RNA-seq dataset of brain tissue from MPSIIIA (Sgsh D31N) mouse model following antisense oligonucleotide treatment

This dataset contains RNA sequencing (RNA-seq) data generated from brain tissue of a murine model of mucopolysaccharidosis type IIIA (MPSIIIA).

The model used was B6.Cg-Sgsh^MPSIIIA/PstJ mice (Jackson Laboratories), which harbor a missense mutation in the murine Sgsh gene (c.91G>A, p.D31N). Homozygous mice exhibit approximately 3–4% of normal SGSH enzymatic activity, resulting in extensive lysosomal accumulation of heparan sulfate (HS) and progressive neurodegeneration.

RNA was extracted from brain tissue of wild-type, disease-model (Sgsh homozygous), and antisense oligonucleotide (ASO)-treated mice. RNA-seq libraries were prepared using the MARS-seq protocol and sequenced on an Illumina NovaSeq platform, generating single-end reads (~26 million reads per sample). Raw sequencing data are provided as FASTQ files, along with accompanying sample metadata.

This dataset enables analysis of differential gene expression and transcriptional signatures associated with MPSIIIA pathology, as well as transcriptional changes following ASO-mediated reduction of EXT1 expression.

The dataset is associated with the study:“Antisense oligonucleotides mediated substrate reduction therapy ameliorates heparan sulfate accumulation in MPSIIIA models.

All animal procedures were approved by the Sheba Medical Center IACUC.