Functional elucidation of causal genetic variants and elements requires precise genome
editing technologies. The type II prokaryotic CRISPR (clustered regularly interspaced short …

Frame-disrupting mutations in the DMD gene, encoding dystrophin, compromise myofiber
integrity and drive muscle deterioration in Duchenne muscular dystrophy (DMD). Removing …

The RNA-guided endonuclease Cas9 has emerged as a versatile genome-editing platform.
However, the size of the commonly used Cas9 from Streptococcus pyogenes (SpCas9) …

Transcription activator-like effectors (TALEs) are a class of naturally occurring DNA-binding
proteins found in the plant pathogen Xanthomonas sp. The DNA-binding domain of each …

Adeno-associated viral (AAV) vectors have emerged as the leading gene delivery platform
for gene therapy and vaccination. Three AAV-based gene therapy drugs, Glybera, …

Gene editing is a powerful tool for genome and cell engineering. Exemplified by CRISPR–Cas,
gene editing could cause DNA damage and trigger DNA repair processes that are often …

Axon regeneration holds great promise for neural repair of CNS axonopathies, including
glaucoma. Pten deletion in retinal ganglion cells (RGCs) promotes potent optic nerve …

Objective Applications of artificial intelligence (AI) have been reported in several cardiovascular
diseases but its interest in patients with peripheral artery disease (PAD) has been so far …

Forest malaria is a complex but common phenomenon occurring in southeast Asia. We
studied its epidemiology through a prospective community-based study in central Vietnam. A …

We propose the Model-based Analysis of Genome-wide CRISPR/Cas9 Knockout (MAGeCK)
method for prioritizing single-guide RNAs, genes and pathways in genome-scale CRISPR/…