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Critical steps in the implementation of hematopoietic progenitor-cell gene therapy using ribozyme vectors: a
Maureen P Boyd1, Frances K Ngok, Alison V Todd
1Johnson & Johnson Research Pty Limited, Eveleigh Sydney, Australia.
Abstract:
The implementation of a hematopoietic progenitor-cell gene-therapy program involves the performance of laboratory procedures and compliance with the current code of Good Manufacturing Practices. This chapter explains the multiple laboratory steps used in our recent Phase I gene transfer study for HIV. This study employed a retroviral vector to deliver an anti-HIV ribozyme to CD34+ hematopoietic progenitor cells.
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