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LONIMECGÉN RENPARVOVEC
RECOMBINANT, SELF-COMPLEMENTARY (DIMERIC), NONREPLICATING ADENO-ASSOCIATED VIRUS SEROTYPE 9 (SCAAV9) VECTOR ENCODING A MINIATURISED FORM OF HUMAN METHYL-CPG BINDING PROTEIN 2 (MECP2) CONTAINING THE METHYL-CPG-BINDING DOMAIN (MBD) AND THE NCOR/SMRT (SILENCING MEDIATOR OF RETINOIC ACID AND THYROID HORMONE RECEPTOR) INTERACTION DOMAIN (NID), UNDER THE CONTROL OF A FRAGMENT OF THE MOUSE MECP2 PROMOTER. THE 3' UNTRANSLATED REGION IS DESIGNED TO DECREASE PROTEIN EXPRESSION THROUGH RNA INTERFERENCE BY CONTAINING A MICRORNA (MIR) RESPONSIVE AUTO-REGULATORY ELEMENT (MIRARE) INSERTED INTO THE REPLICATION DEPENDENT HISTONE 1 (RDH1) POLYADENYLATION REGION. THE CONSTRUCT IS FLANKED BY ADENO-ASSOCIATED VIRUS 2 (AAV2) INVERTED TERMINAL REPEATS (ITRS)