{"id":90,"date":"2016-11-02T10:02:57","date_gmt":"2016-11-02T10:02:57","guid":{"rendered":"\/?p=90"},"modified":"2023-06-07T13:54:37","modified_gmt":"2023-06-07T13:54:37","slug":"5-most-important-considerations-when-testing-a-new-drug","status":"publish","type":"post","link":"https:\/\/lab.research.sickkids.ca\/ivakine\/5-most-important-considerations-when-testing-a-new-drug\/","title":{"rendered":"A mutation-independent approach for muscular dystrophy via upregulation of a modifier gene"},"content":{"rendered":"

A mutation-independent approach for muscular dystrophy via upregulation of a modifier gene<\/h1><\/div><\/div><\/div>
<\/a><\/span><\/div><\/div><\/div>

A CRISPR therapeutic<\/span><\/h2><\/div>

Here we report a mutation-independent strategy to upregulate the expression of a disease-modifying gene associated with congenital muscular dystrophy type 1A (MDC1A) using the CRISPR activation system in mice. <\/p>\n

We modulated the expression of Lama1 in a mouse model of MDC1A using an adeno-associated virus carrying a catalytically inactive Cas9, VP64 transactivators and single-guide RNAs that target the Lama1 promoter.<\/p>\n

Our data demonstrated the feasibility and therapeutic benefit of CRISPR-dCas9-mediated upregulation of Lama1, which may enable mutation-independent treatment for all patients with MDC1A. This approach has a broad applicability to a variety of disease-modifying genes and could serve as a therapeutic strategy for many inherited and acquired diseases.<\/p>\n<\/div>

read full publication here<\/span><\/a><\/div>
<\/div>
<\/div><\/div><\/div><\/div><\/div>\n","protected":false},"excerpt":{"rendered":"

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