Alternative sources for better and safer products |
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Cell lines instead of placenta to treat Gaucher disease
Worldwide, approximately 5,000 people suffer from Gaucher disease. This rare lysosomal storage disease is caused by a deficiency in the enzyme glucocerebrosidase and ultimately leads to severe functional abnormalities. Gaucher disease can be distinguished in types I, II and III. Type I is traditionally treatable with the enzyme gluco-cerebrosidase, extracted from the human placenta. Since there are not enough human placentas available to extract sufficient product for full worldwide marketing, a recombinant product was developed. The company Genzyme Corporation expressed the gene coding for human beta-glucocerebrosidase in a Chinese Hamster Ovary (CHO) cell line. The recombinant cerebrosidase is highly clinically effective for the treatment of Gaucher disease type I, and offers a safe and unlimited supply to treat all patients worldwide.