Today’s Washington Post featured a report on work by a research team that was described in an FSH Society press release. The Post story, “How CRISPR could lead to a cure for muscular dystrophy,” provides an excellent description of the CRISPR/dCas9 technology that was used to suppress the expression of DUX4 and includes an educational video on CRISPR.
The story explains clearly what is novel about this research and why it could have applications to many other diseases: “In the article describing the work in Molecular Therapy, co-authors Charis Himeda, Takako Jones and Peter Jones highlight the important implications for similar types of genetic diseases: “With increasing evidence that the repeat genome (comprising nearly half the human genome) plays important roles in gene regulation, additional diseases will likely be found associated with aberrant repetitive genomic sequences,” they write. “We have provided the first evidence that the repeat genome can be targeted via the CRISPR system, which is likely to prove useful as this hitherto overlooked portion of the genome is decoded.”
It’s important to note that this study, while ground-breaking, is still in the “test tube” stage. It will take more years of effort to determine whether this strategy can lead to a treatment that is safe and effective in human patients.