[Gene therapy in lysosomal diseases].
The study of the mechanisms of secretion and recapture of lysosomal enzymes has lead to the proposal of a treatment of lysosomal diseases by enzyme replacement. Autologous implants of genetically modified cells which secrete enzymes ensure systemic distribution of the lacking enzyme. A procedure which permits reimplantation of genetically modified fibroblasts is described. The stable secretion of human glucuronidase by autologous fibroblasts was thus obtained in animal species. This approach should by applicable to the treatment of Hurler's syndrome by obtaining the production and distribution of alpha-L-iduronidase in patients lacking this enzyme by retroviral transfer of the human alpha-L-iduronidase gene to cultured fibroblasts and by preparation of implants.
Moullier
P
P
Laboratoire Rétrovirus et Transfert génétique, Institut Pasteur, Paris, France.
Salvetti
A
A
Bohl
D
D
Danos
O
O
Heard
J M
JM
D000818:Animals; D002415:Cats; D004285:Dogs; D005347:Fibroblasts; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods; D006801:Humans; D007068:Iduronidase / Q000235:genetics; D016464:Lysosomal Storage Diseases / Q000628:therapy*; D051379:Mice; D008059:Mucopolysaccharidosis I / Q000628:therapy; D051381:Rats; D012092:Replantation
D007068:Iduronidase