Paula Canon, de la Keck School of Medicine de la Universidad de Carolina del Sur, nos expondrá las últimas novedades relativas a la edición genómica como una posible vía para la cura de la infección por VIH.

What news will the GeSIDA Congress show about the use of gene therapy as a formula to achieve a functional cure for HIV?

I will be presenting an overview of work by a large group of scientists, clinicians and biotech partners who are working together to develop gene editing technologies as a way to suppress HIV infection. Our focus was initially on making cells that are resistant to HIV by disrupting the gene that codes for the CCR5 co-receptor. I thought this was a perfect target, as we know that people are fine without the CCR5 gene. Disrupting a gene is also the easiest thing to do with gene editing tools such as zinc finger nucleases and CRISPR/Cas9. It is posible to target CCR5 in both T cells and hematopietic Stem cells (HSC) and my group has focused on developing te technology for HSC. Although clinical trials are ongoing, we do not yet have enough information from them to decide if this approach could lead to a functional cure.

So far, what have been the main results obtained with your current research project?

The clinical trials to date have shown safety – always an important first step, especially when something is being tried for the first time. In studies from other groups who are editing the CCR5 gene in T cells, there are also tantalising hints of effects against the viral reservoir, and of viral control, but these are still very smal numbers of patients.

What are the main challenges or obstacles you are facing in this process and how are you solving them?

One of the biggest challenges is being first to the clinic. In many ways we are trail-blazing for an exciting new type of treatment that I am sure will have applications in other diseases of the blood and immune systems. This makes extensive pre-clinical safety studies such an important part of what we are doing. I hope that once we have shown that gene editing can be done safely, other studies will proceed at a faster pace.

What is the current stage of your research project? What are the next steps you will take?

More recently, as a basic scientist, I have turned my attention to developing better ways to do gene editing that can introduce mutations into a gene, not just knock out its function. This is a lot more challenging to do. We are using this approach to make HIV-resistant versions of restriction factors such as Tetherin and TRIM5a, which could inhibit HIV if the virus had not evolved to circumvent their activity.

Could the functional cure for HIV be achieved only by gene-editing, or would it be necessary to combine this therapy with other techniques?

I dont know the answer to that yet. But gene editing can certainly be combined with other approaches. For example, its being combined with some immune therapies against HIV, such as engineering CAR T cells.

Based on the results of your research, would you dare to fix a date for a functional or even definitive cure of HIV?

I am of course optimistic. I do not have a date in my head, because amazing progress I cannot imagine happens all the time, and I think we will continue to see progress on a continuum.