Genome editing technologies, particularly those based on zinc-finger nucleases (ZFNs),
transcription activator-like effector nucleases (TALENs), and CRISPR (clustered regularly …

Chronic granulomatous disease (CGD) is a primary immunodeficiency of phagocyte function
due to defective NADPH oxidase (phox). Compared with the common types of CYBB/gp91 …

Genome editing has shown great promise for clinical translation but also revealed the risk of
genotoxicity caused by off-target effects of programmable nucleases. Here we describe …

Macrophages derived from human induced pluripotent stem cells (iPSCs) have the potential
to enable the development of cell-based therapies for numerous disease conditions. We …

Introduction: Chronic granulomatous disease (CGD) is a primary immunodeficiency
characterized by recurrent, life-threatening bacterial and fungal infections of the skin, the …

The molecular basis of interindividual clinical variability upon infection with Staphylococcus
aureus is unclear. We describe patients with haploinsufficiency for the linear deubiquitinase …

The replication-defective, non-pathogenic, nearly ubiquitous single-stranded adeno-
associated viruses (AAVs) have gained importance since their discovery about 50 years …

Gene-editing technology is an emerging therapeutic modality for manipulating the
eukaryotic genome by using target-sequence-specific engineered nucleases. Because of …

Chronic granulomatous disease (CGD) is an innate immunodeficiency with a genetic defect
of the nicotinamide adenosine dinucleotide phosphate, reduced, oxidase components. This …

The therapeutic use of adeno-associated viral vector (AAV)-mediated gene disruption using
CRISPR-Cas9 is limited by potential off-target modifications and the risk of uncontrolled …