Critical steps in the implementation of hematopoietic progenitor-cell gene therapy using ribozyme vectors: a laboratory protocol.
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.
Boyd
Maureen P
MP
Johnson & Johnson Research Pty Limited, Eveleigh Sydney, Australia.
Ngok
Frances K
FK
Todd
Alison V
AV
Symonds
Geoff P
GP
Macpherson
Janet L
JL
Amado
Rafael G
RG
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
D016337:RNA, Catalytic