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Antisense oligonucleotides correct the familial dysautonomia splicing defect in IKBKAP transgenic mice.
Sinha, Rahul; Kim, Young Jin; Nomakuchi, Tomoki; Sahashi, Kentaro; Hua, Yimin; Rigo, Frank; Bennett, C Frank; Krainer, Adrian R.
Affiliation
  • Sinha R; Cold Spring Harbor Laboratory, Cold Spring Harbor, NY 11724, USA.
  • Kim YJ; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Stanford, CA 94305, USA.
  • Nomakuchi T; Cold Spring Harbor Laboratory, Cold Spring Harbor, NY 11724, USA.
  • Sahashi K; Stony Brook University School of Medicine, Stony Brook, NY 11790, USA.
  • Hua Y; Cold Spring Harbor Laboratory, Cold Spring Harbor, NY 11724, USA.
  • Rigo F; Stony Brook University School of Medicine, Stony Brook, NY 11790, USA.
  • Bennett CF; Cold Spring Harbor Laboratory, Cold Spring Harbor, NY 11724, USA.
  • Krainer AR; Department of Neurology, Nagoya University Graduate School of Medicine, Nagoya 466-8550, Japan.
Nucleic Acids Res ; 46(10): 4833-4844, 2018 06 01.
Article in En | MEDLINE | ID: mdl-29672717

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Carrier Proteins / Dysautonomia, Familial / Oligonucleotides, Antisense Type of study: Prognostic_studies Limits: Animals / Humans Language: En Journal: Nucleic Acids Res Year: 2018 Document type: Article Affiliation country: United States Country of publication: United kingdom

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Carrier Proteins / Dysautonomia, Familial / Oligonucleotides, Antisense Type of study: Prognostic_studies Limits: Animals / Humans Language: En Journal: Nucleic Acids Res Year: 2018 Document type: Article Affiliation country: United States Country of publication: United kingdom