Clinical gene therapy has faced numerous challenges and obstacles in the last few years.
However, it has made significant advances in treatment and is now in clinical trials and on …

Hematologic malignancies are driven by combinations of genetic lesions that have been
difficult to model in human cells. We used CRISPR/Cas9 genome engineering of primary …

Blood disorders are a group of diseases including hematological neoplasms, clotting
disorders and orphan immune deficiency diseases that affects human health. Current …

The discovery and development of the CRISPR/Cas system is a milestone in precise
medicine. CRISPR/Cas nucleases, base-editing (BE) and prime-editing (PE) are three …

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

The rapid advancement of genome-editing techniques holds much promise for the field of
human gene therapy. From bacteria to model organisms and human cells, genome editing …

Targeted genome editing with designer nucleases, such as zinc finger nucleases, TALE
nucleases, and CRISPR-Cas nucleases, has heralded a new era in gene therapy. Genetic …

The CRISPR/Cas9 system is a powerful genome editing tool that has made enormous
impacts on next-generation molecular diagnostics and therapeutics, especially for genetic …

The possibility of editing complex genomes in a targeted fashion has revolutionized basic
research as well as biomedical and biotechnological applications in the last 5 years. The …

Bit by bit, over the last few decades, functional genomic tools have been piecing together the
molecular puzzle driving tumorigenesis in human patients. Nevertheless, our understanding …