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Finding the Right CRISPR Targets for DM

CRISPR is an exploratory strategy with potential for treatment of RNA-triggered diseases. Will DNA or RNA targeting prove to be the best approach for DM?

Interaction of Age and Gender in DM2

Age and gender impact the onset and progression of DM2, but the pattern shows both similarities and differences from that of DM1.

RAN Translation in DM2

Toxic RNA-mediated mis-splicing and Repeat-Associated Non-AUG (RAN) translation proteins are impacted by free MBNL1 levels and contribute towards CNS pathogenesis in DM2.

Modifying Gene Editing Technology for DM

Investigators at the University of California San Diego, the University of Florida, and the National University of Singapore have recently reported early research that potentially ‘repurposes’ gene editing technology for a set of RNA disorders—myotonic dystrophy type 1 (DM1), myotonic dystrophy type 2 (DM2), a subset of Lou Gehrig’s disease (ALS) patients and Huntington’s disease. They have modified the Cas9 enzyme so it is targeted to toxic RNA, instead of the expanded DNA repeats in these diseases.

New Drosophila Models for DM1 and DM2

New fly models show a DM2 phenotype at least as severe as DM1 and may provide a platform for studies of genetic modifiers and candidate therapy screening in DM.

Do We Treat DM as a Brain Disease?

A recent review article makes the case that DM is a brain disease and that better understanding of and treatment strategies for the neurological consequences of DM are essential.

DM Highlights at American Academy of Neurology Annual Meeting

MDF staff recently attended the 2017 annual meeting of the American Academy of Neurology, in Boston, MA. Here are highlights from that meeting.

Telling the Quacks from the Cures

MDF is also happy to help you understand whether something is in a legitimate clinical trial, an approved therapy…or not.

Using Gene Editing to Correct DM

A potentially revolutionary technology may allow development of a drug for DM that can correct a patient’s DNA by selectively removing the expanded CTG and CCTG repeats in DM1 and DM2, respectively.

Gene Editing for DM

Gene editing is a potential avenue for therapy development in DM. With the safety, efficacy and delivery challenges, how do we get there?

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