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Identification and characterization of modified antisense oligonucleotides targeting DMPK in mice and nonhuman primates for the treatment of myotonic dystrophy type 1.
Pandey, Sanjay K; Wheeler, Thurman M; Justice, Samantha L; Kim, Aneeza; Younis, Husam S; Gattis, Danielle; Jauvin, Dominic; Puymirat, Jack; Swayze, Eric E; Freier, Susan M; Bennett, C Frank; Thornton, Charles A; MacLeod, A Robert.
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  • Pandey SK; 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,
  • Wheeler TM; 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,
  • Justice SL; 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,
  • Kim A; 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,
  • Younis HS; 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,
  • Gattis D; 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,
  • Jauvin D; 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,
  • Puymirat J; 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,
  • Swayze EE; 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,
  • Freier SM; 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,
  • Bennett CF; 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,
  • Thornton CA; 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,
  • MacLeod AR; 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,
J Pharmacol Exp Ther ; 355(2): 329-40, 2015 Nov.
Article en En | MEDLINE | ID: mdl-26330536
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Oligonucleótidos / Oligonucleótidos Antisentido / Proteína Quinasa de Distrofia Miotónica / Distrofia Miotónica Tipo de estudio: Diagnostic_studies Límite: Animals / Humans / Male Idioma: En Revista: J Pharmacol Exp Ther Año: 2015 Tipo del documento: Article Pais de publicación: Estados Unidos
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Oligonucleótidos / Oligonucleótidos Antisentido / Proteína Quinasa de Distrofia Miotónica / Distrofia Miotónica Tipo de estudio: Diagnostic_studies Límite: Animals / Humans / Male Idioma: En Revista: J Pharmacol Exp Ther Año: 2015 Tipo del documento: Article Pais de publicación: Estados Unidos