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Status |
Public on Nov 30, 2021 |
Title |
AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice (ribosome profiling) |
Organisms |
Homo sapiens; Mus musculus |
Experiment type |
Other
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Summary |
In this study, we utilized a mouse model of mucopolysaccharidosis type I (MPS-I) carrying a homozygous nonsense mutation in the Idua gene (Idua-W401X, TGG→TAG) to develop rAAV-delivered sup-tRNA therapeutics that can overcome a pathogenic UAG premature termination codon (PTC).
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Overall design |
Liver ribosome profilings in sup-tRNA treated and untreated homozygous Idua-W401X mice and patient fibroblasts.
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Contributor(s) |
Wang D, Wang J, Zhang Y |
Citation(s) |
35322228 |
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Submission date |
Jul 01, 2021 |
Last update date |
Apr 22, 2022 |
Contact name |
Yue Zhang |
E-mail(s) |
6leensky@gmail.com
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Organization name |
Umass medical school
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Street address |
GeneTherapyCenter, 368 Plantation st
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City |
Worcester |
State/province |
Massachusetts |
ZIP/Postal code |
01605 |
Country |
USA |
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Platforms (2) |
GPL18573 |
Illumina NextSeq 500 (Homo sapiens) |
GPL19057 |
Illumina NextSeq 500 (Mus musculus) |
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Samples (16)
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This SubSeries is part of SuperSeries: |
GSE179275 |
AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice |
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Relations |
BioProject |
PRJNA743042 |
SRA |
SRP326500 |