
P104: BASE EDITING REPAIRS THE HBE MUTATION RESTORING THE PRODUCTION OF NORMAL GLOBIN CHAINS IN SEVERE HBE/β‐THALASSEMIA PATIENT HEMATOPOIETIC STEM AND ERYTHROID CELLS
Author(s) -
Badat M,
Hua P,
Mettananda S,
Fisher C,
Roy N,
Rice S,
Roy A,
Higgs D,
Davies J
Publication year - 2022
Publication title -
hemasphere
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.677
H-Index - 11
ISSN - 2572-9241
DOI - 10.1097/01.hs9.0000821508.47147.52
Subject(s) - frameshift mutation , thalassemia , genetics , biology , globin , mutation , stop codon , allele , gene , microbiology and biotechnology