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Article
A highly efficient transgene knock-in technology in clinically relevant cell types
Inefficient knock-in of transgene cargos limits the potential of cell-based medicines. In this study, we used a CRISPR nuclease that targets a site within an exon of an essential gene and designed a cargo temp...
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Article
Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10
Leber congenital amaurosis type 10 is a severe retinal dystrophy caused by mutations in the CEP290 gene1,2. We developed EDIT-101, a candidate genome-editing therapeutic, to remove the aberrant splice donor creat...
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Article
Open AccessUDiTaS™, a genome editing detection method for indels and genome rearrangements
Understanding the diversity of repair outcomes after introducing a genomic cut is essential for realizing the therapeutic potential of genomic editing technologies. Targeted PCR amplification combined with Nex...
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Article
Open AccessEfficient and robust RNA-seq process for cultured bacteria and complex community transcriptomes
We have developed a process for transcriptome analysis of bacterial communities that accommodates both intact and fragmented starting RNA and combines efficient rRNA removal with strand-specific RNA-seq. We ap...
