Research ArticleDrug Discovery and Translational Medicine
Identification and Characterization of Modified Antisense Oligonucleotides Targeting DMPK in Mice and Nonhuman Primates for the Treatment of Myotonic Dystrophy Type 1
Sanjay K. Pandey, Thurman M. Wheeler, Samantha L. Justice, Aneeza Kim, Husam S. Younis, Danielle Gattis, Dominic Jauvin, Jack Puymirat, Eric E. Swayze, Susan M. Freier, C. Frank Bennett, Charles A. Thornton and A. Robert MacLeod
Journal of Pharmacology and Experimental Therapeutics November 2015, 355 (2) 329-340; DOI: https://doi.org/10.1124/jpet.115.226969
Sanjay K. Pandey
Isis Pharmaceuticals Inc., Carlsbad, CA (S.K.P., S.L.J., A.K., H.S.Y., D.G., E.E.S., S.M.F., C.F.B., A.R.M.); Department of Neurology and Center of Neural Development and Disease, University of Rochester, Rochester, New York (T.M.W., C.A.T.); Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts (T.M.W.); and Department of Human Genetics, Centre Hospitalier Universitaire de Quebec, Quebec City, Canada (D.J., J.P.)
Thurman M. Wheeler
Isis Pharmaceuticals Inc., Carlsbad, CA (S.K.P., S.L.J., A.K., H.S.Y., D.G., E.E.S., S.M.F., C.F.B., A.R.M.); Department of Neurology and Center of Neural Development and Disease, University of Rochester, Rochester, New York (T.M.W., C.A.T.); Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts (T.M.W.); and Department of Human Genetics, Centre Hospitalier Universitaire de Quebec, Quebec City, Canada (D.J., J.P.)
Samantha L. Justice
Isis Pharmaceuticals Inc., Carlsbad, CA (S.K.P., S.L.J., A.K., H.S.Y., D.G., E.E.S., S.M.F., C.F.B., A.R.M.); Department of Neurology and Center of Neural Development and Disease, University of Rochester, Rochester, New York (T.M.W., C.A.T.); Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts (T.M.W.); and Department of Human Genetics, Centre Hospitalier Universitaire de Quebec, Quebec City, Canada (D.J., J.P.)
Aneeza Kim
Isis Pharmaceuticals Inc., Carlsbad, CA (S.K.P., S.L.J., A.K., H.S.Y., D.G., E.E.S., S.M.F., C.F.B., A.R.M.); Department of Neurology and Center of Neural Development and Disease, University of Rochester, Rochester, New York (T.M.W., C.A.T.); Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts (T.M.W.); and Department of Human Genetics, Centre Hospitalier Universitaire de Quebec, Quebec City, Canada (D.J., J.P.)
Husam S. Younis
Isis Pharmaceuticals Inc., Carlsbad, CA (S.K.P., S.L.J., A.K., H.S.Y., D.G., E.E.S., S.M.F., C.F.B., A.R.M.); Department of Neurology and Center of Neural Development and Disease, University of Rochester, Rochester, New York (T.M.W., C.A.T.); Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts (T.M.W.); and Department of Human Genetics, Centre Hospitalier Universitaire de Quebec, Quebec City, Canada (D.J., J.P.)
Danielle Gattis
Isis Pharmaceuticals Inc., Carlsbad, CA (S.K.P., S.L.J., A.K., H.S.Y., D.G., E.E.S., S.M.F., C.F.B., A.R.M.); Department of Neurology and Center of Neural Development and Disease, University of Rochester, Rochester, New York (T.M.W., C.A.T.); Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts (T.M.W.); and Department of Human Genetics, Centre Hospitalier Universitaire de Quebec, Quebec City, Canada (D.J., J.P.)
Dominic Jauvin
Isis Pharmaceuticals Inc., Carlsbad, CA (S.K.P., S.L.J., A.K., H.S.Y., D.G., E.E.S., S.M.F., C.F.B., A.R.M.); Department of Neurology and Center of Neural Development and Disease, University of Rochester, Rochester, New York (T.M.W., C.A.T.); Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts (T.M.W.); and Department of Human Genetics, Centre Hospitalier Universitaire de Quebec, Quebec City, Canada (D.J., J.P.)
Jack Puymirat
Isis Pharmaceuticals Inc., Carlsbad, CA (S.K.P., S.L.J., A.K., H.S.Y., D.G., E.E.S., S.M.F., C.F.B., A.R.M.); Department of Neurology and Center of Neural Development and Disease, University of Rochester, Rochester, New York (T.M.W., C.A.T.); Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts (T.M.W.); and Department of Human Genetics, Centre Hospitalier Universitaire de Quebec, Quebec City, Canada (D.J., J.P.)
Eric E. Swayze
Isis Pharmaceuticals Inc., Carlsbad, CA (S.K.P., S.L.J., A.K., H.S.Y., D.G., E.E.S., S.M.F., C.F.B., A.R.M.); Department of Neurology and Center of Neural Development and Disease, University of Rochester, Rochester, New York (T.M.W., C.A.T.); Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts (T.M.W.); and Department of Human Genetics, Centre Hospitalier Universitaire de Quebec, Quebec City, Canada (D.J., J.P.)
Susan M. Freier
Isis Pharmaceuticals Inc., Carlsbad, CA (S.K.P., S.L.J., A.K., H.S.Y., D.G., E.E.S., S.M.F., C.F.B., A.R.M.); Department of Neurology and Center of Neural Development and Disease, University of Rochester, Rochester, New York (T.M.W., C.A.T.); Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts (T.M.W.); and Department of Human Genetics, Centre Hospitalier Universitaire de Quebec, Quebec City, Canada (D.J., J.P.)
C. Frank Bennett
Isis Pharmaceuticals Inc., Carlsbad, CA (S.K.P., S.L.J., A.K., H.S.Y., D.G., E.E.S., S.M.F., C.F.B., A.R.M.); Department of Neurology and Center of Neural Development and Disease, University of Rochester, Rochester, New York (T.M.W., C.A.T.); Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts (T.M.W.); and Department of Human Genetics, Centre Hospitalier Universitaire de Quebec, Quebec City, Canada (D.J., J.P.)
Charles A. Thornton
Isis Pharmaceuticals Inc., Carlsbad, CA (S.K.P., S.L.J., A.K., H.S.Y., D.G., E.E.S., S.M.F., C.F.B., A.R.M.); Department of Neurology and Center of Neural Development and Disease, University of Rochester, Rochester, New York (T.M.W., C.A.T.); Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts (T.M.W.); and Department of Human Genetics, Centre Hospitalier Universitaire de Quebec, Quebec City, Canada (D.J., J.P.)
A. Robert MacLeod
Isis Pharmaceuticals Inc., Carlsbad, CA (S.K.P., S.L.J., A.K., H.S.Y., D.G., E.E.S., S.M.F., C.F.B., A.R.M.); Department of Neurology and Center of Neural Development and Disease, University of Rochester, Rochester, New York (T.M.W., C.A.T.); Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts (T.M.W.); and Department of Human Genetics, Centre Hospitalier Universitaire de Quebec, Quebec City, Canada (D.J., J.P.)
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In this issue
Research ArticleDrug Discovery and Translational Medicine
Antisense Targeting of DMPK for Treatment of DM1
Sanjay K. Pandey, Thurman M. Wheeler, Samantha L. Justice, Aneeza Kim, Husam S. Younis, Danielle Gattis, Dominic Jauvin, Jack Puymirat, Eric E. Swayze, Susan M. Freier, C. Frank Bennett, Charles A. Thornton and A. Robert MacLeod
Journal of Pharmacology and Experimental Therapeutics November 1, 2015, 355 (2) 329-340; DOI: https://doi.org/10.1124/jpet.115.226969
Research ArticleDrug Discovery and Translational Medicine
Antisense Targeting of DMPK for Treatment of DM1
Sanjay K. Pandey, Thurman M. Wheeler, Samantha L. Justice, Aneeza Kim, Husam S. Younis, Danielle Gattis, Dominic Jauvin, Jack Puymirat, Eric E. Swayze, Susan M. Freier, C. Frank Bennett, Charles A. Thornton and A. Robert MacLeod
Journal of Pharmacology and Experimental Therapeutics November 1, 2015, 355 (2) 329-340; DOI: https://doi.org/10.1124/jpet.115.226969
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