EXAGAMGLOGENE AUTOTEMCEL

UNII:	S53L777GM8
Formula:
Preferred Substance Name:	EXAGAMGLOGENE AUTOTEMCEL

AUTOLOGOUS CD34+ CELLS ISOLATED FROM MOBILISED PERIPHERAL BLOOD BY POSITIVE SELECTION, MODIFIED BY CRISPR/CAS9 (CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEATS/CRISPR-ASSOCIATED PROTEIN 9) MEDIATED GENE EDITING CONSISTING OF A GUIDE RNA (GRNA) INTRODUCED TRANSIENTLY AS RIBONUCLEOPROTEIN (RNP) COMPLEX, TARGETING THE ERYTHROID LINEAGE-SPECIFIC ENHANCER REGION OF BCL11A (B-CELL LYMPHOMA/LEUKEMIA 11A). THE SITE-SPECIFIC CLEAVAGE BY CAS9 FORMS A DOUBLE STRAND BREAK (DSB), WHICH IS SUBSEQUENTLY REPAIRED BY NONHOMOLOGOUS END-JOINING (NHEJ), LEADING TO THE TRANSCRIPTIONAL REPRESSION OF BCL11A, A REPRESSOR OF .GAMMA.-GLOBIN GENE TRANSCRIPTION
CTX001
CTX-001
EXAGAMGLOGENE AUTOTEMCEL [INN]
EXAGAMGLOGENE AUTOTEMCEL [USAN]
EXAGAMGLOGENE AUTOTEMCEL [WHO-DD]

Note

UNIIs are generated based on scientific identity characteristics using ISO 11238 data elements. UNII availability does not imply any regulatory review or approval. Synonyms and mappings are based on the best public information available at the time of publication. Please report any problems/errors associated with this data to FDA-SRS@fda.hhs.gov.

EXAGAMGLOGENE AUTOTEMCEL

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