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Medicinal cannabis plant extract (NTI164) modifies epigenetic, ribosomal, and immune pathways in Paediatric Acute-onset Neuropsychiatric Syndrome

GSE299764 Homo sapiens (human) RNA-seq (gene expression) 24 samples Submitted 2026/03/18 Platform GPL24676
At a glance

RNA-seq (gene expression) · human · 24 samples

What it is
RNA-seq (gene expression)Gene activity measured by sequencing. The summary does not mention single-cell work, so this is most likely bulk: one average per sample, the usual starting point for comparing groups.
Organism
Homo sapiens (human)
Samples
24 samples: 17 distinct titles, 1 of them repeatedGroups are read from the sample names, not from the paper. Check the list below.
Data you can get
A processed table (series matrix) and supplementary files from NCBI; the raw sequencing reads from ENA. Which of these hold counts depends on the study, so check the file list.
Paper
Medicinal cannabis plant extract (NTI164) modifies epigenetic, ribosomal, and immune pathways in paediatric acute-onset neuropsychiatric syndromeNeurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2026
Summary (written by the submitters)
Paediatric Acute-onset Neuropsychiatric Syndrome (PANS) is a syndrome of infection-provoked abrupt-onset obsessive-compulsive disorder (OCD) or eating restriction. This open-label trial investigated the safety and efficacy of NTI164, a cannabis-derived medicinal formulation rich in cannabinoids (minimal tetrahydrocannabinol), at 20mg/kg/day for 12 weeks in 14 children with chronic-relapsing PANS (mean 12.1 years, females 29%).
Read the full summary ↓
Clinical assessments demonstrated significant improvements with NTI164 in disease severity, emotional regulation, anxiety, OCD, ADHD symptoms, and overall quality of kife (all p<0.05). Cytokine and multi-omic analyses, including transcriptomics, proteomics, phosphoproteomics, and DNA methylation revealed baseline dysregulation in epigenetic (chromatin remodelling, methylation, histone modifications, transcription factors), ribosomal, immune, and signalling pathways in PANS versus controls. These abnormalities were significantly modified following NTI164 treatment. These results suggest NTI164 has broad epigenetic and immune modulatory effects, modifying baseline immune cell abnormalities in PANS. While further controlled studies are warranted, NTI164 shows promise as a novel therapeutic for PANS.
Published in
Medicinal cannabis plant extract (NTI164) modifies epigenetic, ribosomal, and immune pathways in paediatric acute-onset neuropsychiatric syndrome
Keating BA, Han VX, Nishida H et al. · Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics 2026 · PMID 41513541 · doi:10.1016/j.neurot.2025.e00828
This dataset
Download · which file do I need?

Direct links to NCBI and ENA, no account and no request form. Pick by what you want to do: take the small processed table to look, the supplementary files to re-analyse, or the original raw data to start over.

1 · Just have a look

Processed table

The series matrix: one table of values per sample as the submitters processed it. The smallest download, and it opens in Excel or R. Start here to see what the data look like.

2 · Re-analyse it

Supplementary files

The count tables and other files the authors uploaded. Take only the samples you need, or the whole study as GSE299764_RAW.tar (it can run to tens of gigabytes).

3 · Start from the raw reads

Raw sequencing reads

The original FASTQ files, straight from the sequencer, held at ENA. Large, and you align them yourself, so only choose this if you need to re-process the study from scratch.

What the files usually look like: For RNA-seq the counts are usually a table among the supplementary files (look for 'counts', 'expression', 'FPKM' or 'TPM' in the file name). The series matrix of a sequencing study is sometimes nearly empty.

Also filed as BioProject PRJNA1276472 and SRA study SRP591893. Searching any of these in the dataset finder brings you back here.

Study design
17 distinct titles, 1 of them repeated
Whole blood, healthy control ×8

How we read this: Read from 24 sample titles: 17 distinct titles with little repetition. These groups are guessed from the sample names, not taken from the paper, so check them against the sample list below before relying on them.

Samples in this study
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