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Drug Screening in a Dish

Drug Screening in a Dish

Dec 6, 2019 | CMT Research Updates, CMTRF Funded Research, Research News

At the CMT Research Foundation we are very much interested in using patient-derived stem cells called iPSCs for drug screening in order to develop therapies for CMT. Indeed, one of the first projects we funded was such a drug screening effort for CMT2A at Acurastem....
Gene Editing Used for the First Time in CMT

Gene Editing Used for the First Time in CMT

Nov 22, 2019 | CMT Research Updates, CMTRF Funded Research, Research News

Gene editing as a therapeutic tool for treating disease has been very much in the news recently.  The most commonly used gene editing tool to permanently fix the underlying genetic cause of a disease is CRISPR/Cas9. A recent paper demonstrates the first...
Chief Science Officer comments on recent work

Chief Science Officer comments on recent work

Nov 11, 2019 | CMT Research Updates, Foundation News, Research News

CMT Research Foundation Chief Science Officer Grace Pavlath, PhD comments on recent work by Morelli et al: Allele-specific RNA interference prevents neuropathy in Charcot-Marie-Tooth disease type 2D mouse models Some types of CMT result from the expression of a mutant...

Emerging Landscape of Modifier Genes in CMT

Oct 21, 2019 | CMT Research Updates, Research News

In CMT, there is a large variation in disease onset, severity, or progression, often seen among individuals with the same genetic subtype of a disease, even within a single family.  Genetic modifiers play important roles in such variations, known as heterogeneity....

Inherited Neuropathy Consortium Awarded $7.2M to Continue Studying, Improve Care for CMT

Oct 11, 2019 | CMT Research Updates, Research News

The National Institutes of Health has awarded the Inherited Neuropathies Consortium (INC) $7.2M over the next five years. This grant will be focused on improving clinical care and clinical research for CMT. The CMT Research Foundation is a proud partner of the INC....
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