<i>PMS1</i> as a target for splice modulation to prevent somatic CAG repeat expansion in Huntington's disease.

McLean, Zachariah L; Gao, Dadi; Correia, Kevin; Roy, Jennie C L; Shibata, Shota; Farnum, Iris N; Valdepenas-Mellor, Zoe; Rapuru, Manasa; Morini, Elisabetta; Ruliera, Jayla; Gillis, Tammy; Lucente, Diane; Kleinstiver, Benjamin P; Lee, Jong-Min; MacDonald, Marcy E; Wheeler, Vanessa C; Pinto, Ricardo Mouro; Gusella, James F

PMS1 as a target for splice modulation to prevent somatic CAG repeat expansion in Huntington's disease.

McLean, Zachariah L; Gao, Dadi; Correia, Kevin; Roy, Jennie C L; Shibata, Shota; Farnum, Iris N; Valdepenas-Mellor, Zoe; Rapuru, Manasa; Morini, Elisabetta; Ruliera, Jayla; Gillis, Tammy; Lucente, Diane; Kleinstiver, Benjamin P; Lee, Jong-Min; MacDonald, Marcy E; Wheeler, Vanessa C; Pinto, Ricardo Mouro; Gusella, James F.

Afiliación

McLean ZL; Molecular Neurogenetics Unit, Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA 02114, USA.
Gao D; Department of Neurology, Harvard Medical School, Boston, MA 02115, USA.
Correia K; Medical and Population Genetics Program, the Broad Institute of M.I.T. and Harvard, Cambridge, MA 02142, USA.
Roy JCL; Molecular Neurogenetics Unit, Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA 02114, USA.
Shibata S; Department of Neurology, Harvard Medical School, Boston, MA 02115, USA.
Farnum IN; Medical and Population Genetics Program, the Broad Institute of M.I.T. and Harvard, Cambridge, MA 02142, USA.
Valdepenas-Mellor Z; Molecular Neurogenetics Unit, Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA 02114, USA.
Rapuru M; Molecular Neurogenetics Unit, Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA 02114, USA.
Morini E; Department of Neurology, Harvard Medical School, Boston, MA 02115, USA.
Ruliera J; Molecular Neurogenetics Unit, Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA 02114, USA.
Gillis T; Department of Neurology, Harvard Medical School, Boston, MA 02115, USA.
Lucente D; Medical and Population Genetics Program, the Broad Institute of M.I.T. and Harvard, Cambridge, MA 02142, USA.
Kleinstiver BP; Molecular Neurogenetics Unit, Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA 02114, USA.
Lee JM; Molecular Neurogenetics Unit, Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA 02114, USA.
MacDonald ME; Molecular Neurogenetics Unit, Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA 02114, USA.
Wheeler VC; Molecular Neurogenetics Unit, Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA 02114, USA.
Pinto RM; Department of Neurology, Harvard Medical School, Boston, MA 02115, USA.
Gusella JF; Molecular Neurogenetics Unit, Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA 02114, USA.

bioRxiv ; 2023 Jul 27.

Article en En | MEDLINE | ID: mdl-37547003

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Texto completo: 1 Bases de datos: MEDLINE Idioma: En Revista: BioRxiv Año: 2023 Tipo del documento: Article País de afiliación: Estados Unidos