GEO series
Single Alkyl Phosphonate Modification of the siRNA Backbone in the Seed Region Enhances Specificity and Therapeutic Profile
GSE285764
Homo sapiens; Mus musculus
Expression profiling by high throughput sequencing
110 samples
Submitted 2025/01/15
Platform GPL24676Platform GPL24247
Summary
We evaluated the effect of alkyl phosphonate linkages in enhancing the specificity and therapeutic profile of siRNA when incorporated into the seed region. Modified siRNAs with a single alkyl phosphonate linkage demonstrated improved specificity, particularly when positioned at the internucleotide linkage between positions 6 and 7 from the 5'-end of the guide strand. These modifications maintained robust on-target activity both in vitro and in vivo while significantly reducing off-target effects, as shown by differential gene expression analysis. Additionally, we observed enhanced safety in mice, as evidenced by reduced ALT/AST elevation and the absence of histopathological changes.
Published in
Nikan M, Li Q, Tanowitz M et al.
· Nucleic acids research 2025
· PMID 40737088
· doi:10.1093/nar/gkaf692
This dataset
Download
Direct links to NCBI, no account and no request form: the whole study as GSE285764_RAW.tar, processed values as the series matrix, the supplementary file directory, and per-sample supplementary files for any of the 110 samples. Raw sequencing reads are also available from ENA.
Also filed as BioProject PRJNA1205781 and SRA study SRP555014. Searching any of these in the dataset finder brings you back here.
Study design
40 conditions, each sampled once — no replicated groups
Read from the first 40 of 110 sample titles: 40 distinct titles with little repetition. Check it against the sample list below before relying on it.
Samples in this study
0 selected
- GSM8708392 ALow_MD302_PBS_1 mg/kg_7 Days_liver_A-3_A3
- GSM8708393 ALow_MD302_PBS_1 mg/kg_7 Days_liver_A-2_A2
- GSM8708394 ALow_MD302_PBS_1 mg/kg_7 Days_liver_A-1_A1
- GSM8708395 ALow_MD302_PBS_1 mg/kg_7 Days_liver_A-4_A4
- GSM8708396 ALow_MD302_1708616_10 mg/kg_7 Days_liver_F-3_B19
- GSM8708397 ALow_MD302_1708616_10 mg/kg_7 Days_liver_F-2_B18
- GSM8708398 ALow_MD302_1708616_10 mg/kg_7 Days_liver_F-1_B17
- GSM8708399 ALow_MD302_1708616_10 mg/kg_7 Days_liver_F-4_B20
- GSM8708400 ALow_MD302_1708616_100 mg/kg_7 Days_liver_H-3_C3
- GSM8708401 ALow_MD302_1708616_100 mg/kg_7 Days_liver_H-1_C1
- GSM8708402 ALow_MD302_1708616_100 mg/kg_7 Days_liver_H-2_C2
- GSM8708403 ALow_MD302_1708616_100 mg/kg_7 Days_liver_H-4_C4
- GSM8708404 ALow_MD302_1708616_30 mg/kg_7 Days_liver_G-3_B23
- GSM8708405 ALow_MD302_1708616_30 mg/kg_7 Days_liver_G-2_B22
- GSM8708406 ALow_MD302_1708616_30 mg/kg_7 Days_liver_G-4_B24
- GSM8708407 ALow_MD302_1708616_30 mg/kg_7 Days_liver_G-1_B21
- GSM8708408 ALow_MD303_PBS_1 mg/kg_A-4_F4
- GSM8708409 ALow_MD303_PBS_1 mg/kg_A-3_F3
- GSM8708410 ALow_MD303_PBS_1 mg/kg_A-2_F2
- GSM8708411 ALow_MD303_PBS_1 mg/kg_A-1_F1
- GSM8708412 ALow_MD303_1715157_10 mg/kg_F-2_G18
- GSM8708413 ALow_MD303_1715157_10 mg/kg_F-1_G17
- GSM8708414 ALow_MD303_1715157_10 mg/kg_F-3_G19
- GSM8708415 ALow_MD303_1715157_10 mg/kg_F-4_G20
- GSM8708416 ALow_MD303_1735511_10 mg/kg_M-2_H22
- GSM8708417 ALow_MD303_1735511_10 mg/kg_M-4_H24
- GSM8708418 ALow_MD303_1735511_10 mg/kg_M-3_H23
- GSM8708419 ALow_MD303_1735511_10 mg/kg_M-1_H21
- GSM8708420 ALow_MD303_1715157_100 mg/kg_H-2_H2
- GSM8708421 ALow_MD303_1715157_100 mg/kg_H-4_H4
- GSM8708422 ALow_MD303_1715157_100 mg/kg_H-3_H3
- GSM8708423 ALow_MD303_1715157_100 mg/kg_H-1_H1
- GSM8708424 ALow_MD303_1735511_100 mg/kg_O-2_I6
- GSM8708425 ALow_MD303_1735511_100 mg/kg_O-3_I7
- GSM8708426 ALow_MD303_1735511_100 mg/kg_O-4_I8
- GSM8708427 ALow_MD303_1735511_100 mg/kg_O-1_I5
- GSM8708428 ALow_MD303_1715157_30 mg/kg_G-3_G23
- GSM8708429 ALow_MD303_1715157_30 mg/kg_G-4_G24
- GSM8708430 ALow_MD303_1715157_30 mg/kg_G-1_G21
- GSM8708431 ALow_MD303_1715157_30 mg/kg_G-2_G22
+ 70 more — browse all 110 samples with per-sample file links →
Similar datasets
- GSE307527 ARID1A and ARID1B preserve B cell identity, prevent myeloid transformation and reveal therapeutic vulnerabilities 30 samples
- GSE328575 Single cell gene expression profiling of renal cell carcinoma 21 samples
- GSE303451 Engineered circular RNA compatible with complete nucleoside modification and rolling circle translation through a Cap-independent translation enhancer (CITE) 16 samples
- GSE236107 Single cell transcriptomic analysis of acute and early ART-treated HIV-1-infected CD4+T cells from a reservoir-marking humanized mouse model 14 samples
- GSE291551 Analysis of individual patient pathway coordination in a cross-species single-cell kidney atlas 11 samples
- GSE289420 Astrocyte-derived cholesterol drives synaptic gene expression in developing neurons and reciprocal astrocytic transcriptional programs 416 samples
- GSE320162 Minimizing far-extending chromatin perturbation in genome editing preserves stem cell identity [RNA-Seq] 347 samples
- GSE317764 High-Fat Diet Causes Rapid Loss of Intestinal Group 3 Innate Lymphoid Cells Through Microbiota-Driven Inflammation 270 samples
Share this dataset
Metadata from NCBI GEO, cached and refreshed periodically — the NCBI page above is authoritative. Downloads link straight to NCBI/ENA; nothing is proxied through BioTransfer.