For decades, some unlucky dog lovers have [witnessed their puppies’] seemingly healthy muscles literally waste away…until they could no longer stand and breathe.[T]he genetic cause of this specific muscle-wasting disease…[is] a single mutation that left them unable to produce an essential protein known as myotubularin. What’s more, it was the exact kind of mutation and disease also long found in male human babies, too.
…[Researchers gave 10-week-old puppies] a treatment that repaired their defective myotubularin gene, [causing them to avoid] 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…signal that a scaled-up treatment could save the lives of boys with the same sort of genetic flaw.
The changes seen after a single treatment have lasted for several years in the small sample of dogs the team has raised. So it’s possible that people won’t need repeated doses or they would be infrequent, said Dr. Martin Childers of the University of Washington — a big positive, given how expensive gene therapy is today.[The study can be found here.]
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