The incidence of esophageal adenocarcinoma (EAC) has increased in recent decades, and
its 5-year survival rate is less than 20%. As a well-established precursor, patients with …

Nuclease-based genome editing strategies hold great promise for the treatment of blood
disorders. However, a major drawback of these approaches is the generation of potentially …

CRISPR–Cas proteins are RNA-guided nucleases used to introduce double-stranded
breaks (DSBs) at targeted genomic loci. DSBs are repaired by endogenous cellular …

Induction of fetal hemoglobin (HbF) via clustered regularly interspaced short palindromic
repeats/Cas9–mediated disruption of DNA regulatory elements that repress γ-globin gene …

X-linked chronic granulomatous disease is an immunodeficiency characterized by defective
production of microbicidal reactive oxygen species (ROS) by phagocytes. Causative …

The advent of next-generation genome engineering tools like CRISPR-Cas9 has
transformed the field of gene therapy, rendering targeted treatment for several incurable …

The thalassemias are compelling targets for therapeutic genome editing in part because
monoallelic correction of a subset of hematopoietic stem cells (HSCs) would be sufficient for …

Targeted nucleases are powerful genomic tools to precisely change the target genome of
living cells, controlling functional genes with high exactness. The clustered regularly …

Methods for sensitive and high-throughput evaluation of CRISPR RNA-guided nucleases
(RGNs) off-targets (OTs) are essential for advancing RGN-based gene therapies. Here we …

The treatment of glioblastoma has been a big challenge for decades in the oncological field
mainly owing to its unique biological characteristics, such as high heterogeneity, diffusing …