Method of treating diseases associated with myd88 pathways using crispr-gndm system
AssigneeModalis Therapeutics Corporation
InventorsTetsuya YAMAGATA, Yuanbo QIN, lain Robert THOMPSON, Haruhiko MORITA
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AssigneeModalis Therapeutics Corporation
InventorsTetsuya YAMAGATA, Yuanbo QIN, lain Robert THOMPSON, Haruhiko MORITA
Estimating the term…
The present invention provides a method of treating a disease associated with activated MYD88 signaling in a subject, including suppressing MYD88 expression in a subject by targeting an expression regulatory region of MYD88 gene by using a CRISPR-Guide Nucleotide Directed Modulation (GNDM), without affecting the expression of an adjacent ACAA1 gene. Also, provided is a CRISPR-GNDM system for suppressing MYD88, including expression comprising (a) a protein selected from the group consisting of dCas9 or dCpf1, a fusion protein of dCas9 or dCpf1 and Kruppel associated box (KRAB) and (b) a guide RNA (gRNA) targeting an expression regulatory region of MYD88 gene.
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