Use of Enzyme Artificial Cells for Genetic Enzyme Defects that Increase Systemic Substrates to Toxic Levels

The following sections are included:

• Introduction

  • General

  • Enzyme replacement therapy

• Acatalasemia: Congenital Defect in the Enzyme Catalase

• Preliminary Study on the Use of Catalase Artificial Cells to Replace Defective Catalase in Acatalasemia

  • Implantation of catalase artificial cells

  • Catalase artificial cells retained in a chamber for body fluid perfusion

• In Vivo Kinetics of Catalase Artificial Cells for Acatalasemic Mice

  • Summary

  • Methods of preparation of catalase artificial cells and experimental procedures

  • Characteristics of acatalasemic mice

  • Kinetics of recovery of injected perborate

• Immunological Studies on Catalase Artificial Cells in Acatalasemic Mice

  • Effects of immunizing doses of catalase solution and catalase artificial cells on antibody production

  • Permeability of membrane of collodion artificial cells to catalase antibody

  • Response of immunized acatalasemic mice to injections of catalase artificial cells or catalase solution

  • Appearance of catalase activity in the blood after injection of catalase solution or catalase artificial cells

• Conclusion from Results of Basic Study Using the Acatalasemic Mice Model

• Oral Xanthine Oxidase Artificial Cells in a Patient with Lesch-Nyhan Disease

  • Volume ratio of artificial cells/substrate solution

  • Effects of pH on enzyme activity

  • Patient with Lesch-Nyhan disease

• Conclusion Based on Study

• Phenylketonuria: Genetic Defect in Enzyme Phenylalanine Hydroxylase

  • Phenylketonuria

  • Enzyme replacement therapy

  • Oral PAL artificial cells for PKU based on novel theory of enterorecirculation of amino acids

• Research Leading to Proposal of Enterorecirculation of Amino Acids

  • Classical theory of the main source of amino acids in the intestinal lumen

  • Research to test the classical theory

  • Animals on 24 h of protein-free diet

  • Results of study

  • Proposed theory of extensive enterorecirculation of amino acids

• Oral Enzyme Artificial Cells for Genetic Enzyme Defects that Result in Elevated Systemic Amino Acid Levels

  • General design for study of use of oral PAL artificial cells in PKU rats

  • Methods of preparation ofcatalase artificial cells and experiment procedures

  • Phenylketonuria rat model

  • Intestinal phenylalanine

  • Plasma PHE levels in PKU rats

  • CSF cerebrospinal fluid in PKU rats

  • Growth as shown by body weight changes

  • Congenital PKU mice model

  • Summary discussion

  • Oral enzyme artificial cells to deplete other amino acids