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 Wed Apr 02 20:44:26 GMT 2025
Edited
by admin
on Wed Apr 02 20:44:26 GMT 2025
Nucleic Acid Type VECTOR
Sequence Origin VIRAL, HUMAN
Sequence Type COMPLETE
Record UNII
HX2RYV9JXP
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
Dalnacogene ponparvovec transgene insert
Common Name English
Dalnacogene ponparvovec transgene insert encoding a codon-optimised (CpG reduced) gain of function variant (Padua variant; R338L) of human coagulation factor IX
BAN  
Preferred Name English
Code System Code Type Description
FDA UNII
HX2RYV9JXP
Created by admin on Wed Apr 02 20:44:26 GMT 2025 , Edited by admin on Wed Apr 02 20:44:26 GMT 2025
PRIMARY
Sugar Site Range Site Count
Deoxyribose (DNA) 1_1-1_4403 4403 / 4403
Linkage Site Range Site Count
Phosphate 1_2-1_4403 4402 / 4402
Name Property Type Amount Referenced Substance Defining Parameters References
Enhancer NUCLEIC ACID FEATURE
Intron 1 of the human Ig heavy chain VH4 region germline (humhv4c2) gene NUCLEIC ACID FEATURE
Coding sequence of the human blood-coagulation factor IX NUCLEIC ACID FEATURE
ITRm NUCLEIC ACID FEATURE
Intron1 NUCLEIC ACID FEATURE
Promoter region of the Homo sapiens gene for alpha-1-antitrypsin NUCLEIC ACID FEATURE
5'UTR -reverse NUCLEIC ACID FEATURE
ITR NUCLEIC ACID FEATURE
5' UTR NUCLEIC ACID FEATURE
mini-polyA NUCLEIC ACID FEATURE
Coding sequence of the human blood-coagulation factor IX (FIX) gene (reverse) NUCLEIC ACID FEATURE
LXP2.1 promoter-reverse NUCLEIC ACID FEATURE
Intron2-reverse NUCLEIC ACID FEATURE
LXP2.1 promoter NUCLEIC ACID FEATURE
mini-polyA-reverse NUCLEIC ACID FEATURE