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 …

The bacteria-derived CRISPR/Cas (an acronym for regularly interspaced short palindromic
repeats/CRISPR-associated protein) system is currently the most widely used, versatile, and …

Therapeutic advances for neurological disorders are challenging due to limited accessibility
of the human central nervous system and incomplete understanding of disease …

Genome editing tools have already revolutionized biomedical research and are also
expected to have an important impact in the clinic. However, their extensive use in research …

As one of the most severe forms of muscle dystrophy, Duchenne muscular dystrophy (DMD)
results in progressive muscle wasting, ultimately resulting in premature death due to …

CRISPR screen technology enables systematic and scalable interrogation of gene function
by using the CRISPR-Cas9 system to perturb gene expression. In the field of cancer …

Significant progress has been achieved in understanding Duchenne muscular dystrophy
(DMD) mechanisms and developing treatments to slow disease progression. This review …

Abstract 3D bioengineered skeletal muscle macrotissues are increasingly important for
studies of cell biology and development of therapeutics. Tissues derived from immortalized …

CRISPR-Cas9 gene editing has emerged as a transformative tool in pharmaceuticals,
revolutionizing the understanding and treatment of genetic diseases. This review explores …

Background: Gene therapy has proved to be a boon for neuromuscular diseases. A concept
introduced in the early 1960's, has been put into clinical practice in the past 5-10 years. The …