An original research paper by Neha Dhoke, PhD has just been published in Cells: “A Novel CRISPR-Cas9 Strategy to Target DYSTROPHIN Mutations Downstream of Exon 44 in Patient-Specific DMD iPSCs.”  Dr. Dhoke is a Researcher 5 in the lab of Rita Perlingeiro, PhD. In the paper, Dr. Dhoke describes a novel gene knock-in approach to correct Duchenne Muscular Dystrophy (DMD) mutations downstream of exon 44. This technique may prove critical to the development of cell therapies for DMD.
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