[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 …
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 …
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 …
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 …
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 …
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 …
their microenvironment to exert its function and to maintain its homeostasis and regenerative …
Dystrophin–glycoprotein complex sequesters Yap to inhibit cardiomyocyte proliferation
The regenerative capacity of the adult mammalian heart is limited, because of the reduced
ability of cardiomyocytes to progress through mitosis. Endogenous cardiomyocytes have …
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 …
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?
GPR41 and GPR43 are a pair of mammalian G protein-coupled receptors (GPCRs)
expressed in human adipocytes, colon epithelial cells, and peripheral blood mononuclear …
expressed in human adipocytes, colon epithelial cells, and peripheral blood mononuclear …
Comparative transcriptomics in human and mouse
Cross-species comparisons of genomes, transcriptomes and gene regulation are now
feasible at unprecedented resolution and throughput, enabling the comparison of human …
feasible at unprecedented resolution and throughput, enabling the comparison of human …