Promising AAV.U7snRNAs vectors targeting <i>DMPK</i> improve DM1 hallmarks in patient-derived cell lines.

Almeida, Camila F; Robriquet, Florence; Vetter, Tatyana A; Huang, Nianyuan; Neinast, Reid; Hernandez-Rosario, Lumariz; Rajakumar, Dhanarajan; Arnold, W David; McBride, Kim L; Flanigan, Kevin M; Weiss, Robert B; Wein, Nicolas

Promising AAV.U7snRNAs vectors targeting DMPK improve DM1 hallmarks in patient-derived cell lines.

Almeida, Camila F; Robriquet, Florence; Vetter, Tatyana A; Huang, Nianyuan; Neinast, Reid; Hernandez-Rosario, Lumariz; Rajakumar, Dhanarajan; Arnold, W David; McBride, Kim L; Flanigan, Kevin M; Weiss, Robert B; Wein, Nicolas.

Afiliación

Almeida CF; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, United States.
Robriquet F; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, United States.
Vetter TA; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, United States.
Huang N; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, United States.
Neinast R; Center for Cardiovascular Research, Nationwide Children's Hospital, Columbus, OH, United States.
Hernandez-Rosario L; Center for Cardiovascular Research, Nationwide Children's Hospital, Columbus, OH, United States.
Rajakumar D; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, United States.
Arnold WD; Department of Neurology, The Ohio State University, Columbus, OH, United States.
McBride KL; Department of Physical Medicine and Rehabilitation, University of Missouri School of Medicine, Columbia, MO, United States.
Flanigan KM; Center for Cardiovascular Research, Nationwide Children's Hospital, Columbus, OH, United States.
Weiss RB; Department of Medical Genetics, University of Calgary, Calgary, AB, Canada.
Wein N; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, United States.

Front Cell Dev Biol ; 11: 1181040, 2023.

Article en En | MEDLINE | ID: mdl-37397246

Palabras clave

U7snRNA; aav; gene therapy; myotonic dystrophy; spliceopathy

Texto completo

Añadir a Mi BVS

Imprimir

XML

PubMed Links

Buscar en Google

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