CRISPR/Cas9 genome editing for tissue‐specific in vivo targeting: nanomaterials and translational perspective

DK Sahel, LK Vora, A Saraswat, S Sharma… - Advanced …, 2023 - Wiley Online Library
Clustered randomly interspaced short palindromic repeats (CRISPRs) and its associated
endonuclease protein, ie, Cas9, have been discovered as an immune system in bacteria …

CRISPR-based therapeutic gene editing for Duchenne muscular dystrophy: advances, challenges and perspectives

G Chen, T Wei, H Yang, G Li, H Li - Cells, 2022 - mdpi.com
Duchenne muscular dystrophy (DMD) is a severe neuromuscular disease arising from loss-
of-function mutations in the dystrophin gene and characterized by progressive muscle …

[HTML][HTML] Correction of human nonsense mutation via adenine base editing for Duchenne muscular dystrophy treatment in mouse

M Jin, J Lin, H Li, Z Li, D Yang, Y Wang, Y Yu… - … Therapy-Nucleic Acids, 2024 - cell.com
Duchenne muscular dystrophy (DMD) is the most prevalent herediatry disease in men,
characterized by dystrophin deficiency, progressive muscle wasting, cardiac insufficiency …

CRISPR-Cas9 base editing of pathogenic CaMKIIδ improves cardiac function in a humanized mouse model

S Lebek, XM Caravia, LG Straub… - The Journal of …, 2024 - Am Soc Clin Investig
Cardiovascular diseases are the most common cause of worldwide morbidity and mortality,
highlighting the necessity for advanced therapeutic strategies. Ca2+/calmodulin-dependent …

CRISPR-editing therapy for Duchenne muscular dystrophy

F Chemello, EN Olson, R Bassel-Duby - Human gene therapy, 2023 - liebertpub.com
Duchenne muscular dystrophy (DMD) is a debilitating genetic disorder that results in
progressive muscle degeneration and premature death. DMD is caused by mutations in the …

CRISPR/Cas9 technology: applications in oocytes and early embryos

Y Zhang, T Yin, L Zhou - Journal of Translational Medicine, 2023 - Springer
CRISPR/Cas9, a highly versatile genome-editing tool, has garnered significant attention in
recent years. Despite the unique characteristics of oocytes and early embryos compared to …

Calcium handling maturation and adaptation to increased substrate stiffness in human iPSC-derived cardiomyocytes: The impact of full-length dystrophin deficiency

JM Pioner, L Santini, C Palandri, M Langione… - Frontiers in …, 2022 - frontiersin.org
Cardiomyocytes differentiated from human induced Pluripotent Stem Cells (hiPSC-CMs) are
a unique source for modelling inherited cardiomyopathies. In particular, the possibility of …

Prevention of early-onset cardiomyopathy in Dmd exon 52–54 deletion mice by CRISPR-Cas9-mediated exon skipping

M Rok, TWY Wong, E Maino, A Ahmed, G Yang… - … Therapy-Methods & …, 2023 - cell.com
Duchenne muscular dystrophy (DMD) is a disease with a life-threatening trajectory resulting
from mutations in the dystrophin gene, leading to degeneration of skeletal muscle and …

Progress in and Prospects of Genome Editing Tools for Human Disease Model Development and Therapeutic Applications

HTL Phan, K Kim, H Lee, JK Seong - Genes, 2023 - mdpi.com
Programmable nucleases, such as zinc finger nucleases (ZFNs), transcription activator-like
effector nucleases (TALENs), and clustered regularly interspaced short palindromic repeats …

Discovering weak signals of emerging topics with a triple-dimensional framework

M Ma, J Mao, G Li - Information Processing & Management, 2024 - Elsevier
In the rapidly evolving landscape of innovation, the early identification of emerging topics is
crucial across diverse research domains. This study views weak signals as the preliminary …