ColVI-CMD research at the ICG will be funded by CureCMD

The ICG is happy to announce, that Jeanette Erdmann and Franziska Haarich are amongst the recipients of Cure CMD´s 2020 Grant Awards. According to the CureCMD press release “Grants have been awarded in five subtypes: Dystroglycanopathy, Collagen VI, LAMA2, LMNA, and SELENON (SEPN1). The projects range from a study in mouse models, to new projects in gene therapy, to an extended grant focused on genetic modifiers.”

“The process was extremely competitive this year,” says Cure CMD’s Scientific Director, Dr. Gustavo Dziewczapolski. “In previous cycles we have had perhaps a dozen applicants. This year, we received a record-breaking 30 applications, which is thrilling because it indicates an expanded interest in CMD research — something we at Cure CMD have continued to cultivate over the last decade.”

The study by J. Erdmann and F. Haarich entitled: First steps towards an RNA-based therapy for COL6-MD using CRISPR interference will evaluate the unique characteristics of the gene copy (allele) carrying some of the more common Collagen VI dominant mutations, in order to target and inhibit the mutant allele and spare the unaffected allele. The team will evaluate a novel treatment strategy, called CRISPR interference (CRISPRi), to achieve suppression of the faulty gene. CRISPRi is a type of CRISPR technology, which may be safer by acting on RNA rather than directly on DNA.

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