Associated Papers
CRISPR/Cas9 gene editing in muscle-related genetic disorders: Restoring function and exercise capacity.
Wang Y et al.
Tissue & Cell, 2026
A review of recent studies on CRISPR/Cas9-mediated genome editing in a variety of muscle-related genetic disorders.
Lyu G et al.
Journal of Translational Medicine, 2025
CRISPR-Cas9 editing of a TNPO3 mutation in a muscle cell model of limb-girdle muscular dystrophy type D2.
Poyatos-García J et al.
Molecular Therapy. Nucleic Acids, 2023
LGMD D2 TNPO3-Related: From Clinical Spectrum to Pathogenetic Mechanism.
Costa R et al.
Frontiers in Neurology, 2022
Transcriptomic Evidence of the Immune Response Activation in Individuals With Limb Girdle Muscular Dystrophy Dominant 2 (LGMDD2) Contributes to Resistance to HIV-1 Infection.
Diez-Fuertes F et al.
Frontiers in Cell and Developmental Biology, 2022
A novel pathogenic variant in TNPO3 in a Hungarian family with limb-girdle muscular dystrophy 1F.
Pál E et al.
European Journal of Medical Genetics, 2019
Long term follow-up and further molecular and histopathological studies in the LGMD1F sporadic TNPO3-mutated patient.
Gibertini S et al.
Acta Neuropathologica Communications, 2018
Next-generation sequencing identifies transportin 3 as the causative gene for LGMD1F.
Torella A et al.
Plos One, 2013
Select a paper to view details