Vector systems for prenatal gene therapy: principles of retrovirus vector design and production.
Vectors derived from the Retroviridae family have several attributes required for successful gene delivery. Retroviral vectors have an adequate payload size for the coding regions of most genes; they are safe to handle and simple to produce. These vectors can be manipulated to target different cell types with low immunogenicity and can permanently insert genetic information into the host cells' genome. Retroviral vectors have been used in gene therapy clinical trials and successfully applied experimentally in vitro, in vivo, and in utero.
Molecular Immunology Unit, Wolfson Centre for Gene Therapy, UCL Institute of Child Health, London, UK. s.howe@ich.ucl.ac.uk
Steven J
SJ
Howe
Anil
A
Chandrashekran
D004797:Enzyme-Linked Immunosorbent Assay; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000096:biosynthesis* / Q000235:genetics*; D006801:Humans; D016133:Polymerase Chain Reaction; D011295:Prenatal Care / Q000379:methods*; D012190:Retroviridae / Q000235:genetics*; D017186:Titrimetry