Translation of the CRISPR–Cas9 system to human therapeutics holds high promise. However,
specificity remains a concern especially when modifying stem cell populations. We show …

Permanent modification of the human genome in vivo is impractical owing to the low frequency
of homologous recombination in human cells, a fact that hampers biomedical research …

The β-haemoglobinopathies, such as sickle cell disease and β-thalassaemia, are caused by
mutations in the β-globin (HBB) gene and affect millions of people worldwide. Ex vivo gene …

The CRISPR–Cas9 system is a powerful tool for genome editing, which allows the precise
modification of specific DNA sequences. Many efforts are underway to use the CRISPR–Cas9 …

The CRISPR/Cas9 system has recently been shown to facilitate high levels of precise
genome editing using adeno-associated viral (AAV) vectors to serve as donor template DNA …

CRISPR-Cas-mediated genome editing relies on guide RNAs that direct site-specific DNA
cleavage facilitated by the Cas endonuclease. Here we report that chemical alterations to …

Gene correction in human long-term hematopoietic stem cells (LT-HSCs) could be an effective
therapy for monogenic diseases of the blood and immune system. Here we describe an …

Safeguard mechanisms can ameliorate the potential risks associated with cell therapies but
currently rely on the introduction of transgenes. This limits their application owing to …

Correction of gene defects in human somatic cells by targeting as has been used in murine
embryonic stem cells (1, 2) has been precluded by the low spontaneous rate of gene …

Oncogenes are activated through well-known chromosomal alterations such as gene fusion,
translocation, and focal amplification. In light of recent evidence that the control of key …