A producer cell containing a DNA sequence lacks functional env and functional gag-pol genes capable of producing a retroviral vector containing neither and its use in gene therapy

摘要

A producer cell capable of producing a replication defective retroviral vector in an infective retroviral vector particle comprises: a) a first DNA sequence encoding the replication defective retroviral vector comprising: (i) a defective retroviral genome lacking functional env and functional gag-pol genes but having the remaining components essential for retroviral function and (ii) at least one heterologous gene and b) a set of DNA sequences encoding packaging components env and gag-pol wherein the DNA sequence encoding env is present on a separate construct to the DNA sequence encoding gag-pol; which producer cell is a fresh cell from a subject and is capable of delivering the heterologous gene to a target cell within a subject by in vivo production of the replication defective vector particles.