For decades, some unluckydog lovers have [witnessed their puppies] seemingly healthy muscles literally waste awayuntil 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 asmyotubularin.Whats 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 defectivemyotubularin 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 forthose that got the highest doses.

The findingssignal 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 its possible that people wont 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.]

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post:Gene Therapy Saves Puppies From A Fatal DiseaseAnd Maybe Us Next

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Muscle-wasting disease in dogs cured using gene therapy, offering hope to suffering boys - Genetic Literacy Project