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Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy
Passini MA,
Bu J,
Richards AM,
Kinnecom C,
Sardi SP,
Stanek LM,
Hua ,
Rigo F,
Matson J,
Hung G,
Kaye EM,
Shihabuddin LS,
Krainer AR,
Bennett CF,
Cheng SH

Science Translational Medicine
  3 (72): Mar 2 2011   Elsevier PMC3140425

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