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NULABEGLOGÉN AUTOGEDTEMCEL
NULABÉGLOGENE AUTOGEDTEMCEL
AUTOLOGOUS CD34+ HEMATOPOIETIC STEM AND PROGENITOR CELLS (HSPCS) OBTAINED BY APHERESIS FROM SICKLE CELL PATIENTS, GENETICALLY MODIFIED EX VIVO BY CRISPR/CAS9 (CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEATS/CRISPR-ASSOCIATED PROTEIN 9)-MEDIATED GENE EDITING CONSISTING OF A SINGLE GUIDE RNA (SGRNA) TARGETING THE FIRST EXON OF THE HUMAN BETA-GLOBIN (HBB) GENE, AND USING A HOMOLOGY-DIRECTED REPAIR MECHANISM TO CORRECT THE NUCLEIC ACID SEQUENCE ENCODING THE GLUTAMIC ACID TO VALINE MUTATION AT POSITION 6 IN THE HBB PROTEIN VIA AN ADENO-ASSOCIATED VIRUS SEROTYPE 6 (AAV6) VECTOR IN WHICH THE CORRECTIVE SEQUENCE IS LOCATED BETWEEN TWO HBB HOMOLOGY ARMS. FOLLOWING GENETIC MODIFICATION, EACH CELL CAN HAVE ONE OF SIX COMBINATIONS OF HBB ALLELE...; CORRECTED RECOMMENDED INN: LIST 89