Tyrosine-type site-specific recombinases (T-SSRs) have opened new avenues for the
predictable modification of genomes as they enable precise genome editing in heterologous …

Inherited blood disorders comprise a large spectrum of diseases due to germline mutations
in genes with key function in the hematopoietic system; they include immunodeficiencies …

In contrast to CRISPR/Cas9 nucleases, CRISPR base editors (BE) and prime editors (PE)
enable predefined nucleotide exchanges in genomic sequences without generating DNA …

The pathological consequences of structural variants disrupting 3D genome organization
can be difficult to elucidate in vivo due to differences in gene dosage sensitivity between …

X-linked chronic granulomatous disease (X-CGD) is an immune deficiency resulting from
defective production of microbicidal reactive oxygen species (ROS) by phagocytes …

Induced pluripotent stem cells (iPSCs) are derived from somatic cells through a
reprogramming process, which converts them to a pluripotent state, akin to that of embryonic …

Chronic granulomatous disease (CGD) is the most common symptomatic phagocytic defect.
It is caused by mutations in genes encoding protein subunits of the nicotinamide adenine …

The breakthrough in human induced pluripotent stem cells (hiPSCs) has revolutionized the
field of biomedical and pharmaceutical research and opened up vast opportunities for drug …

In the last two decades, the exponential progress in the field of genetics could reveal the
genetic impact on the onset and progression of several diseases affecting the immune …

Chronic granulomatous disease (CGD) is a rare inherited immunodeficiency due to
dysfunction of the phagocytic nicotinamide adenine dinucleotide phosphate (NADPH) …