CRISPRa-induced upregulation of human <i>LAMA1</i> compensates for <i>LAMA2</i>-deficiency in Merosin-deficient congenital muscular dystrophy.

Arockiaraj, Annie I; Johnson, Marie A; Munir, Anushe; Ekambaram, Prasanna; Lucas, Peter C; McAllister-Lucas, Linda M; Kemaladewi, Dwi U

CRISPRa-induced upregulation of human LAMA1 compensates for LAMA2-deficiency in Merosin-deficient congenital muscular dystrophy.

Arockiaraj, Annie I; Johnson, Marie A; Munir, Anushe; Ekambaram, Prasanna; Lucas, Peter C; McAllister-Lucas, Linda M; Kemaladewi, Dwi U.

Afiliación

Arockiaraj AI; Department of Human Genetics, School of Public Health, University of Pittsburgh, Pittsburgh, USA.
Johnson MA; Department of Pediatrics, University of Pittsburgh School of Medicine, Pittsburgh, USA.
Munir A; Department of Pediatrics, University of Pittsburgh School of Medicine, Pittsburgh, USA.
Ekambaram P; Department of Human Genetics, School of Public Health, University of Pittsburgh, Pittsburgh, USA.
Lucas PC; Department of Pediatrics, University of Pittsburgh School of Medicine, Pittsburgh, USA.
McAllister-Lucas LM; Department of Pediatrics, University of Pittsburgh School of Medicine, Pittsburgh, USA.
Kemaladewi DU; Department of Pathology, University of Pittsburgh School of Medicine, Pittsburgh, USA.

bioRxiv ; 2023 Mar 07.

Article en En | MEDLINE | ID: mdl-36945402

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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Tipo de estudio: Prognostic_studies Idioma: En Revista: BioRxiv Año: 2023 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Estados Unidos

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