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Designer Nucleases: Gene-Editing Therapies using CCR5 as an Emerging Target in HIV

[ Vol. 17 , Issue. 5 ]


Maria João Almeida and Ana Matos*   Pages 306 - 323 ( 18 )


Acquired Immunodeficiency Syndrome (AIDS), caused by the Human Immunodeficiency Virus (HIV), is a life-threatening disorder that persists worldwide as a severe health problem. Since it was linked with the HIV attachment process, the Chemokine receptor, CCR5, has been at the development leading edge of several gene-based therapies. Given the shortcomings of the current antiretroviral treatment procedure and the non-availability of a licensed vaccine, the aptitude to modify complex genomes with Designer Nucleases has had a noteworthy impact on biotechnology. Over the last years, ZFN, TALEN and CRISPR/Cas9 gene-editing technology have appeared as a promising solution that mimics the naturally occurring CCR5/Δ32 mutation and permanently guarantees the absence of CCR5-expression on the surface of HIV target-cells, leading to a continuous resistance to the virus entry and, ultimately, proving that cellular immunization from infection could be, in fact, a conceivable therapeutic approach to finally achieve the long-awaited functional cure of HIV.


AIDS, HIV, CCR5, designer nucleases, CCR5/Δ32 mutation, functional cure.


Laboratory of Microbiology, Faculty of Pharmacy, University of Coimbra, Coimbra, Laboratory of Microbiology, Faculty of Pharmacy, University of Coimbra, Coimbra

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