Genome editing, which involves the precise manipulation of cellular DNA sequences to alter
cell fates and organism traits, has the potential to both improve our understanding of human …

Cell type-and differentiation-specific gene expression is precisely controlled by genomic non-
coding regulatory elements (NCREs), which include promoters, enhancers, silencers and …

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 …

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

Abstract We recently introduced Cleavage Under Targets & Tagmentation (CUT&Tag), an
epigenomic profiling strategy in which antibodies are bound to chromatin proteins in situ in …

Systematic evaluation of the impact of genetic variants is critical for the study and treatment
of human physiology and disease. While specific mutations can be introduced by genome …

Base editors enable targeted single-nucleotide conversions in genomic DNA. Here we show
that expression levels are a bottleneck in base-editing efficiency. We optimize cytidine (BE4) …

Re-expression of the paralogous γ-globin genes (HBG1/2) could be a universal strategy to
ameliorate the severe β-globin disorders sickle cell disease (SCD) and β-thalassemia by …

Previously, we described a novel alternative to chromatin immunoprecipitation, CUT&RUN,
in which unfixed permeabilized cells are incubated with antibody, followed by binding of a …

Long non-coding RNAs (lncRNAs) outnumber protein-coding transcripts, but their functions
remain largely unknown. In this Review, we discuss the emerging roles of lncRNAs in the …