Antisense oligonucleotide therapy rescues aggresome formation in a novel spinocerebellar ataxia type 3 human embryonic stem cell line.

Moore, Lauren R; Keller, Laura; Bushart, David D; Delatorre, Rodrigo G; Li, Duojia; McLoughlin, Hayley S; do Carmo Costa, Maria; Shakkottai, Vikram G; Smith, Gary D; Paulson, Henry L

Moore, Lauren R; Keller, Laura; Bushart, David D; Delatorre, Rodrigo G; Li, Duojia; McLoughlin, Hayley S; do Carmo Costa, Maria; Shakkottai, Vikram G; Smith, Gary D; Paulson, Henry L.

Afiliación

Moore LR; Department of Neurology, 109 Zina Pitcher Place, University of Michigan, Ann Arbor, MI 48109, United States; Neuroscience Graduate Program, University of Michigan, Ann Arbor, MI 48109, United States.
Keller L; MStem Cell Laboratories, University of Michigan, Ann Arbor, MI 48109, United States.
Bushart DD; Department of Neurology, 109 Zina Pitcher Place, University of Michigan, Ann Arbor, MI 48109, United States.
Delatorre RG; Department of Neurology, 109 Zina Pitcher Place, University of Michigan, Ann Arbor, MI 48109, United States.
Li D; Department of Neurology, 109 Zina Pitcher Place, University of Michigan, Ann Arbor, MI 48109, United States.
McLoughlin HS; Department of Neurology, 109 Zina Pitcher Place, University of Michigan, Ann Arbor, MI 48109, United States.
do Carmo Costa M; Department of Neurology, 109 Zina Pitcher Place, University of Michigan, Ann Arbor, MI 48109, United States.
Shakkottai VG; Department of Neurology, 109 Zina Pitcher Place, University of Michigan, Ann Arbor, MI 48109, United States.
Smith GD; MStem Cell Laboratories, University of Michigan, Ann Arbor, MI 48109, United States; Departments of Ob/Gyn, Physiology, Urology, University of Michigan, Ann Arbor, MI 48109, United States.
Paulson HL; Department of Neurology, 109 Zina Pitcher Place, University of Michigan, Ann Arbor, MI 48109, United States. Electronic address: henryp@med.umich.edu.

Stem Cell Res ; 39: 101504, 2019 08.

Article en En | MEDLINE | ID: mdl-31374463

Asunto(s)

Células Madre Embrionarias Humanas/metabolismo; Enfermedad de Machado-Joseph/genética; Oligonucleótidos Antisentido/genética; Ataxina-3/genética; Células Cultivadas; Electrofisiología; Humanos; Immunoblotting; Inmunohistoquímica; Masculino

Palabras clave

Aggresome; Antisense oligonucleotide; Ataxin-3; Machado-Joseph disease; Neurodegeneration; Polyglutamine disease

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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Oligonucleótidos Antisentido / Enfermedad de Machado-Joseph / Células Madre Embrionarias Humanas Límite: Humans / Male Idioma: En Revista: Stem Cell Res Año: 2019 Tipo del documento: Article País de afiliación: Estados Unidos

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