Article
Prenatal delivery of a therapeutic antisense oligonucleotide achieves broad biodistribution in the brain and ameliorates Angelman syndrome phenotype in mice.
Molecular therapy : the journal of the American Society of Gene Therapy - 3 Apr 2024
Clarke Maria T, Remesal Laura, Lentz Lea, Tan Danielle J, Young David, Thapa Slesha, Namuduri Shalini R, Borges Beltran, Kirn Georgia, Valencia Jasmine, Lopez Manuel E, Lui Jan H, Shiow Lawrence R, Dindot Scott, Villeda Saul, Sanders Stephan J, MacKenzie Tippi C
Abstract excerpt
Angelman syndrome (AS), an early-onset neurodevelopmental disorder characterized by abnormal gait, intellectual disabilities, and seizures, occurs when the maternal allele of the UBE3A gene is disrupted, since the paternal allele is silenced in neurons by the UBE3A antisense (UBE3A-AS) transcript. Given the importance of early treatment, we hypothesized that prenatal delivery of an antisense oligonucleotide (ASO)...
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