The next generation of CRISPR–Cas technologies and applications
A Pickar-Oliver, CA Gersbach - Nature reviews Molecular cell biology, 2019 - nature.com
The prokaryote-derived CRISPR–Cas genome editing systems have transformed our ability
to manipulate, detect, image and annotate specific DNA and RNA sequences in living cells …
to manipulate, detect, image and annotate specific DNA and RNA sequences in living cells …
Therapeutic developments for Duchenne muscular dystrophy
IEC Verhaart, A Aartsma-Rus - Nature Reviews Neurology, 2019 - nature.com
Duchenne muscular dystrophy (DMD) is caused by the lack of functional dystrophin protein.
Improvements in patient care and disease management have slowed down disease …
Improvements in patient care and disease management have slowed down disease …
CRISPR-Cas12a-based nucleic acid amplification-free DNA biosensor via Au nanoparticle-assisted metal-enhanced fluorescence and colorimetric analysis
Cell-free DNA (cfDNA) has attracted significant attention due to its high potential to diagnose
diseases, such as cancer. Still, its detection by amplification method has limitations because …
diseases, such as cancer. Still, its detection by amplification method has limitations because …
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 …
Therapeutic approaches for Duchenne muscular dystrophy
TC Roberts, MJA Wood, KE Davies - Nature Reviews Drug Discovery, 2023 - nature.com
Duchenne muscular dystrophy (DMD) is a monogenic muscle-wasting disorder and a
priority candidate for molecular and cellular therapeutics. Although rare, it is the most …
priority candidate for molecular and cellular therapeutics. Although rare, it is the most …
Making muscle: skeletal myogenesis in vivo and in vitro
J Chal, O Pourquié - Development, 2017 - journals.biologists.com
Skeletal muscle is the largest tissue in the body and loss of its function or its regenerative
properties results in debilitating musculoskeletal disorders. Understanding the mechanisms …
properties results in debilitating musculoskeletal disorders. Understanding the mechanisms …
Therapeutic approaches for cardiac regeneration and repair
H Hashimoto, EN Olson, R Bassel-Duby - Nature Reviews Cardiology, 2018 - nature.com
Ischaemic heart disease is a leading cause of death worldwide. Injury to the heart is
followed by loss of the damaged cardiomyocytes, which are replaced with fibrotic scar …
followed by loss of the damaged cardiomyocytes, which are replaced with fibrotic scar …
Induced pluripotent stem cells meet genome editing
D Hockemeyer, R Jaenisch - Cell stem cell, 2016 - cell.com
It is extremely rare for a single experiment to be so impactful and timely that it shapes and
forecasts the experiments of the next decade. Here, we review how two such experiments …
forecasts the experiments of the next decade. Here, we review how two such experiments …
CRISPR-Cas tools and their application in genetic engineering of human stem cells and organoids
CRISPR-Cas technology has revolutionized biological research and holds great therapeutic
potential. Here, we review CRISPR-Cas systems and their latest developments with an …
potential. Here, we review CRISPR-Cas systems and their latest developments with an …
Molecular and cellular basis of genetically inherited skeletal muscle disorders
JJ Dowling, CC Weihl, MJ Spencer - Nature Reviews Molecular Cell …, 2021 - nature.com
Neuromuscular disorders comprise a diverse group of human inborn diseases that arise
from defects in the structure and/or function of the muscle tissue—encompassing the muscle …
from defects in the structure and/or function of the muscle tissue—encompassing the muscle …