CRISPR-Cas9 Base Editing in Sickle Cell Disease
Mechanistic animation of adenine base editing at the HBB locus, depicting guide RNA binding, adenosine deaminase activity, and haemoglobin switching in erythroid progenitors.
Mechanistic animation of adenine base editing at the HBB locus, depicting guide RNA binding, adenosine deaminase activity, and haemoglobin switching in erythroid progenitors.
Intravenous administration, retrograde motor neuron tropism, nuclear import, and episomal SMN1 transgene expression in lumbar anterior horn cells following onasemnogene abeparvovec delivery.