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2015 ; 7
(7
): 3910-36
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Bone Marrow Gene Therapy for HIV/AIDS
#MMPMID26193303
Herrera-Carrillo E
; Berkhout B
Viruses
2015[Jul]; 7
(7
): 3910-36
PMID26193303
show ga
Bone marrow gene therapy remains an attractive option for treating chronic
immunological diseases, including acquired immunodeficiency syndrome (AIDS)
caused by human immunodeficiency virus (HIV). This technology combines the
differentiation and expansion capacity of hematopoietic stem cells (HSCs) with
long-term expression of therapeutic transgenes using integrating vectors. In this
review we summarize the potential of bone marrow gene therapy for the treatment
of HIV/AIDS. A broad range of antiviral strategies are discussed, with a
particular focus on RNA-based therapies. The idea is to develop a durable gene
therapy that lasts the life span of the infected individual, thus contrasting
with daily drug regimens to suppress the virus. Different approaches have been
proposed to target either the virus or cellular genes encoding co-factors that
support virus replication. Some of these therapies have been tested in clinical
trials, providing proof of principle that gene therapy is a safe option for
treating HIV/AIDS. In this review several topics are discussed, ranging from the
selection of the antiviral molecule and the viral target to the optimal vector
system for gene delivery and the setup of appropriate preclinical test systems.
The molecular mechanisms used to formulate a cure for HIV infection are
described, including the latest antiviral strategies and their therapeutic
applications. Finally, a potent combination of anti-HIV genes based on our own
research program is described.
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