Treatment of genetic diseases

著者

書誌事項

Treatment of genetic diseases

edited by Robert J. Desnick

Churchill Livingstone, 1991

大学図書館所蔵 件 / 全5件

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注記

Includes bibliographical references and index

内容説明・目次

内容説明

This text examines the various techniques available for the treatment of genetic diseases and is aimed at medical geneticists, paediatricians and haematologists. The 66 contributors, from the USA, France, The Netherlands, Japan, Australia and the USSR consider metabolic therapy, bone marrow transplantation, enzyme replacement therapy, gene transfer strategies and in-utero therapy.

目次

  • Part 1 Metabolic therapy: Nutritional therapy in inborn errors of metabolism
  • approaches to the dietary management of hyperphenylalaninemia
  • advances in the treatment of amino acid and organic acid disorders
  • therapeutic applications of l-carnitine in metabolic disorders
  • treatment of urea cycle disorders
  • therapy for cystinosis
  • therapy for x-linked adrenoleukodystrophy. Part 2 Enzyme replacement therapy: Enzyme replacement - overview and prospects
  • enzyme replacement therapy for type I Gaucher disease
  • PEG-enzyme replacement therapy for adenosine deaminase deficiency. Part 3 Bone marrow transplantation: Transplantation in animal model systems
  • bone marrow transplantation for storage diseases
  • bone marrow transplantation as treatment for globoid cell leukodystrophy. Part IV Gene therapy and in-utero therapy: Human gene therapy - strategies and prospects for inborn errors of metabolism
  • gene transfer into hematopoietic and skin cells
  • delivery and expression of genes in hepatocycles
  • herpes simplex virus a vector for neurones
  • inutero therapy for genetic diseases.

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