基于造血干细胞的基因治疗艾滋病。
Hematopoietic-stem-cell-based gene therapy for HIV disease.
机构信息
Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.
出版信息
Cell Stem Cell. 2012 Feb 3;10(2):137-47. doi: 10.1016/j.stem.2011.12.015.
Abstract
Although combination antiretroviral therapy can dramatically reduce the circulating viral load in those infected with HIV, replication-competent virus persists. To eliminate the need for indefinite treatment, there is growing interest in creating a functional HIV-resistant immune system through the use of gene-modified hematopoietic stem cells (HSCs). Proof of concept for this approach has been provided in the instance of an HIV-infected adult transplanted with allogeneic stem cells from a donor lacking the HIV coreceptor, CCR5. Here, we review this and other strategies for HSC-based gene therapy for HIV disease.
摘要
虽然联合抗逆转录病毒疗法可以显著降低感染 HIV 的人的循环病毒载量，但复制型病毒仍然存在。为了消除对无限期治疗的需求，人们越来越有兴趣通过使用基因修饰的造血干细胞（HSCs）来创建功能性抗 HIV 免疫系统。在接受来自缺乏 HIV 核心受体 CCR5 的供体的同种异体干细胞移植的 HIV 感染成人的情况下，已经为这种方法提供了概念验证。在这里，我们回顾了基于 HSC 的 HIV 疾病基因治疗的这种方法和其他策略。
相似文献
1
Hematopoietic-stem-cell-based gene therapy for HIV disease.
Cell Stem Cell. 2012 Feb 3;10(2):137-47. doi: 10.1016/j.stem.2011.12.015.
2
Coreceptor-Based Hematopoietic Stem Cell Gene Therapy for HIV Disease.
Curr Stem Cell Res Ther. 2019;14(7):591-597. doi: 10.2174/1574888X14666190523094556.
3
CCR5-targeted hematopoietic stem cell gene approaches for HIV disease: current progress and future prospects.
Curr Stem Cell Res Ther. 2012 Jul;7(4):310-7. doi: 10.2174/157488812800793108.
4
Knowledge From London and Berlin: Finding Threads to a Functional HIV Cure.
Front Immunol. 2021 May 27;12:688747. doi: 10.3389/fimmu.2021.688747. eCollection 2021.
5
Novel cell and gene therapies for HIV.
Cold Spring Harb Perspect Med. 2012 Oct 1;2(10):a007179. doi: 10.1101/cshperspect.a007179.
6
CCR5 Δ32 homozygous cord blood allogeneic transplantation in a patient with HIV: a case report.
Lancet HIV. 2015 Jun;2(6):e236-42. doi: 10.1016/S2352-3018(15)00083-1. Epub 2015 May 19.
7
The use of cell-delivered gene therapy for the treatment of HIV/AIDS.
Immunol Res. 2010 Dec;48(1-3):84-98. doi: 10.1007/s12026-010-8169-7.
8
Genetically modified hematopoietic stem cell transplantation for HIV-1-infected patients: can we achieve a cure?
Mol Ther. 2014 Feb;22(2):257-264. doi: 10.1038/mt.2013.264. Epub 2013 Nov 13.
9
Generation of an HIV-1-resistant immune system with CD34(+) hematopoietic stem cells transduced with a triple-combination anti-HIV lentiviral vector.
J Virol. 2012 May;86(10):5719-29. doi: 10.1128/JVI.06300-11. Epub 2012 Mar 7.
10
From Berlin to London: HIV-1 Reservoir Reduction Following Stem Cell Transplantation.
Curr HIV/AIDS Rep. 2020 Aug;17(4):385-393. doi: 10.1007/s11904-020-00505-2.
引用本文的文献
1
Clinical hematopoietic stem cell-based gene therapy.
Mol Ther. 2025 Jun 4;33(6):2663-2678. doi: 10.1016/j.ymthe.2025.04.029. Epub 2025 Apr 24.
2
Breaking Barriers to an HIV-1 Cure: Innovations in Gene Editing, Immune Modulation, and Reservoir Eradication.
Life (Basel). 2025 Feb 11;15(2):276. doi: 10.3390/life15020276.
3
Advancing towards HIV-1 remission: Insights and innovations in stem cell therapies.
Arch Stem Cell Ther. 2024;5(1):5-13. doi: 10.46439/stemcell.5.020.
4
Spotlight on the impact of viral infections on Hematopoietic Stem Cells (HSCs) with a focus on COVID-19 effects.
Cell Commun Signal. 2023 May 8;21(1):103. doi: 10.1186/s12964-023-01122-3.
5
A lentiviral vector B cell gene therapy platform for the delivery of the anti-HIV-1 eCD4-Ig-knob-in-hole-reversed immunoadhesin.
Mol Ther Methods Clin Dev. 2023 Feb 11;28:366-384. doi: 10.1016/j.omtm.2023.02.004. eCollection 2023 Mar 9.
6
Engineered Zinc Finger Protein Targeting 2LTR Inhibits HIV Integration in Hematopoietic Stem and Progenitor Cell-Derived Macrophages: In Vitro Study.
Int J Mol Sci. 2022 Feb 19;23(4):2331. doi: 10.3390/ijms23042331.
7
Stem Cell Therapy and Its Significance in HIV Infection.
Cureus. 2021 Aug 27;13(8):e17507. doi: 10.7759/cureus.17507. eCollection 2021 Aug.
8
Research and Clinical Significance of the Differentially Expressed Genes TP63 and LMO4 in Human Immunodeficiency Virus-Related Penile Squamous Cell Carcinoma.
Am J Mens Health. 2021 Mar-Apr;15(2):15579883211011380. doi: 10.1177/15579883211011380.
9
Experimental Treatment of SIV-Infected Macaques via Autograft of -Disrupted Hematopoietic Stem and Progenitor Cells.
Mol Ther Methods Clin Dev. 2020 Mar 13;17:520-531. doi: 10.1016/j.omtm.2020.03.004. eCollection 2020 Jun 12.
10
Comparison of various radioactive payloads for a human monoclonal antibody to glycoprotein 41 for elimination of HIV-infected cells.
Nucl Med Biol. 2020 Mar-Apr;82-83:80-88. doi: 10.1016/j.nucmedbio.2020.02.009. Epub 2020 Feb 19.
本文引用的文献
1
TAL effectors: customizable proteins for DNA targeting.
Science. 2011 Sep 30;333(6051):1843-6. doi: 10.1126/science.1204094.
2
Concise review: managing genotoxicity in the therapeutic modification of stem cells.
Stem Cells. 2011 Oct;29(10):1479-84. doi: 10.1002/stem.716.
3
On target? Tracing zinc-finger-nuclease specificity.
Nat Methods. 2011 Aug 30;8(9):725-6. doi: 10.1038/nmeth.1680.
4
Site-specific integration and tailoring of cassette design for sustainable gene transfer.
Nat Methods. 2011 Aug 21;8(10):861-9. doi: 10.1038/nmeth.1674.
5
An unbiased genome-wide analysis of zinc-finger nuclease specificity.
Nat Biotechnol. 2011 Aug 7;29(9):816-23. doi: 10.1038/nbt.1948.
6
Engineering HIV-resistant human CD4+ T cells with CXCR4-specific zinc-finger nucleases.
PLoS Pathog. 2011 Apr;7(4):e1002020. doi: 10.1371/journal.ppat.1002020. Epub 2011 Apr 14.
7
HIV disease progression despite suppression of viral replication is associated with exhaustion of lymphopoiesis.
Blood. 2011 May 12;117(19):5142-51. doi: 10.1182/blood-2011-01-331306. Epub 2011 Mar 24.
8
Successful targeting and disruption of an integrated reporter lentivirus using the engineered homing endonuclease Y2 I-AniI.
PLoS One. 2011 Feb 9;6(2):e16825. doi: 10.1371/journal.pone.0016825.
9
Long-term regulation of genetically modified primary hematopoietic cells in dogs.
Mol Ther. 2011 Jul;19(7):1287-94. doi: 10.1038/mt.2011.8. Epub 2011 Feb 15.
10
Clinical use of CCR5 inhibitors in HIV and beyond.
J Transl Med. 2011 Jan 27;9 Suppl 1(Suppl 1):S9. doi: 10.1186/1479-5876-9-S1-S9.
Zotero 插件