Gene therapy for Gaucher disease
Ohashi, T.
Nihon Rinsho. Japanese Journal of Clinical Medicine 53(12): 3089-3094
1995
ISSN/ISBN: 0047-1852 PMID: 8577064 Document Number: 453862
Gaucher disease is a lysosomal storage disease which is characterized by deficient activity of lysosomal enzyme, known as glucocerebrosidase. This resulted in progressive accumulation of glucocerebroside only in bone marrow derived macrophages. This unique pathophysiology makes Gaucher disease an excellent candidate of gene therapy based on transferring therapeutic gene to hematopoietic stem cell. The extensive study based on transferring therapeutic gene to hematopoietic stem cell. The extensive study was already done using mouse in vivo system and human in vitro system. In mouse system, all of the macrophages from long term reconstituted mice were transduced. In human system, 40 approximately 60% of bone marrow progenitor cells were transduced. Because of this success, the clinical protocol for gene therapy for Gaucher disease was approved by RAC and FDA. The clinical trial was started in this year. The brief history of development and current limitation of gene therapy for Gaucher disease were discussed.