Beam Therapeutics Inc.
METHODS OF EDITING SINGLE NUCLEOTIDE POLYMORPHISM USING PROGRAMMABLE BASE EDITOR SYSTEMS

Abstract:

The invention features compositions and methods for altering mutations associated with Rett Syndrome (RTT). Provided herein are compositions and methods of using base editors comprising a polynucleotide programmable nucleotide binding domain and a nucleobase editing domain in conjunction with a guide polynucleotide. Also provided herein are base editor systems for editing nucleobases of target nucleotide sequences.