[HTML][HTML] Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy

D Duan - Molecular Therapy, 2018 - cell.com
Duchenne muscular dystrophy (DMD) is a lethal muscle disease caused by dystrophin gene
mutation. Conceptually, replacing the mutated gene with a normal one would cure the …

Skeletal muscle fibrosis: an overview

MAA Mahdy - Cell and tissue research, 2019 - Springer
Extracellular matrix (ECM) is an essential component of skeletal muscle. It provides a
framework structure that holds myofibers and blood capillaries and nerves supplying the …

Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy

L Amoasii, JCW Hildyard, H Li, E Sanchez-Ortiz… - Science, 2018 - science.org
Mutations in the gene encoding dystrophin, a protein that maintains muscle integrity and
function, cause Duchenne muscular dystrophy (DMD). The deltaE50-MD dog model of DMD …

[HTML][HTML] Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models

CH Hakim, SRP Kumar, DO Pérez-López… - Nature …, 2021 - nature.com
Abstract Adeno-associated virus (AAV)-mediated CRISPR-Cas9 editing holds promise to
treat many diseases. The immune response to bacterial-derived Cas9 has been speculated …

Inhibition of ATP synthase reverse activity restores energy homeostasis in mitochondrial pathologies

R Acin‐Perez, C Benincá, L Fernandez del Rio… - The EMBO …, 2023 - embopress.org
The maintenance of cellular function relies on the close regulation of adenosine
triphosphate (ATP) synthesis and hydrolysis. ATP hydrolysis by mitochondrial ATP Synthase …

[HTML][HTML] The jam session between muscle stem cells and the extracellular matrix in the tissue microenvironment

M Loreti, A Sacco - NPJ Regenerative medicine, 2022 - nature.com
Skeletal muscle requires a highly orchestrated coordination between multiple cell types and
their microenvironment to exert its function and to maintain its homeostasis and regenerative …

Dystrophin–glycoprotein complex sequesters Yap to inhibit cardiomyocyte proliferation

Y Morikawa, T Heallen, J Leach, Y Xiao, JF Martin - Nature, 2017 - nature.com
The regenerative capacity of the adult mammalian heart is limited, because of the reduced
ability of cardiomyocytes to progress through mitosis. Endogenous cardiomyocytes have …

RNA splicing and disease: animal models to therapies

M Montes, BL Sanford, DF Comiskey, DS Chandler - Trends in Genetics, 2019 - cell.com
Alternative splicing of pre-mRNA increases genetic diversity, and recent studies estimate
that most human multiexon genes are alternatively spliced. If this process is not highly …

[HTML][HTML] GPR41 and GPR43 in obesity and inflammation–protective or causative?

Z Ang, JL Ding - Frontiers in immunology, 2016 - frontiersin.org
GPR41 and GPR43 are a pair of mammalian G protein-coupled receptors (GPCRs)
expressed in human adipocytes, colon epithelial cells, and peripheral blood mononuclear …

Comparative transcriptomics in human and mouse

A Breschi, TR Gingeras, R Guigó - Nature Reviews Genetics, 2017 - nature.com
Cross-species comparisons of genomes, transcriptomes and gene regulation are now
feasible at unprecedented resolution and throughput, enabling the comparison of human …