The promise and challenge of therapeutic genome editing
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 fates and organism traits, has the potential to both improve our understanding of human …
Gene therapy using haematopoietic stem and progenitor cells
Haematopoietic stem and progenitor cell (HSPC) gene therapy has emerged as an effective
treatment modality for monogenic disorders of the blood system such as primary …
treatment modality for monogenic disorders of the blood system such as primary …
Potent and uniform fetal hemoglobin induction via base editing
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 …
cell disease. We compared five strategies in CD34+ hematopoietic stem and progenitor …
[HTML][HTML] Massively parallel base editing to map variant effects in human hematopoiesis
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 …
of human physiology and disease. While specific mutations can be introduced by genome …
Highly efficient therapeutic gene editing of human hematopoietic stem cells
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 …
ameliorate the severe β-globin disorders sickle cell disease (SCD) and β-thalassemia by …
Dual base editor catalyzes both cytosine and adenine base conversions in human cells
Although base editors are useful tools for precise genome editing, current base editors can
only convert either adenines or cytosines. We developed a dual adenine and cytosine base …
only convert either adenines or cytosines. We developed a dual adenine and cytosine base …
Recent advances in the treatment of sickle cell disease
Sickle cell anemia (SCA) was first described in the Western literature more than 100 years
ago. Elucidation of its molecular basis prompted numerous biochemical and genetic studies …
ago. Elucidation of its molecular basis prompted numerous biochemical and genetic studies …
The vaso‐occlusive pain crisis in sickle cell disease: definition, pathophysiology, and management
Early diagnosis, treatment, and prevention of a vaso‐occlusive crisis (VOC) are critical to the
management of patients with sickle cell disease. It is essential to differentiate between VOC …
management of patients with sickle cell disease. It is essential to differentiate between VOC …
Genome editing of HBG1 and HBG2 to induce fetal hemoglobin
Induction of fetal hemoglobin (HbF) via clustered regularly interspaced short palindromic
repeats/Cas9–mediated disruption of DNA regulatory elements that repress γ-globin gene …
repeats/Cas9–mediated disruption of DNA regulatory elements that repress γ-globin gene …
HIC2 controls developmental hemoglobin switching by repressing BCL11A transcription
The fetal-to-adult switch in hemoglobin production is a model of developmental gene control
with relevance to the treatment of hemoglobinopathies. The expression of transcription factor …
with relevance to the treatment of hemoglobinopathies. The expression of transcription factor …