METHODS AND COMPOSITIONS FOR TREATING ALPHA-1 ANTITRYPSIN DEFICIENCY

Fuente: WIPO "tomato"
Compositions and methods for editing deleterious mutations associated with Alpha-1 Antitrypsin Deficiency (A1AD). In particular embodiments, the invention provides methods for treating A1AD using a modified adenosine base editor with improved on-target editing and decreased off-target editing to correct mutations associated with A1AD.