Hope for Humans and Dogs with Genetic Disorders
X-linked myotubular myopathy is a particularly nasty genetic disorder. It affects boys who seem healthy at birth, only to have their muscles waste away until they can’t support their bodies anymore — or even breathe — over the course of a few years. Humans are not the only animal with the mutation that causes this disorder. Puppies (also male) suffer and ultimately die from it, as well. But from tragedy, scientists are now bringing us hope: if these affected puppies can teach us how to treat x-linked myotubular myopathy, both species will ultimately benefit:
Gene Therapy Saves Puppies From A Fatal Disease—And Maybe Us Next
The dogs who were given a treatment that repaired their defective myotubularin gene avoided the crippling muscle degeneration that killed the placebo-treated dogs by week 17. And by the ninth month of study, the saved puppies’ muscle and neurological function continued to match readings from healthy dogs, particularly for those that got the highest doses.
The findings, building on an earlier proof-of-concept study of dogs and mice by the researchers, signal that a scaled-up treatment could save the lives of boys with the same sort of genetic flaw.
Animal science working at the genetic level to improve the welfare and lives of multiple species — this is just some fantastic work!
PS. if you were at our annual conference last November and this sounds familiar, you aren’t imagining it — there was an inspiring (and tear-jerking) presentation about the animal-based research that is bringing us closer to a cure for x-linked myotubular myopathy. This would be a good time to remind you to plan for the 2017 NAIA conference, Oct 2-4 in Washington DC!