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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...

    Morgan L. Maeder, Michael Stefanidakis, Christopher J. Wilson in Nature Medicine (2019)

  2. Article

    Open Access

    UDiTaS™, 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...

    Georgia Giannoukos, Dawn M. Ciulla, Eugenio Marco, Hayat S. Abdulkerim in BMC Genomics (2018)

  3. Article

    Hsp90 Inhibition Results in Glucocorticoid Receptor Degradation in Association with Increased Sensitivity to Paclitaxel in Triple-Negative Breast Cancer

    Targetable molecular drivers for triple-negative breast cancer (TNBC) have been difficult to identify; therefore, standard treatment remains limited to conventional chemotherapy. Recently, new-generation small...

    Abena S. Agyeman, Wesley J. Jun, David A. Proia, Caroline R. Kim in Hormones and Cancer (2016)

  4. No Access

    Article

    Serum and glucocorticoid-regulated kinase 1 (SGK1) activation in breast cancer: requirement for mTORC1 activity associates with ER-alpha expression

    Mammalian target of rapamycin (mTOR) is an attractive target for cancer treatment. While rapamycin and its derivatives (e.g., everolimus) have been shown to inhibit mTOR signaling and cell proliferation in pre...

    Ben A. Hall, Tae Yeon Kim, Maxwell N. Skor in Breast Cancer Research and Treatment (2012)