RECOMBINANT, NON-REPLICATING ADENO-ASSOCIATED VIRUS SEROTYPE 9 (RAAV9) VECTOR, ENCODING CODON-OPTIMISED HUMAN .BETA.-HEXOSAMINIDASE SUBUNITS .BETA. AND .ALPHA., JOINED BY A PORCINE TESCHOVIRUS 2A PEPTIDE LINKER, UNDER CONTROL OF A SYNTHETIC PROMOTER (CYT
Common Name
English
engabexagene cincesparvovec [INN]
Common Name
English
DNA (RECOMBINANT ADENO-ASSOCIATED VIRUS VECTOR TSHA-101 SYNTHETIC CAG PROMOTER PLUS HUMAN .BETA.-N-ACETYLHEXOSAMINIDASE SUBUNIT .BETA. PRECURSOR FUSION PROTEIN WITH PORCINE TESCHOVIRUS 2A PEPTIDE FUSION PROTEIN WITH HUMAN .BETA.-N-ACETYLHEXOSAMINIDASE SU
