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Gene therapy for chronic myelogenous leukemia
C M Verfaillie1, R S McIvor, R C Zhao
1Division of Hematology, Oncology and Transplantation, Dept of Medicine and Cancer Center, University of Minnesota, Box 806 UMHC, 420 Delaware Street SE, Minneapolis, MN 55455, USA. verfa001@tc.umn.edu
Molecular Medicine Today
|August 4, 1999
Summary
Targeting the BCR-ABL fusion gene in chronic myelogenous leukemia (CML) offers new hope. Strategies aim to block BCR-ABL at multiple levels, potentially eliminating residual disease and normalizing malignant cells.
Area of Science:
- Hematology
- Molecular Biology
- Oncology
Background:
- Chronic myelogenous leukemia (CML) is driven by the BCR-ABL fusion gene, resulting from a specific chromosomal translocation.
- Hematopoietic stem cell transplantation (HSCT) can prolong survival, but residual malignant cells in the graft or host lead to relapse.
Purpose of the Study:
- To review experimental strategies targeting the BCR-ABL gene and its downstream products for CML treatment.
- To explore novel therapeutic approaches for overcoming CML relapse post-HSCT.
Main Methods:
- Review of experimental strategies targeting BCR-ABL at the gene, mRNA, and protein levels.
- Discussion of cotransduction of hematopoietic stem cells (HSCs) with anti-BCR-ABL antisense sequences and drug resistance genes.
Main Results:
- Multiple strategies are being developed to inhibit BCR-ABL activity.
- Cotransduction of HSCs with anti-BCR-ABL sequences and drug resistance genes shows promise for residual disease elimination.
Conclusions:
- Targeting BCR-ABL offers potential for improved CML management.
- Therapeutic strategies involving gene modification of HSCs may provide a way to eliminate residual CML and normalize malignant cells.