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
Inhibition of autophagy rescues muscle atrophy in a LGMDD2 Drosophila model.
Blázquez-Bernal Á et al.
Faseb Journal : Official Publication of the Federation of American Societies for Experimental Biology, 2021
[The TNPO3 mutation that causes LGMD1F induces protection against HIV-1 infection].
Allamand V
Medecine Sciences : M/s, 2019
Novel mutation in TNPO3 causes congenital limb-girdle myopathy with slow progression.
Vihola A et al.
Neurology. Genetics, 2019
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