Mechanism and applications of CRISPR/Cas-9-mediated genome editing
M Asmamaw, B Zawdie - Biologics: targets and therapy, 2021 - Taylor & Francis
Clustered regularly interspaced short palindromic repeat (CRISPR) and their associated
protein (Cas-9) is the most effective, efficient, and accurate method of genome editing tool in …
protein (Cas-9) is the most effective, efficient, and accurate method of genome editing tool in …
Senescence mechanisms and targets in the heart
Cellular senescence is a state of irreversible cell cycle arrest associated with ageing.
Senescence of different cardiac cell types can direct the pathophysiology of cardiovascular …
Senescence of different cardiac cell types can direct the pathophysiology of cardiovascular …
Single-cell and spatial transcriptomics identify a macrophage population associated with skeletal muscle fibrosis
G Coulis, D Jaime, C Guerrero-Juarez… - Science …, 2023 - science.org
Macrophages are essential for skeletal muscle homeostasis, but how their dysregulation
contributes to the development of fibrosis in muscle disease remains unclear. Here, we used …
contributes to the development of fibrosis in muscle disease remains unclear. Here, we used …
Targeting RAGE prevents muscle wasting and prolongs survival in cancer cachexia
S Chiappalupi, G Sorci, A Vukasinovic… - Journal of cachexia …, 2020 - Wiley Online Library
Background Cachexia, a multifactorial syndrome affecting more than 50% of patients with
advanced cancer and responsible for~ 20% of cancer‐associated deaths, is still a poorly …
advanced cancer and responsible for~ 20% of cancer‐associated deaths, is still a poorly …
Genetic testing to inform epilepsy treatment management from an international study of clinical practice
D McKnight, A Morales, KE Hatchell, SL Bristow… - JAMA …, 2022 - jamanetwork.com
Importance It is currently unknown how often and in which ways a genetic diagnosis given to
a patient with epilepsy is associated with clinical management and outcomes. Objective To …
a patient with epilepsy is associated with clinical management and outcomes. Objective To …
[HTML][HTML] Complexity of skeletal muscle degeneration: Multi-systems pathophysiology and organ crosstalk in dystrophinopathy
K Ohlendieck, D Swandulla - Pflügers Archiv-European Journal of …, 2021 - Springer
Duchenne muscular dystrophy is a highly progressive muscle wasting disorder due to
primary abnormalities in one of the largest genes in the human genome, the DMD gene …
primary abnormalities in one of the largest genes in the human genome, the DMD gene …
Discovery of a highly potent and selective degrader targeting hematopoietic prostaglandin D synthase via in silico design
H Yokoo, N Shibata, A Endo, T Ito… - Journal of Medicinal …, 2021 - ACS Publications
Targeted protein degradation by proteolysis-targeting chimera (PROTAC) is one of the
exciting modalities for drug discovery and biological discovery. It is important to select an …
exciting modalities for drug discovery and biological discovery. It is important to select an …
Role of CRISPR/Cas9 in the treatment of Duchenne muscular dystrophy and its delivery strategies
Duchenne muscular dystrophy (DMD) is a neuromuscular disorder brought on by mutations
in the DMD gene, which prevent muscle cells from expressing the dystrophin protein …
in the DMD gene, which prevent muscle cells from expressing the dystrophin protein …
Efficient Downregulation of Alk4 in Skeletal Muscle After Systemic Treatment with Conjugated siRNAs in a Mouse Model for Duchenne Muscular Dystrophy
S Engelbeen, S Pasteuning-Vuhman… - nucleic acid …, 2023 - liebertpub.com
Downregulation of genes involved in the secondary pathology of Duchenne muscular
dystrophy, for example, inflammation, fibrosis, and adiposis, is an interesting approach to …
dystrophy, for example, inflammation, fibrosis, and adiposis, is an interesting approach to …
[HTML][HTML] Innate and adaptive AAV-mediated immune responses in a mouse model of Duchenne muscular dystrophy
MR Emami, A Espinoza, CS Young, F Ma… - … Therapy-Methods & …, 2023 - cell.com
High systemic doses of adeno-associated viruses (AAVs) have been associated with
immune-related serious adverse events (SAEs). Although AAV was well tolerated in …
immune-related serious adverse events (SAEs). Although AAV was well tolerated in …