RNA-seq dataset of brain tissue from MPSIIIA (Sgsh D31N) mouse model following antisense oligonucleotide treatment
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| Format: | Recurso digital |
| Sprache: | Englisch |
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2026
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| _version_ | 1866901260581470208 |
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| author | Mor, Nofar Vaknin, Noam |
| author_facet | Mor, Nofar Vaknin, Noam |
| contents | <p>This dataset contains RNA sequencing (RNA-seq) data generated from brain tissue of a murine model of mucopolysaccharidosis type IIIA (MPSIIIA).</p> <p>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.</p> <p>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.</p> <p>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.</p> <p>The dataset is associated with the study:<br>“Antisense oligonucleotides mediated substrate reduction therapy ameliorates heparan sulfate accumulation in MPSIIIA models.</p> <p>All animal procedures were approved by the Sheba Medical Center IACUC.</p> |
| format | Recurso digital |
| id | zenodo_https___doi_org_10_5281_zenodo_19351918 |
| institution | Zenodo |
| language | eng |
| publishDate | 2026 |
| publisher | Zenodo |
| record_format | zenodo |
| spellingShingle | RNA-seq dataset of brain tissue from MPSIIIA (Sgsh D31N) mouse model following antisense oligonucleotide treatment Mor, Nofar Vaknin, Noam RNA-Seq Oligonucleotides, Antisense Lysosomal Storage Diseases Sgsh Mucopolysaccharidosis Type IIIA MPS3A Sanfilippo A syndrome Heparan Sulfate Proteoglycans Heparan Sulfate Proteoglycans/genetics Heparan Sulfate Proteoglycans/metabolism Heparan Sulfate Proteoglycans/biosynthesis <p>This dataset contains RNA sequencing (RNA-seq) data generated from brain tissue of a murine model of mucopolysaccharidosis type IIIA (MPSIIIA).</p> <p>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.</p> <p>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.</p> <p>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.</p> <p>The dataset is associated with the study:<br>“Antisense oligonucleotides mediated substrate reduction therapy ameliorates heparan sulfate accumulation in MPSIIIA models.</p> <p>All animal procedures were approved by the Sheba Medical Center IACUC.</p> |
| title | RNA-seq dataset of brain tissue from MPSIIIA (Sgsh D31N) mouse model following antisense oligonucleotide treatment |
| topic | RNA-Seq Oligonucleotides, Antisense Lysosomal Storage Diseases Sgsh Mucopolysaccharidosis Type IIIA MPS3A Sanfilippo A syndrome Heparan Sulfate Proteoglycans Heparan Sulfate Proteoglycans/genetics Heparan Sulfate Proteoglycans/metabolism Heparan Sulfate Proteoglycans/biosynthesis |
| url | https://doi.org/10.5281/zenodo.19351918 |