Regenerating blood: towards engineering HIV-1-resistant hematopoietic stem cells
Free to access editorial from our latest Regenerative Medicine issue by Gero Hütter
“...HIV‑1 gene therapy has opened the promise of a single shoot treatment that might replace, or at least amend, current antiretroviral lifelong treatment.”
It has been now three decades since HIV‑1 was first recognized and nearly 20 years since combined antiretroviral therapy (HAART) improved the outcome of the infection effectively. However, although this success story has helped to transform HIV‑1 infection into a more chronic disease, there is still no vaccine or curative treatment available. Additionally, medicines to treat HIV‑1, particularly the newest and most tolerable drugs, are expensive and only available in high income countries, whereas the peak of infected people live in low or middle income countries. [...] The only and most compelling evidence of a medical intervention that cured HIV‑1 infection has been a successful hematopoietic stem cell transplant in an adult infected patient (the ‘Berlin patient’) transferring donor derived cells from a individual homozygous for the CCR5-Δ32 deletion, which confers resistance against HIV‑1 transmission. The lack of rebounding virus after 6 years without therapy, the failure to isolate infectious virus and the fading HIV‑specific immune responses indicate that the patient has been effectively cured[7,8].
By Gero Hütter
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7. Hütter G, Nowak D, Mossner M et al.Long term control of HIV by CCR5 Delta32/Delta32 stem cell transplantation.
N. Engl. J. Med. 360(7), 692–698 (2009).
8. Yukl SA, Boritz E, Busch M et al. Challenges in detecting HIV persistence during potentially curative interventions: a study of the Berlin patient. PLoS Pathog. 9(5), e1003347 (2013)