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A humanized monoclonal antibody targeting human ENPP1, an ectonucleotidase expressed in the diseased human kidney, enhances tubular proliferation and kidney repair and is safe in non-human primates

GSE330629 Mus musculus (mouse) Single-cell RNA-seq 24 samples Submitted 2026/05/16 Platform GPL21103

At a glance

Single-cell RNA-seq · mouse · 24 samples

What it is: Single-cell RNA-seqGene activity measured in thousands of individual cells, so cell types can be told apart. Read from the summary: GEO's own type field only says 'expression profiling by sequencing', so confirm it in the paper.
Organism: Mus musculus (mouse)
Samples: 24 samples: 5 conditions, mostly with about 4 replicates eachGroups 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.

Summary (written by the submitters)

The human kidney possesses a poor ability to robustly regenerate after acute injury and chronic kidney disease affects millions of individuals worldwide. There are no drugs that enhance tissue repair after acute kidney injury. Here, we demonstrate, in a cohort of individuals with kidney disease, that the ectonucleotidase ENPP1 (ectonucleotide pyrophosphatase/phosphodiesterase-1) is robustly expressed in diseased human kidneys and strongly correlates with clinical indices of renal dysfunction.

Read the full summary ↓

Genetic targeting of ENPP1 in mouse models of kidney disease enhanced renal repair, decreased fibrosis and was associated with significant rescue of post injury kidney function. A humanized monoclonal antibody, targeting the catalytic domain of human ENPP1 when administered to humanized mouse models led to proliferation of tubular cells, enhanced renal glomerular filtration rate, decreased fibrosis and rescued kidney function in multiple pre-clinical models of kidney injury. In animals that express human ENPP1 and exhibit clearance kinetics of immunoglobulins similar to those in humans, a single dose of the monoclonal was sufficient to rescue kidney function after an acute insult. Metabolic profiling demonstrated that antibody mediated ENPP1 inhibition led to beneficial effects on nucleotide metabolism and cellular energetics enabling proliferation and rescuing cell cycle arrest. Single cell transcriptomics and pulse chase labeling demonstrated that hENPP1mAb promotes tubular repair by expanding regenerative tubular cell subsets and reducing populations associated with failed repair. In a multi dose GLP (good laboratory practice) compliant study, the humanized monoclonal antibody was found to be non-toxic and highly tolerated when administered to non-human primates at high doses. Taken together, our findings identify ENPP1 as a central regulator of epithelial injury and metabolic control after acute kidney injury and provide proof of concept of an engineered humanized monoclonal antibody targeting human ENPP1, as a potential therapeutic agent for enhancing human kidney repair.

This dataset

Open GSE330629 in finder → Find more mouse RNA-seq datasets →

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.

Series matrix ↓

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 GSE330629_RAW.tar (it can run to tens of gigabytes).

All supplementary files ↗ Whole study · RAW.tar ↓

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.

Raw reads on ENA ↗

What the files usually look like: Single-cell studies usually ship their counts as 10x files (matrix.mtx, barcodes.tsv, features.tsv), an .h5 file, or an .rds / .h5ad object that Seurat or Scanpy can open. Look for those among the supplementary files.

Also filed as BioProject PRJNA1464733 and SRA study SRP699451. Searching any of these in the dataset finder brings you back here.

NCBI GEO page ↗

Study design

5 conditions, mostly with about 4 replicates each

ENPP1 knockout adenine ×6 wild-type adenine diet ×6 CHOW diet control ×4 hENPP1 monoclonal antibody ×4 IgG ×4

Supports a case/control comparison: 6 samples read as cases, 10 as controls.

How we read this: 5 replicated groups read from 24 sample titles; they account for 24 of them. 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

GSM9729534 ENPP1 knockout adenine rep 1 ENPP1 knockout adenine
GSM9729535 ENPP1 knockout adenine rep 2 ENPP1 knockout adenine
GSM9729536 ENPP1 knockout adenine rep 3 ENPP1 knockout adenine
GSM9729537 ENPP1 knockout adenine rep 4 ENPP1 knockout adenine
GSM9729538 ENPP1 knockout adenine rep 5 ENPP1 knockout adenine
GSM9729539 ENPP1 knockout adenine rep 6 ENPP1 knockout adenine
GSM9729540 wild-type adenine diet rep 1 wild-type adenine diet
GSM9729541 wild-type adenine diet rep 2 wild-type adenine diet
GSM9729542 wild-type adenine diet rep 3 wild-type adenine diet
GSM9729543 wild-type adenine diet rep 4 wild-type adenine diet
GSM9729544 wild-type adenine diet rep 5 wild-type adenine diet
GSM9729545 wild-type adenine diet rep 6 wild-type adenine diet
GSM9729546 CHOW diet control rep 1 CHOW diet control
GSM9729547 CHOW diet control rep 2 CHOW diet control
GSM9729548 CHOW diet control rep 3 CHOW diet control
GSM9729549 CHOW diet control rep 4 CHOW diet control
GSM9729550 hENPP1 monoclonal antibody rep 1 hENPP1 monoclonal antib…
GSM9729551 hENPP1 monoclonal antibody rep 2 hENPP1 monoclonal antib…
GSM9729552 hENPP1 monoclonal antibody rep 3 hENPP1 monoclonal antib…
GSM9729553 hENPP1 monoclonal antibody rep 4 hENPP1 monoclonal antib…
GSM9729554 IgG rep 1 IgG
GSM9729555 IgG rep 2 IgG
GSM9729556 IgG rep 3 IgG
GSM9729557 IgG rep 4 IgG

Datasets on a similar topic

GSE330642 A humanized monoclonal antibody targeting human ENPP1, an ectonucleotidase expressed in the diseased human kidney, enhances tubular proliferation and kidney repair and is safe in non-human primates [scRNA-seq] 11 samples shares enpp1, ectonucleotidase, proliferation, monoclonal, humanized, targeting, expressed, non-human
GSE255826 A Humanized Monoclonal Antibody Targeting an Ectonucleotidase Rescues Heart Function after Myocardial Infarction 10 samples shares ectonucleotidase, monoclonal, humanized, targeting
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GSE277788 Oral ENPP1 inhibitor designed using generative AI as next generation STING modulator for solid tumors 12 samples shares enpp1
GSE309379 Nucleophosmin supports WNT-driven hyperproliferation and tumour initiation 111 samples shares proliferation
GSE296763 Mitochondrial respiration is necessary for CD8 T cell proliferation and cell fate 99 samples shares proliferation

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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.