Welcome to this week’s Sunday Science. The big science news of the past week has been the latest advance in gene editing. This deserves a longer look, so I’ll be sending out a post on that later today/tomorrow. This week I’ve got a focus on medical/biological advances.
Firstly, after many years, the first gene therapy has been approved on NHS. This is for so-called “bubble baby” syndrome, which is a genetic condition called adenosine deaminase deficiency, or ADA-SCID. Sufferers have a severely compromised immune system and must live in a sterile environment as much as possible. It’s very rare, but usually fatal. The gene therapy replaces the faulty gene in bone marrow cells, which are then transplanted back to the patient, and is lifelong.
A new drug has been approved for treating sleeping sickness. This is an endemic and problematic parasitic disease in Africa, one of many “neglected tropical diseases.” This week’s featured image shows trypanosome parasites in a blood sample from an infected patient. I actually previously blogged about the potential of this drug in a piece about these diseases a while back, so it’s good to see it’s promise fulfilled. Previous treatments have had high toxicity or been difficult to administer: this is a pill to be taken orally.
Finally, a study has been done that shows for the first time how the brain maps connections from an artificial prosthetic limb, in those cases where where residual limb nerves are rerouted towards intact muscles and skin regions to control a robotic limb. (I recently blogged about prosthetics versus transplants here). There is surprisingly good control, but the brain doesn’t interpret them exactly as it does the original limb, so more work is needed to improve the mobility and sensation from these devices. Studies like this can help researchers design artificial limbs both more useful and more natural feeling to use.
Photo credit: CDC/Dr. Myron G. Schultz – CDC Public Health Image Library (PHIL), Public Domain, https://commons.wikimedia.org/w/index.php?curid=740877