Repression of phosphatidylinositol transfer protein a ameliorates the pathology of Duchenne muscular dystrophy
Vieira, Natassia M, Spinazzola, Janelle M, Alexander, Matthew S, Moreira, Yuri B, Kawahara, Genri, Gibbs, Devin E, Mead, Lillian C, Verjovski-Almeida, Sergio, Zatz, Mayana, Kunkel, Louis M
Published in Proceedings of the National Academy of Sciences - PNAS (06.06.2017)
Published in Proceedings of the National Academy of Sciences - PNAS (06.06.2017)
Get full text
Journal Article
Transgenic zebrafish model of DUX4 misexpression reveals a developmental role in FSHD pathogenesis
Pakula, Anna, Lek, Angela, Widrick, Jeffrey, Mitsuhashi, Hiroaki, Bugda Gwilt, Katlynn M, Gupta, Vandana A, Rahimov, Fedik, Criscione, June, Zhang, Yuanfan, Gibbs, Devin, Murphy, Quinn, Manglik, Anusha, Mead, Lillian, Kunkel, Louis
Published in Human molecular genetics (15.01.2019)
Published in Human molecular genetics (15.01.2019)
Get full text
Journal Article
Repression of phosphatidylinositol transfer protein α ameliorates the pathology of Duchenne muscular dystrophy
Vieira, Natassia M., Spinazzola, Janelle M., Alexander, Matthew S., Moreira, Yuri B., Kawahara, Genri, Gibbs, Devin E., Mead, Lillian C., Verjovski-Almeida, Sergio, Zatz, Mayana, Kunkel, Louis M.
Published in Proceedings of the National Academy of Sciences - PNAS (06.06.2017)
Published in Proceedings of the National Academy of Sciences - PNAS (06.06.2017)
Get full text
Journal Article
Applying genome-wide CRISPR-Cas9 screens for therapeutic discovery in facioscapulohumeral muscular dystrophy
Lek, Angela, Zhang, Yuanfan, Woodman, Keryn G, Huang, Shushu, DeSimone, Alec M, Cohen, Justin, Ho, Vincent, Conner, James, Mead, Lillian, Kodani, Andrew, Pakula, Anna, Sanjana, Neville, King, Oliver D, Jones, Peter L, Wagner, Kathryn R, Lek, Monkol, Kunkel, Louis M
Published in Science translational medicine (25.03.2020)
Published in Science translational medicine (25.03.2020)
Get more information
Journal Article