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  • Title: Multi-unit anti-BCR-ABL ribozyme therapy in chronic myelogenous leukemia.
    Author: Leopold LH, Shore SK, Reddy EP.
    Journal: Leuk Lymphoma; 1996 Aug; 22(5-6):365-73. PubMed ID: 8882949.
    Abstract:
    In this review, we summarize and update our data on the development of a multi-unit anti-BCR/ABL ribozyme. In vitro studies comparing several anti-BCR/ABL ribozymes demonstrated that a triple-unit ribozyme is the most efficient. Detailed kinetic analysis revealed this ribozyme to have a lower Kcat, most likely due to non homologous bases at restriction enzyme sites used in ribozyme construction. Delivery of this ribozyme to a BCR/ABL transformed cell line by a novel vehicle targeting the folate receptor resulted in a 3 log reduction in BCR/ABL mRNA when analyzed by RT-PCR. This delivery strategy reversed the IL-3 independence of this cell line. Retroviral vectors containing genes coding for the multi-unit ribozyme have been constructed and their use to effect BCR/ABL transformed cell biology is discussed.
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