CRISPR screens are a powerful source of biological discovery, enabling the unbiased
interrogation of gene function in a wide range of applications and species. In pooled …

Over the past decade, CRISPR has become as much a verb as it is an acronym,
transforming biomedical research and providing entirely new approaches for dissecting all …

Cell-based therapeutics are an emerging modality with the potential to treat many currently
intractable diseases through uniquely powerful modes of action. Despite notable recent …

Genome-wide association studies (GWAS) test hundreds of thousands of genetic variants
across many genomes to find those statistically associated with a specific trait or disease …

Cytosine base editors (CBEs) are larger and can suffer from higher off-target activity or lower
on-target editing efficiency than current adenine base editors (ABEs). To develop a CBE that …

Thalassaemia is a diverse group of genetic disorders with a worldwide distribution affecting
globin chain synthesis. The pathogenesis of thalassaemia lies in the unbalanced globin …

Inducing fetal hemoglobin (HbF) in red blood cells can alleviate β-thalassemia and sickle
cell disease. We compared five strategies in CD34+ hematopoietic stem and progenitor …

Chronic antigen stimulation during viral infections and cancer can lead to T cell exhaustion,
which is characterized by reduced effector function and proliferation, and the expression of …

Gene editing to disrupt the GATA1-binding site at the+ 58 BCL11A erythroid enhancer could
induce γ-globin expression, which is a promising therapeutic strategy to alleviate β …

Genome editing has therapeutic potential for treating genetic diseases and cancer.
However, the currently most practicable approaches rely on the generation of DNA double …