Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated
protein 9 (Cas9) gene-editing technology is the ideal tool of the future for treating diseases …

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 …

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 …

Sickle cell disease (SCD) is caused by a mutation in the β-globin gene HBB. We used a
custom adenine base editor (ABE8e-NRCH), to convert the SCD allele (HBB S) into …

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 …

Applications of adenine base editors (ABEs) have been constrained by the limited
compatibility of the deoxyadenosine deaminase component with Cas homologs other than …

Transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) are severe
monogenic diseases with severe and potentially life-threatening manifestations. BCL11A is …

CRISPR-mediated genome editing of primary human lymphocytes is typically carried out via
electroporation, which can be cytotoxic, cumbersome and costly. Here we show that the …

CRISPR/Cas9 genome editing has gained rapidly increasing attentions in recent years,
however, the translation of this biotechnology into therapy has been hindered by efficient …