2175009-08-0
Chemical Structure
Fazirsiran
Synonym(s): ARO-AAT
- CAS No.: 2175009-08-0
- Formula:N/A
- Molecular Weight:15587.60
SMILES: [Fazirsiran]
Biological Activity: Fazirsiran (ARO-AAT) is a second-generation RNAi agent. Fazirsiran consistes of a cholesterol-conjugated RNAi trigger (chol-RNAi) to selectively reduce Alpha1-antitrypsin (AAT) synthesis and a melittin-derived peptide conjugated to N-acetylgalactosamine (NAG) formulated as the excipient EX1 to promote endosomal escape of the chol-RNAi in hepatocytes[1]. Fazirsiran can be used in the study of Alpha-1 Antitrypsin Deficiency (AATD) liver disease. Alpha-1 antitrypsin deficiency (AATD) is caused by mutations in the alpha-1 antitrypsin (SERPINA1) gene.
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Fazirsiran | Fazirsiran (ARO-AAT) is a second-generation RNAi agent. Fazirsiran consistes of a cholesterol-conjugated RNAi trigger (chol-RNAi) to selectively reduce Alpha1-antitrypsin (AAT) synthesis and a melittin-derived peptide conjugated to N-acetylgalactosamine (NAG) formulated as the excipient EX1 to promote endosomal escape of the chol-RNAi in hepatocytes. Fazirsiran can be used in the study of Alpha-1 Antitrypsin Deficiency (AATD) liver disease. Alpha-1 antitrypsin deficiency (AATD) is caused by mutations in the alpha-1 antitrypsin (SERPINA1) gene. | |||||||||||||||||||||
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Keywords
