Increased mtDNA Abundance and Improved Function in Human Barth Syndrome Patient Fibroblasts Following AAV-<i>TAZ</i> Gene Delivery.

Suzuki-Hatano, Silveli; Sriramvenugopal, Mughil; Ramanathan, Manash; Soustek, Meghan; Byrne, Barry J; Cade, W Todd; Kang, Peter B; Pacak, Christina A

Increased mtDNA Abundance and Improved Function in Human Barth Syndrome Patient Fibroblasts Following AAV-TAZ Gene Delivery.

Suzuki-Hatano, Silveli; Sriramvenugopal, Mughil; Ramanathan, Manash; Soustek, Meghan; Byrne, Barry J; Cade, W Todd; Kang, Peter B; Pacak, Christina A.

Afiliación

Suzuki-Hatano S; Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL 32610, USA.
Sriramvenugopal M; Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL 32610, USA.
Ramanathan M; Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL 32610, USA.
Soustek M; Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL 32610, USA.
Byrne BJ; Department of Molecular Genetics and Microbiology, University of Florida College of Medicine, Gainesville, FL 32610, USA.
Cade WT; Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL 32610, USA.
Kang PB; Department of Molecular Genetics and Microbiology, University of Florida College of Medicine, Gainesville, FL 32610, USA.
Pacak CA; Program in Physical Therapy, Washington University School of Medicine, St. Louis, MO 63110, USA.

Int J Mol Sci ; 20(14)2019 Jul 11.

Article en En | MEDLINE | ID: mdl-31336787

Asunto(s)

Síndrome de Barth/genética; Síndrome de Barth/metabolismo; ADN Mitocondrial; Fibroblastos/metabolismo; Dosificación de Gen; Factores de Transcripción/genética; Aciltransferasas; Síndrome de Barth/terapia; Fragmentación del ADN; Dependovirus/genética; Exones; Técnicas de Transferencia de Gen; Terapia Genética; Humanos; Mutación

Palabras clave

AAV; Barth syndrome; TMEM65; fibroblasts; gene therapy; mitochondrial disease; mtDNA copy numbers

Texto completo

Imprimir

XML

PubMed Links

Buscar en Google

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Factores de Transcripción / ADN Mitocondrial / Dosificación de Gen / Síndrome de Barth / Fibroblastos Límite: Humans Idioma: En Revista: Int J Mol Sci Año: 2019 Tipo del documento: Article País de afiliación: Estados Unidos

Texto completo

Imprimir

XML

PubMed Links

Buscar en Google