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- W2046083532 abstract "EVERAL thousand metabolic diseases develop because of mutations or deletions in cellular genes, resulting in a deficiency or absence of the proteins required for cellular metabolism (1). In many of these inborn errors of metabolism the dysfunctionality of the liver is responsible for the onset and maintenance of the disease. Well-known examples are hemophilia A and B due to deficiencies of factor VIII and IX, respectively, or hypercholesterolemia and premature coronary disease due to inherited deficiencies of the low density lipoprotein (LDL) receptors that are required for the uptake of cholesterol-transporting lipoproteins. Other monogenic diseases primarily affecting liver function are Wilson’s disease, urea cycle disorders, lysosomal storage diseases and phenylketonuria. For some of these diseases, a possible method of treatment is to remove the deficient liver and to replace it with a functional organ (2). However, orthotopic liver transplantation is associated with considerable morbidity and mortality, and requires lifelong immunosuppression. An alternative mode of treatment of genetic diseases is the transfer of therapeutic nucleic acids into the affected organs. The most attractive concept would be the replacement of the defective gene by a copy of the functional gene. Unfortunately, no reliable and safe techniques allowing the site-specific integration of DNA into the human genome are available at the moment (3). Therefore, almost all methods of genetic treatment focus on simply transferring the therapeutic gene into somatic cells without replacing the abnormal gene. Gene transfer into the target organs can be per" @default.
- W2046083532 created "2016-06-24" @default.
- W2046083532 creator A5042895691 @default.
- W2046083532 creator A5055436543 @default.
- W2046083532 date "1995-12-01" @default.
- W2046083532 modified "2023-10-18" @default.
- W2046083532 title "Liver-directed gene therapy: molecular tools and current preclinical and clinical studies" @default.
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- W2046083532 doi "https://doi.org/10.1016/0168-8278(95)80044-1" @default.
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