Select Publications

Erwood, S., Bily T.M.I., Lequyer J, Yan J, Gulati N, Brewer R.A., Zhou L, Pelletier L, Ivakine R.D., Cohn R.D. Saturation variant interpretation using CRISPR prime editing. Nature Biotechnology (2022)

 

Erwood, S., Laselva, O., Bily, T.M.I., Brewer, R.A.,  Rutherford, A.H., Bear, C.E., Ivakine, E.A. Allele-specific prevention of nonsense-mediated decay in cystic fibrosis using homology-independent genome editing Molecular Therapy – Methods and Clinical Development 17:1118-1128 (2020).

 

Erwood, S., Gu, B. Embryo-based large fragment knock-in in mammals: why, how and what’s next. Genes (Basel.) (2020).

 

Erwood, S., Brewer, R.A., Bily, T.M.I., Maino, E., Zhou, L., Cohn, R.D., Ivakine, E.A. Modeling Niemann–Pick disease type C in a human haploid cell line allows for patient variant characterization and clinical interpretation. Genome Research (2019).

 

Garcia, B., Lee, J., Edraki, A., Hidalgo-Reyes, Y., Erwood, S., Mir, A., Trost, C.N., Seroussi, U., Stanley, S.Y., Cohn, R.D., Claycomb, J.M., Sontheimer, E.J., Maxwell, K.L., Davidson, A.R. Anti-CRISPR AcrIIA5 potently inhibits all Cas9 homologs used for genome editing. Cell Reports (2019).

 

Kemaladewi, D. U., Bassi, P. S., Erwood, S., Al-Basha, D., Gawlik, K. I., Lindsay, K., Hyatt, E., Kember, R., Place, K. M., Marks, R.M., Durbeej, M., Prescott, S.A., Ivakine, E. A., Cohn, R. D. A mutation-independent approach for muscular dystrophy via upregulation of a modifier gene. Nature (2019).

 

Kemaladewi, D. U., Benjamin, J. S., Hyatt, E., Ivakine, E. A., Cohn, R. D. Increased polyamines as protective disease modifiers in congenital muscular dystrophy. Human Molecular Genetics (2018).

 

Dowling, J. J., D Gonorazky, H., Cohn, R. D., Campbell, C. Treating pediatric neuromuscular disorders: The future is now. Am. J. Med. Genet. A (2018).

 

Kemaladewi, D. U., Maino, E., Hyatt, E., Hou, H., Ding, M., Place, K. M., Xinyi, Z., Bassi, P. S., Baghestani, Z., Deshwar, A. G., Merico, D., Xiong, H. Y., Frey, B. J., Wilson, M. D., Ivakine, E. A., Cohn, R. D. Correction of a splicing defect in a mouse model of congenital muscular dystrophy type 1A using a homology-directed-repair-independent mechanism. Nature Medicine (2017).

 

Wong, T. W. Y. & Cohn, R. D. Therapeutic Applications of CRISPR/Cas for Duchenne Muscular Dystrophy. Current Gene Therapy (2017).

 

Kemaladewi, D. U. & Cohn, R. D. Exon Snipping in Duchenne Muscular Dystrophy. Trends Mol Med (2016).

 

Wojtal, D., Kemaladewi, D. U., Malam, Z., Abdullah, S., Wong, T. W., Hyatt, E., Baghestani, Z., Pereira, S., Stavropoulos, J., Mouly, V., Mamchaoui, K., Muntoni, F., Voit, T., Gonorazky, H. D., Dowling, J. J., Wilson, M. D., Mendoza-Londono, R., Ivakine, E. A., Cohn, R. D. Spell Checking Nature: Versatility of CRISPR/Cas9 for Developing Treatments for Inherited Disorders. Am. J. Hum. Genet. (2016).

 

Malam, Z. & Cohn, R. D. Stem cells on alert: priming quiescent stem cells after remote injury. Cell Stem Cell (2014).

 

Andres-Mateos, E., Brinkmeier, H., Burks, T. N., Mejias, R., Files, D. C., Steinberger, M., Soleimani, A., Marx, R., Slimmers, J. L., Lin, B., Finager Hedderick, E., Marr, T. G., Lin, B. M., Hourdé, C., Leinwand, L. A., Kuhl, D., Föller, M., Vogelsang, S., Hernandez-Diaz, I., Vaughan, D. K., Alvarez de la Rosa, D., Lang, F., Cohn, R. D. Activation of serum/glucocorticoid-induced kinase 1 (SGK1) is important to maintain skeletal muscle homeostasis and prevent atrophy. EMBO Mol Med (2013).

 

Cohn, R. D., Liang, H.-Y., Shetty, R., Abraham, T. & Wagner, K. R. Myostatin does not regulate cardiac hypertrophy or fibrosis. Neuromuscular Disorders (2007).

 

Cohn, R. D., Van Erp, C., Habashi, J. P., Soleimani, A. A., Klein, E. C., Lisi, M. T., Gamradt, M., ap Rhys, C. M., Holm, T. M., Loeys, B. L., Ramirez, F., Judge, D. P., Ward, C. W., Dietz, H. C. Angiotensin II type 1 receptor blockade attenuates TGF-beta-induced failure of muscle regeneration in multiple myopathic states. Nature Medicine (2007).

 

Cohn, R. D., Henry, M. D., Michele, D. E., Barresi, R., Saito, F., Moore, S. A., Flanagan, J. D., Skwarchuk, M. W., Robbins, M. E., Mendell, J. R., Williamson, R. A., Campbell, K. P. Disruption of DAG1 in differentiated skeletal muscle reveals a role for dystroglycan in muscle regeneration. Cell (2002).

 

Moore, S. A., Saito, F., Chen, J., Michele, D. E., Henry, M. D., Messing, A., Cohn., R. D., Ross-Barta, S. E., Westra, S., Williamson, R. A., Hoshi, T., Campbell, K. P. Deletion of brain dystroglycan recapitulates aspects of congenital muscular dystrophy. Nature (2002).

 

Michele, D. E., Barresi, R., Kanagawa, M., Saito, F., Cohn, R. D., Satz, J. S., Dollar, J., Nishino, I., Kelley, R. I., Somer, H., Straub, V., Mathews, K. D., Moore, S. A., Campbell, K. P. Post-translational disruption of dystroglycan-ligand interactions in congenital muscular dystrophies. Nature (2002).