Nalla Arun K, Trobridge Grant D
Pharmaceutical Sciences, College of Pharmacy, Washington State University Spokane, Spokane, WA 99202, USA.
School of Molecular Biosciences, Washington State University, Pullman, WA 99164, USA.
Biomedicines. 2016 Mar 29;4(2):8. doi: 10.3390/biomedicines4020008.
Stem cell gene therapy approaches for Human Immunodeficiency Virus (HIV) infection have been explored in clinical trials and several anti-HIV genes delivered by retroviral vectors were shown to block HIV replication. However, gammaretroviral and lentiviral based retroviral vectors have limitations for delivery of anti-HIV genes into hematopoietic stem cells (HSC). Foamy virus vectors have several advantages including efficient delivery of transgenes into HSC in large animal models, and a potentially safer integration profile. This review focuses on novel anti-HIV transgenes and the potential of foamy virus vectors for HSC gene therapy of HIV.
针对人类免疫缺陷病毒(HIV)感染的干细胞基因治疗方法已在临床试验中进行了探索,并且通过逆转录病毒载体递送的几种抗HIV基因显示出可阻断HIV复制。然而,基于γ逆转录病毒和慢病毒的逆转录病毒载体在将抗HIV基因递送至造血干细胞(HSC)方面存在局限性。泡沫病毒载体具有多个优点,包括在大型动物模型中能有效地将转基因递送至HSC,以及具有潜在更安全的整合模式。本综述聚焦于新型抗HIV转基因以及泡沫病毒载体用于HIV的HSC基因治疗的潜力。