Minigene-Containing Retroviral Vectors Using an Alphavirus/Retrovirus Hybrid Vector System: Production and Use

Retroviral vectors are optimal gene transfer vehicles for gene therapy, since they integrate into the target cell genome and therefore can provide permanent expression of a therapeutic gene. This is a particularly desired feature in gene therapy of monogenic inherited diseases, in which the ultimate goal is a single gene transfer event that would lead to life-long correction of the genetic defect. Moreover, retroviral vectors are thoroughly characterized, and their extensive use in preclinical and clinical studies has verified their efficacy and safety.