Research Roundup: Gene-silencing drug gets FDA approval after 20 years; new drug may render liver transplants unnecessary and more!

Welcome to this week’s Research Roundup. These Friday posts aim to inform our readers about the many stories that relate to animal research each week. Do you have an animal research story we should include in next week’s Research Roundup? You can send it to us via our Facebook page or through the contact form on the website.

  • Gene-silencing drug gets approval after 20 year wait. The US FDA have approved the drug therapy, patisiran — used to silence genes associated with the disease hereditary transthyretin amyloidosis — by employing the technique of  RNA interference (RNAi). RNAi, which won the creators’ a Nobel Prize for work done 20 years ago, was pioneered in worms; although subsequent animal tests for safety and efficacy, prompted caution before introduction to human clinical trials. This is a good example of how basic research can in the longer term lead to application to humans, and how the regulatory process works to ensure human safety.

  • New drug treatment may render liver transplants unnecessary. The liver has a natural ability to repair itself but the ability to do so may be lost due to disease or excessive drug use. In a new study, researchers found that this loss of ability was due to early aging of the organ and that a chemical signal, TGFβ, may be responsible. Using mice and an experimental cancer therapy, they were able to block this chemical signal, increasing survival rates. While this work is still in the early stages, the potential value, given the ongoing organ crisis is great. Published in Science Translational Medicine.
  • Why eating leafy vegetables may decrease the risk of colon cancer. We all know that eating green leafy vegetables is good for the colon and can decrease cancer risk — but this is often attributed to fiber content. Now using mice and miniature bowels grown in the lab, researchers have found that it is the chemical, indole-3-carbinol, which may be responsible. Without this particular chemical in the diet, mice prone to cancer, rapidly developed tumours and vice versa. Replication of these results and assessment of generality to humans are obvious next steps, but these results are a promising first step. Published in Immunity.

  • Reversing blindness in mice. Scientists at the National Eye Institute have used gene therapy to restore vision in mice born with congenital blindness. This was done by reprogramming base retinal cells into functional rod photoreceptors. The research was initially carried out in zebrafish who regenerate these photoreceptors in their eyes by themselves. Now the scientists have used the zebrafish technique in blind mice through gene therapy. The next step is to test the visual abilities of the mice and begin testing the technique in human retina cells, in vitro. Published in Nature.
  • The gene that protects elephants from cancer. The bigger the animal, the higher the probability of cancer, or so you would think; this is called Peto’s paradox. Large animals such as elephants, aren’t more prone to cancer, and new research has identified that a unique gene, LIF6, may be responsible. LIF6, kills cells whose DNA have been damaged; a feature of cancer cells. This gene which is dormant in other species, became “active” in elephants and their distant relatives (proboscideans) somewhere in the course of evolution. Dr. Lynch, stated that these findings “…“might tell us something fundamental about cancer as a process. And if we’re lucky, it might tell us something about how to treat human disease.” Published in Cell Reports.
African bush elephant, Loxodonta africana

 

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