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Lixmabegagene relduparvovec, formerly known as LYS-GM101, is a recombinant adeno-associated virus vector serotype rh.10 (AAVrh.10) carrying the human β-galactosidase gene (GBL1). It is under development for the treatment of GM1 gangliosidosis.
Lixmabegagene relduparvovec, formerly known as LYS-GM101, is a recombinant adeno-associated virus vector serotype rh.10 (AAVrh.10) carrying the human β-galactosidase gene (GBL1). It is under development for the treatment of GM1 gangliosidosis.