The development of customizable sequence-specific nucleases such as TALENs, ZFNs and
the powerful CRISPR/Cas9 system has revolutionized the field of genome editing. The …

Cancer is caused by a series of alterations in genome and epigenome mostly resulting in
activation of oncogenes or inactivation of cancer suppressor genes. Genetic engineering …

The major function of the lymphatic system is to drain interstitial fluid from tissue. Functional
drainage causes increased fluid flow that triggers lymphatic expansion, which is …

Abstract Introduction The Comprehensive in vitro Proarrhythmia Assay (C i PA) is a
nonclinical Safety Pharmacology paradigm for discovering electrophysiological mechanisms …

Fabry disease (FD) is a progressive, X-linked inherited disorder of glycosphingolipid
metabolism due to deficient or absent lysosomal α-galactosidase A (α-Gal A) activity which …

Crohn's disease (CD) is caused by a trigger, almost certainly enteric infection by one of a
multitude of organisms that allows faeces access to the tissues, at which stage the response …

The emergence of targeted and efficient genome editing technologies, such as repurposed
bacterial programmable nucleases (eg, CRISPR-Cas systems), has abetted the …

The generation of targeted mutants is a crucial step toward studying the biomedical effect of
genes of interest. The generation of such mutants in human induced pluripotent stem cells …

Functional genomics studies of the immune system require genetic manipulations that
involve both deletion of target genes and addition of elements to proteins of interest …

The cause of Crohn's disease (CD) has posed a conundrum for at least a century. A large
body of work coupled with recent technological advances in genome research have at last …