Treatment of Genetic Diseases

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.