2016 Research Breakthroughs: 3 Areas to Watch

January 11th, 2016 by Dr. Mary Sunderland

It is notoriously difficult to predict the next big scientific discovery… but, it’s fun to try! Here are some of the research areas that we will be watching very closely over the coming year.

CRISPR

Everyone is talking about the new gene-editing tool: CRISPR.

Once an obscure laboratory tool, CRISPR became a media sensation last December when science and medical experts convened an international summit in Washington, DC to discuss its potential to transform biology – for better and for worse.

DNA is the stuff that genes are made of and CRISPR is a tool – sort of like a microscopic pair of scissors – that can cut DNA at specific spots. Importantly, after the DNA has been cut, CRISPR can be used to insert a new DNA sequence. For people living with inherited retinal diseases, CRISPR could be used to remove the faulty gene and then insert a functional copy of the gene. Through this basic process of replacing faulty genes, CRISPR has the potential to treat a variety of diseases (not just retinal diseases), which is why the biomedical community is so excited about it! However, CRISPR could also be used to create ‘designer babies’ which is why ethicists have been consulted about how to best govern its use.

Fortunately, we don’t have to worry too much about designer babies just yet because scientists have unanimously agreed that using CRISPR to alter human embryos would be irresponsible. The good news is that scientists are moving full speed ahead with studying how CRISPR can be used to develop new medical treatments.

Currently, CRISPR is only being tested in the lab – but we have high hopes that this technique will dramatically transform our ability to treat inherited retinal disease.

STEM CELLS

Here at the FFB we have been investing in stem cell science for more than a decade. Finally, potential stem cell treatments are being tested in the clinic! There are a number of active clinical trials that are testing if stem cells (and progenitor cells) can be used to treat age-related macular degeneration, Stargardt disease and retinitis pigmentosa. Currently, most of these trials are focusing on transplanting retinal pigmented epithelial (RPE) cells into diseased eyes. RPE cells provide support to photoreceptors, which are the eye’s light sensing cells. RPE cells are much simpler than photoreceptor cells. If we want to restore sight, we need to figure out how to replace damaged photoreceptors. We have high hopes for 2016 because 2015 finished off with two important scientific developments, including the FFB-funded work of Dr. Gilbert Bernier, who developed a technique to generate cone photoreceptors from stem cells. And research by a Japanese team that confirmed human stem cell-derived retina can form mature photoreceptors after transplantation.

COMBINING APPROACHES

Many scientists predict that treating blinding eye diseases will require a combination of different approaches. For example, scientists like Dr. Cathy Tsilfidis are considering how gene therapies might be combined with protective drugs therapies to ensure that cells will stop dying in both the short term and the long term. And scientists like Dr. Andras Nagy, are combining stem cell technologies and gene therapy approaches to develop an innovative single shot therapy for wet age-related macular degeneration. One of the greatest challenges of stem cell transplantation is getting the newly transplanted cells to stay alive. What if a neuroprotective drug could be delivered along with stem cells to help them to survive? FFB-funded scientists are trying to find answers. More discoveries and developments are on the way in 2016 – this is one predication that I’d be willing to bet on.