
Terapia Biológica , Proteínas de Escherichia coli/genética , Escherichia coli/enzimologia , Fenilalanina Amônia-Liase/genética , Fenilalanina/metabolismo , Fenilcetonúrias/metabolismo , Fenilcetonúrias/terapia , Técnicas Biossensoriais , Cinamatos , Escherichia coli/genética , Escherichia coli/metabolismo , Proteínas de Escherichia coli/metabolismo , Humanos , Fenilalanina Amônia-Liase/metabolismo , Engenharia de Proteínas
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