Critical steps in the implementation of hematopoietic progenitor-cell gene therapy using ribozyme vectors: a laboratory protocol

Methods Mol Biol. 2004:252:599-616. doi: 10.1385/1-59259-746-7:599.

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.

MeSH terms

  • Genetic Therapy / methods*
  • Genetic Therapy / standards*
  • Genetic Vectors*
  • Hematopoietic Stem Cell Mobilization / methods
  • Humans
  • RNA, Catalytic / therapeutic use*
  • Stem Cell Transplantation / methods*
  • Transplantation, Autologous / methods
  • Transplantation, Autologous / standards

Substances

  • RNA, Catalytic