Generation of two Duchenne muscular dystrophy patient-specific induced pluripotent stem cell lines DMD02 and DMD03 (MUNIi001-A and MUNIi003-A).
Stem Cell Res
; 40: 101562, 2019 10.
Article
em En
| MEDLINE
| ID: mdl-31526943
ABSTRACT
Duchenne muscular dystrophy (DMD) affects 13500-5000 newborn boys and manifests with progressive skeletal muscle wasting, respiratory failure and eventual heart failure. Symptoms show different onset from patients' childhood to the second decade of age. We reprogrammed fibroblasts from two independent DMD patients with a complete loss of dystrophin expression, carrying deletions of exons 45-50 and 48-50. The resulting hiPSCs show expression of pluripotency markers (NANOG, OCT4, SSEA4), differentiation capacity into all three germ layers, normal karyotype, genetic identity to the originating parental fibroblasts and the patient-specific dystrophin mutation.
Texto completo:
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Base de dados:
MEDLINE
Assunto principal:
Linhagem Celular
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Distrofia Muscular de Duchenne
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Células-Tronco Pluripotentes Induzidas
Idioma:
En
Ano de publicação:
2019
Tipo de documento:
Article