technology
Base editing
Precision genome editing via cytidine (CBE) or adenine (ABE) base editors that convert single nucleotides without DNA double-strand breaks.
Where it's used
Base editing
Base editors (CBE/ABE) make single-nucleotide changes C·G→T·A and A·T→G·C without double-strand breaks — treating monogenic disease (sickle cell, beta-thalassemia) at up to 90% efficiency with no indels.
Genome editing
Programmable genome editors — CRISPR-Cas9, base editors and prime editors — delivered ex vivo or in vivo via lipid nanoparticles to treat genetic disease, from the first approved CRISPR therapy Casgevy to in-vivo redosable CRISPR and single-dose cardiovascular base editing.