U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

Approval Year

Substance Class Nucleic Acid
Created
by admin
on Sat Dec 16 18:27:37 GMT 2023
Edited
by admin
on Sat Dec 16 18:27:37 GMT 2023
Nucleic Acid Type VECTOR
Nucleic Acid Subtype TRANSGENE
Sequence Type COMPLETE
Record UNII
KND6MY3R59
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
NULABEGLOGENE AUTOGEDTEMCEL TRANSGENE INSERT
Common Name English
NULABEGLOGENE AUTOGEDTEMCEL TRANSGENE INSERT ENCODING HUMAN HBB
Common Name English
Code System Code Type Description
FDA UNII
KND6MY3R59
Created by admin on Sat Dec 16 18:27:37 GMT 2023 , Edited by admin on Sat Dec 16 18:27:37 GMT 2023
PRIMARY
Sugar Site Range Site Count
Deoxyribose (DNA) 1_1-1_2700 2700 / 2700
Linkage Site Range Site Count
Phosphate 1_2-1_2700 2699 / 2699
Name Property Type Amount Referenced Substance Defining Parameters References
3’ ITR NUCLEIC ACID FEATURE
GAG (Glutamate codon to correct the valine at this ) NUCLEIC ACID FEATURE
R-02(Mutated-guide RNA homologous target sequence) NUCLEIC ACID FEATURE
Homology arm upstream of the R-02 sequence NUCLEIC ACID FEATURE
Haemoglobin subunit beta NUCLEIC ACID FEATURE
5’-ITR NUCLEIC ACID FEATURE
Homology arm downstream of the R-02 sequence-HA-3' NUCLEIC ACID FEATURE
ATG site (Haemoglobin translation initiation site) NUCLEIC ACID FEATURE