title: Critical steps in the implementation of hematopoietic progenitor-cell gene therapy using ribozyme vectors: a laboratory protocol.
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.
authors.affiliation: Johnson & Johnson Research Pty Limited, Eveleigh Sydney, Australia.
authors.forename: Maureen P
authors.initials: MP
authors.lastname: Boyd
authors.forename: Frances K
authors.initials: FK
authors.lastname: Ngok
authors.forename: Alison V
authors.initials: AV
authors.lastname: Todd
authors.forename: Geoff P
authors.initials: GP
authors.lastname: Symonds
authors.forename: Janet L
authors.initials: JL
authors.lastname: Macpherson
authors.forename: Rafael G
authors.initials: RG
authors.lastname: Amado
mesh_terms: D015316:Genetic Therapy / Q000379:methods* / Q000592:standards*; D005822:Genetic Vectors; D019650:Hematopoietic Stem Cell Mobilization / Q000379:methods; D006801:Humans; D016337:RNA, Catalytic / Q000627:therapeutic use*; D033581:Stem Cell Transplantation / Q000379:methods*; D014182:Transplantation, Autologous / Q000379:methods / Q000592:standards
chemical_list: D016337:RNA, Catalytic