Deliverome GO Collaboration

IN_PROGRESS

Species: human, mouse

Deliverome GO Collaboration

Overview

The Deliverome Project proposes an open atlas of human surface proteins for targeted therapeutic delivery. The public framing emphasizes surface abundance, tissue and cell-type specificity, internalization, downstream trafficking, and functional delivery outcome. This AIGR project tracks two directions:

This PR keeps the work scoped to project framing plus a human and mouse Rab7 comparison. It does not add a full Deliverome pilot review set.

What "Delivery Address" Means

By "delivery address" I mean a gene product that therapeutic cargo can use as a molecular address. In practice this is usually an extracellular-facing cell-surface protein with a usable ligand-binding epitope, appropriate tissue or cell-type distribution, and a trafficking route that carries bound cargo toward a useful destination.

The address is not just "present on the membrane." The key questions are whether it internalizes, recycles, routes to lysosomes, transcytoses, or releases cargo productively.

How GO Could Contribute

GO can provide an evidence-aware prior over plausible human delivery-address proteins before Deliverome-specific measurements are available. Useful filters include cellular-component annotations to cell surface, external side of plasma membrane, plasma membrane, receptor complexes, and endosomal compartments, plus molecular-function or biological-process annotations for receptor activity, endocytosis, transcytosis, and ligand uptake.

GO is also useful as a vocabulary for assay outputs: surface localization, receptor internalization, receptor-mediated endocytosis, recycling, lysosomal routing, transcytosis, and endosome-to-plasma-membrane transport. These terms can support data dictionaries and triage even when a particular engineered-delivery result should not become a canonical GO annotation.

GO-CAMs are especially useful where delivery value depends on a causal route:

  1. ligand or cargo binding at the external side of the plasma membrane;
  2. receptor internalization;
  3. early endosome entry;
  4. sorting toward recycling endosome, late endosome, lysosome, transcytosis, or cytosolic escape;
  5. downstream endogenous or engineered payload effect.

For normal biology this can become ordinary GO-CAM curation. For engineered therapeutic cargo, the same shape is better treated as a GO-CAM-like Deliverome model outside canonical GO.

How Deliverome Data Could Contribute To GO

Deliverome data could support standard GO annotations when the assay measures a normal property of the endogenous gene product:

Deliverome evidence Potential GO contribution Curation caution
Surface proteomics shows endogenous protein at the cell surface Cellular-component annotations such as cell surface, external side of plasma membrane, or plasma membrane Require localization specificity; bulk tissue data may need cell-type context
Internalization assay shows endogenous receptor uptake Receptor internalization or receptor-mediated endocytosis Engineered cargo uptake alone may be a delivery phenotype
Trafficking assay tracks the receptor through endosomes or lysosomes Compartment and routing annotations when the receptor itself is observed Do not annotate receptor destination if only payload destination was measured
Perturbation screen identifies delivery-routing machinery Review genes for endocytosis, vesicle transport, recycling, lysosome targeting, or transcytosis Separate direct trafficking machinery from indirect viability or cell-state effects
Low-internalization result for an expected receptor Possible NOT annotation only in narrow cases Failed delivery is usually not enough for a GO NOT annotation

Model Systems

Human and mammalian systems should be primary for delivery-address biology because useful addresses depend on human cell-surface abundance, ligand specificity, glycosylation, expression context, and safety. Good near-term systems are primary or iPSC-derived hepatocytes for ASGR1/ASGR2, polarized endothelial or epithelial barrier systems for FCGRT and TFRC transcytosis, immune and myeloid cells for FcRn and ESCRT/retromer routing, and cancer cell lines or organoids for oncology delivery targets.

Fission yeast (Schizosaccharomyces pombe) is useful for machinery-level internal trafficking: conserved endocytosis, ESCRT, retromer, Rab/Ypt, actin-polarity, and vacuolar/lysosomal routing. It is not a good model for human delivery addresses themselves because most address receptors and extracellular ligand-binding systems are metazoan or mammalian.

Mouse is important for pharmacokinetics, immune context, placenta/neonatal Fc receptor biology, in vivo biodistribution, and post-internalization routing. Species differences in receptor expression and ligand affinity still need explicit tracking.

The mouse Rab7/Rab7a review gives this project a concrete internal-routing model. Rab7 is not a delivery-address receptor; it is a late-endosome/lysosome routing switch that helps decide whether internalized cargo proceeds toward degradation, recycling/retrograde sorting, or productive escape. Mouse liver LysoTag/LNP evidence shows that Rab7 loss increases LNP cytosolic escape, making mouse Rab7 useful for "after internalization" questions that cannot be resolved from human surface abundance alone.

The human and mouse comparison is reassuring for GO transfer: both reviews converge on conserved Rab-family GTPase activity, early-to-late endosome maturation, endosome-to-lysosome transport, phagosome/autophagosome fusion, and retromer-dependent retrograde traffic. Human RAB7A remains the stronger disease/genetics anchor, especially for CMT2B, while mouse Rab7/Rab7a is the stronger Deliverome model-system anchor because it connects Rab7-mediated endosomal maturation to in vivo LNP escape.

Question Best-fit model systems Good pilot proteins
Human address specificity and uptake Human primary/iPSC/organoid/cell-line panels ASGR1, ASGR2, SORT1, TFRC, FCGRT
Clathrin/AP-2 uptake machinery Human cells, yeast for conserved principles, fly/worm neurons for specialized uptake CLTC, AP2M1, DAB2, LDLRAP1
ESCRT/MVB and lysosomal routing Human cells, pombe/budding yeast, worm and fly for organismal genetics HGS, RAB7A
Retromer and recycling Human cells, pombe/budding yeast, worm and fly neurons and epithelia VPS35, RAB11A
Barrier/transcytosis delivery Human BBB/epithelial models, mouse and zebrafish for in vivo context FCGRT, TFRC
Endosomal escape versus degradative routing Mouse liver LysoTag/LNP assays plus matched human-cell trafficking assays RAB7A/Rab7a, HGS, VPS35

GO Anchors

Deliverome concept Current GO anchor
Surface localization GO:0009986 cell surface
Extracellular-facing membrane localization GO:0009897 external side of plasma membrane
Plasma membrane localization GO:0005886 plasma membrane
Protein localization to surface GO:0034394 protein localization to cell surface
Internalization GO:0031623 receptor internalization
General endocytosis GO:0006897 endocytosis
Receptor-mediated uptake GO:0006898 receptor-mediated endocytosis
Clathrin-dependent uptake GO:0072583 clathrin-dependent endocytosis
Caveolin-mediated uptake GO:0072584 caveolin-mediated endocytosis
Transcellular routing GO:0045056 transcytosis
Early endosome GO:0005769 early endosome
Late endosome GO:0005770 late endosome
Recycling route GO:0055037 recycling endosome
Lysosomal destination GO:0005764 lysosome
Early-to-late endosome route GO:0045022 early endosome to late endosome transport
Late endosome-to-lysosome route GO:1902774 late endosome to lysosome transport
Recycling to surface GO:0099638 endosome to plasma membrane protein transport

Open Questions


STATUS

2026-07-19

NOTES

2026-07-19