Based on engineered or bacterial nucleases, the development of genome editing
technologies has opened up the possibility of directly targeting and modifying genomic …

Programmable nucleases allow defined alterations in the genome with ease-of-use,
efficiency, and specificity. Their availability has led to accurate and widespread genome …

CRISPR/Cas9 has been widely used in generating site-specific genetically modified animal
models. Myostatin (MSTN) is a negative regulator of muscle mass, related to muscle growth …

Duchenne muscular dystrophy (DMD) is an X-linked muscle-wasting disorder caused by
mutations in the dystrophin gene, with an incidence of 1 in 3500 in new male births. Mdx …

Abstract The CRISPR RNA-guided Cas9 nuclease gene-targeting system has been
extensively used to edit the genome of several organisms. However, most mutations …

Dear Editor, Tay–Sachs disease (TSD) is a progressive neurodegenerative disorder due to
an autosomal recessively inherited deficiency of β-hexosaminidase A (HexA) 1. The four …

Pigs share many physiological, biochemical, and anatomical similarities with humans and
have emerged as valuable large animal models for biomedical research. Considering the …

Cataracts are the leading cause of vision loss in the world, although surgical treatment can
restore vision in cataract patients. Until now, there have been no adequate animal models …

The prokaryotic clustered regularly interspaced short palindromic repeat (CRISPR)-
associated system (Cas) is a simple, robust and efficient technique for gene targeting in …

Limb girdle muscular dystrophy type 2L (LGMD2L) and Miyoshi myopathy type 3 (MMD3)
are autosomal recessive muscular dystrophy caused by mutations in the gene encoding …