Chung Janet, Rossi John J, Jung Ulrike
Division of Molecular & Cell Biology, Beckman Research Institute of the City of Hope, 1500 East Duarte Road, CA 91010, USA.
Future Virol. 2011 Nov 1;6(11):1319-1328. doi: 10.2217/fvl.11.113.
HIV-1 causes AIDS, a syndrome that affects millions of people globally. Existing HAART is efficient in slowing down disease progression but cannot eradicate the virus. Furthermore the severity of the side effects and the emergence of drug-resistant mutants call for better therapy. Gene therapy serves as an attractive alternative as it reconstitutes the immune system with HIV-resistant cells and could thereby provide a potential cure. The feasibility of this approach was first demonstrated with the 'Berlin patient', who was functionally cured from HIV/AIDS with undetectable HIV-1 viral load after transplantation of bone marrow harboring a naturally occurring CCR5 mutation that blocks viral entry. Here, we give an overview of the current status of HIV gene therapy and remaining challenges and obstacles.
人类免疫缺陷病毒1型（HIV-1）引发获得性免疫缺陷综合征（AIDS），这是一种影响全球数百万人的综合征。现有的高效抗逆转录病毒疗法（HAART）在减缓疾病进展方面很有效，但无法根除病毒。此外，副作用的严重性以及耐药突变体的出现需要更好的治疗方法。基因疗法是一种有吸引力的替代方案，因为它能用抗HIV的细胞重建免疫系统，从而有可能实现治愈。这种方法的可行性首先在“柏林病人”身上得到证明，该病人在移植了携带自然发生的CCR5突变（可阻断病毒进入）的骨髓后，其HIV-1病毒载量检测不到，从而功能性治愈了HIV/AIDS。在此，我们概述了HIV基因疗法的现状以及尚存的挑战和障碍。
