A single gene mutation can cause a number of human diseases that affect the quality of life.
Until the development of clustered regularly interspaced short palindromic repeats …

Discovery of the CRISPR-Cas (clustered regularly interspaced short palindromic repeat,
CRISPR-associated) system a decade ago has opened new possibilities in the field of …

Duchenne muscular dystrophy (DMD) is a severe, progressive, muscle-wasting disease,
characterized by progressive deterioration of skeletal muscle that causes rapid loss of …

Neurological disorders are primarily diseases with sophisticated etiology that are always
refractory and recrudescent. The major obstruction to effective therapies for neurological …

The CRISPR gene editing tool holds great potential for curing genetic disorders. However,
the safe, efficient, and specific delivery of the CRISPR/Cas9 components into cells and …

Methods for sensitive and high-throughput evaluation of CRISPR RNA-guided nucleases
(RGNs) off-targets (OTs) are essential for advancing RGN-based gene therapies. Here we …

In recent years, we have realized that extracellular vesicles (EVs) play a critical role in
regulating the intercellular communication between tumor and immune cells in the tumor …

The transcription factor cyclic-AMP response element-binding protein 1 (CREB1) responds
to cAMP level and controls the expression of target genes, which regulates nutrition …

Genetic variations (including substitutions, insertions, and deletions) exert a profound
influence on DNA sequences. These variations are systematically classified as synonymous …

Duchenne muscular dystrophy (DMD) is caused by the dystrophin gene mutations and is
one of the most common and lethal human hereditary disorders. A novel therapeutic …