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Rose, A.

Publications and source records attributed to Rose, A..

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An integrated single cell and spatial omics atlas of human prenatal development

Single cell genomics has enabled analysis of human prenatal development at unprecedented resolution. However, most studies have relied on dissociated tissues during restricted windows of development, limiting insights into how spatially distributed networks of cells, and multicellular niches emerge and adapt to distinct organ microenvironments in situ. Moreover, existing human developmental atlases have not yet been harmonised, and we thus lack a comprehensive catalogue of known cell types in the developing human body. Here, we introduce the Human Developmental Cell Atlas (HDCA), a unified structural, cellular and molecular resource for prenatal human development. The HDCA integrates published and unpublished single cell/nucleus RNAseq atlases across prenatal organs, and includes a newly generated, spatially resolved, multimodal cell atlas of intact human embryos. Spanning 4-22 post conceptional weeks, capturing embryonic and early to mid fetal stages, the HDCA contains [~]4.6 million cells/nuclei which resolve into [~]450 cell types, explorable with a bespoke web portal. For a global overview of the human embryos multicellular communities, we applied unsupervised deep learning to our intact human embryo spatial data, charting 114 tissue niches that are structural and signalling hubs for the cellular interactions of the embryo. Guided by these niches, we profiled cellular networks over space and time, not examinable using single-organ atlases. In so doing, we revealed tissue-specific fibroblast patterning from previously undescribed mesenchyme progenitors, early diversification of organ-specific blood capillaries and lymphatic vasculature, emergence of neural crest cell fates, the formation of placode-and neural crest-derived peripheral sensory neurons, and how tissue niches guide peripheral neuron maturation and axonal migration. The HDCA thus serves as a comprehensive step towards a comprehensive understanding of human prenatal development, and a template towards unravelling the biology of congenital disorders.

developmental biology↗

River pollution threatens preferred macroinvertebrate prey of endemic fishes in Aotearoa, New Zealand

O_LIMacroinvertebrates are a critical source of food for riverine fishes. Anthropogenic stressors can transform riverine macroinvertebrate communities, yet the consequences of such transformations to the fishes that feed on them are poorly understood. Changes in any combination of food abundance, accessibility or quality may be referred to as changes in the foodscape of fishes. Stressor-driven increases of inaccessible prey that are difficult for fish to detect, capture or ingest, may render foodscapes less profitable to fishes. C_LIO_LIWe examined how nutrient enrichment, fine sediment, acid mine drainage (AMD), flooding and drying reshape fish foodscapes by altering the accessibility of macroinvertebrate prey for two New Zealand river fishes, Galaxias vulgaris and Gobiomorphus breviceps. We determined relative accessibility of macroinvertebrate taxa and traits to these fishes by comparing the abundance of prey taxa and traits in the environment with their corresponding abundances in fish diets. To make these comparisons, invertebrate and fish gut samples were collected from the same river reaches, at the same times, over three years. Environmental DNA (eDNA) metabarcoding of fish gut contents facilitated efficient diet characterisation of over 700 fish guts. Finally, to determine how multiple stressors might affect fish foodscapes, we related invertebrate trait-specific accessibilities to published data defining how the composition of those macroinvertebrate traits varies along stressor gradients. C_LIO_LIMacroinvertebrate traits that were most accessible to fishes were those promoting detection (e.g., active foraging behaviours exhibited by crawlers and scrapers) and ingestion (e.g., moderate size, morphologies lacking defensive structures), typical of mayfly and free-living caddisfly larvae. In contrast, prey with traits inhibiting detection (e.g., small size and burrowing behaviour) and ingestion by gape-limited fishes (e.g., large size), were less accessible to the fishes we studied. C_LIO_LITrait abundance-stressor relationships revealed that that increasing fine sediment, nutrients, AMD and drying, and decreased flooding frequency, favoured macroinvertebrates with traits that erode accessibility to fishes. These results show that multiple stressors may decrease the profitability of fish foodscapes by filtering out macroinvertebrate prey with traits promoting accessibility to fishes. C_LIO_LIFood web approaches to understanding multi-stressor impacts are rare, but are necessary to understand how the effects of multiple stressors on primary consumers then go on to affect predators of higher trophic position. A major barrier to fish foodscape research is the difficulty and cost of estimating trophic networks. We discuss how eDNA metabarcoding, coupled with trait-based approaches may help overcome these barriers, enabling generalisable assessments of foodscape changes caused by multiple stressors. C_LI

ecology↗

Beyond Visual Inspection: Principled Benchmarking of Single-Cell Trajectory Representations with scTRAM

Single-cell representation learning aims to construct low-dimensional embeddings that preserve biologically salient manifold structures. Yet current evaluation protocols remain inadequate for assessing trajectory fidelity--essential for studying cell differentiation, immune responses, disease progression and perturbation--relying on heuristic visualizations or scalar surrogates that overlook multiscale trajectory geometry. To address this gap, we present scTRAM--single-cell trajectory representation metrics--a principled benchmarking framework that quantitatively evaluates how well single-cell embeddings retain groundtruth trajectories across complementary failure modes, from local neighborhood scrambling to global branch mis-ordering. Through our experiments, we show that scTRAM reveals modelspecific trade-offs in preserving distinct aspects of trajectory structure, with edge-specific decomposition exposing localized representational biases at high resolution. Systematic validation across diverse biological contexts confirms that the metric suite is sensitive to genuine structural properties rather than statistical artifacts. By shifting from qualitative, ad-hoc judgments to quantitative, principled approach, scTRAM enables rigorous comparison of embedding methods and informs downstream analyses where trajectory integrity is essential for biological interpretation.

bioinformatics↗

Integrating multi-covariate disentanglement with counterfactual analysis on synthetic data enables cell type discovery and counterfactual predictions.

Single-cell gene expression is influenced by diverse covariates such as genomics protocol, tissue origin, donor attributes, and microenvironment, which are challenging to disentangle. We present CellDISECT, a novel method combining disentangled representations and causal inference for multi-batch, multi-covariate single-cell data analysis. CellDISECT employs a mixture of expert variational autoencoders to learn covariate-specific and unsupervised latent spaces, enabling counterfactual predictions and biological discovery. Drawing inspiration from LLM training on synthetic data, CellDISECT generates synthetic counterfactuals during training and their quality is scored in the loss function. This semi-autoencoding of counterfactuals during training increases model performance in counterfactual predictions at test time. Benchmarking across datasets, CellDISECT outperformed existing methods in disentanglement, counterfactual in-silico prediction of responses to perturbations, and cell type discovery. CellDISECT predicted responses of cells to changing tissue microenvironments and identified a novel pre-natal megakaryocyte subpopulation with immune characteristics distinct from classical platelet-producing MKs, highlighting its unique capabilities in single-cell analysis to help identify novel subpopulations and reduce concerns of technical effects during integration.

genomics↗

Observing adaptation of duckweeds to their local microbiome depends on local pondwater

Populations can locally adapt to both the biotic and abiotic factors of an environment, but observing adaptation to biotic factors can sometimes depend on the abiotic conditions, and vice versa. One important aspect of the biotic environment is the microbiome: interactions between microbiomes and their hosts are critical for host fitness and trait expression. Hosts may adapt to their local microbiomes, and hosts may depend on interactions with microbes for adaptation to the local environment. Using Lemna minor (duckweed) as a model host organism, we examined differences in host fitness when grown in local and nonlocal microbiomes and in local and nonlocal water. We experimentally recombined duckweeds, microbes, and water from 4 different ponds around Durham, New Hampshire in well-plate microcosms in a growth chamber. Duckweed, microbe, and water source all affected microbial and duckweed growth, as well as duckweed traits. However, we observed only weak positive local adaptation that resulted in higher frond final area when duckweeds were paired with their local water and microbes. We also found that microbial growth was reduced when duckweeds were paired with microbes or water from their local site.

ecology↗

Fecal Metabolite Quantitation for Rapid Assessment of the Gut Microbiome

The intestinal microbiome is composed of myriad microbial species that produce metabolites that impact host health. While loss of bacterial species and beneficial metabolites from the fecal microbiome is associated with development of a range of diseases and medical complications, there are currently no diagnostic tests that rapidly identify individuals with microbiome deficiencies. Reduced concentrations of fecal butyrate and deoxycholic acid are associated with adverse clinical outcomes and result from the loss of a subset of health-associated bacterial species. We present a rapid diagnostic test based on 3-nitrophenylhydrazine derivatization and ultrahigh-performance liquid chromatography-mass spectrometry that measures fecal butyrate and deoxycholic acid concentrations as markers of microbiome function. A matrix-matched calibration curve was developed using a simulated fecal mixture to optimize accuracy and facilitate adherence to clinical laboratory regulations. The assay showed an analytical measurement range from 4.3-3030.1 {micro}M (LLOQ = 9.75 {micro}M) for butyrate and from 0.9- 64.9 {micro}M (LLOQ = 0.9 {micro}M) for deoxycholic acid. Precision demonstrated a coefficient of variation <15% at all QC levels tested. The assay can be performed in under an hour from extraction to provision of quantitative results, enabling the rapid identification of patients with defective microbiome function.

pathology↗

Mesoscale Explorer - Visual Exploration of Large-Scale Molecular Models

The advent of cryo-electron microscopy (cryo-EM) and cryo-electron tomography (cryo-ET), coupled with computational modeling, has enabled the creation of integrative 3D models of viruses, bacteria, and cellular organelles. These models, composed of thousands of macromolecules and billions of atoms, have historically posed significant challenges for manipulation and visualization without specialized molecular graphics tools and hardware. With the recent advancements in GPU rendering power and web browser capabilities, it is now feasible to render interactively large molecular scenes directly on the web. In this work, we introduce Mesoscale Explorer, a web application built using the Mol* framework, dedicated to the visualization of large-scale molecular models ranging from viruses to cell organelles. Mesoscale Explorer provides unprecedented access and insight into the molecular fabric of life, enhancing perception, streamlining exploration, and simplifying visualization of diverse data types, showcasing the intricate details of these models with unparalleled clarity. Statement: Mesoscale Explorer leverages advanced GPU rendering and web technologies to facilitate and democratize the interactive 3D visualization of large-scale molecular models from viruses to cellular organelles composed of millions of atoms. Mesoscale Explorer enables broader exploration and deeper understanding of the complex structure of these large molecular landscapes.

bioinformatics↗

TarDis: Achieving Robust and Structured Disentanglement of Multiple Covariates

Addressing challenges in domain invariance within single-cell genomics necessitates innovative strategies to manage the heterogeneity of multi-source datasets while maintaining the integrity of biological signals. We introduce TarDis, a novel deep generative model designed to disentangle intricate covariate structures across diverse biological datasets, distinguishing technical artifacts from true biological variations. By employing tailored covariate-specific loss components and a self-supervised approach, TarDis effectively generates multiple latent space representations that capture each continuous and categorical target covariate separately, along with unexplained variation. Our extensive evaluations demonstrate that TarDis outperforms existing methods in data integration, covariate disentanglement, and robust out-of-distribution predictions. The models capacity to produce interpretable and structured latent spaces, including its pioneering work in ordered latent representations for continuous covariates, markedly enhances its utility in hypothesis-driven research. Consequently, TarDis offers a promising analytical platform for advancing scientific discovery, providing insights into cellular dynamics, and enabling targeted therapeutic interventions. Progress and potentialModern single-cell genomics provides an unprecedented view into cellular heterogeneity, yet the very richness that propels new discoveries also complicates downstream analysis. Gene-expression patterns emerge from overlapping biological processes (e.g., differentiation programs, disease progression) and extrinsic factors (e.g., laboratory protocols, technical artifacts). Disentanglement, in this context, aims to parse these intertwined influences into interpretable latent representations, a crucial step for elucidating how complex covariates shape cellular states. While methods that correct for batch effects have become standard, these strategies often fall short in achieving the deeper objective of capturing subtle, high-dimensional biological dynamics. In single-cell experiments, cells navigate intricate developmental trajectories, respond nonlinearly to environmental or pharmaceutical perturbations, and exhibit myriad context-specific behaviors. Without disentanglement, these diverse signals frequently remain intermingled, limiting biological interpretability and hindering hypothesis-driven research. Disentangling biological covariates is particularly vital for addressing nuanced questions in single-cell research. For example, in a disease model involving multiple genetic variants and variable drug dosing, researchers may wish to examine the effect of each variant independently or investigate how dosage influences a specific mutant background. Similarly, in developmental biology, uncovering how cells evolve across a continuum of pseudotime (e.g., from pluripotent to fully differentiated states) is critical for identifying the genes that orchestrate fate decisions while isolating the influence of developmental time from tissue-specific contexts, along with other confounding factors such as culture conditions, sample preparation, or donor genetic characteristics. Alternatively, disentangling lineage commitment signals from spatial patterning cues enables the identification of master regulators driving fate decisions. Moreover, by explicitly isolating and representing each covariate as an independent latent dimension, one can systematically navigate and interrogate a rich multidimensional covariate space. This approach extends beyond merely observing biological states, it enables exploration of novel or unmeasured cellular conditions through latent-space manipulations. For instance, disentangled latent spaces could allow researchers to computationally predict cellular responses at drug dosages or developmental stages that were never experimentally observed, significantly broadening the scope and predictive power of experimental datasets. Such analyses yield testable hypotheses for unexplored biological phenomena and enable informed planning of subsequent experimental validations. The challenge of covariate disentanglement stems fundamentally from the complexity of modeling joint distributions of gene expression conditioned simultaneously on multiple covariates, both categorical (e.g., tissue type, disease condition) and continuous (e.g., pseudotime, dosage). This is inherently an underdetermined problem because single-cell measurements represent only sparse snapshots within a vast combinatorial space of covariate conditions. Conventional modeling approaches often conflate correlated covariates, collapsing biological variability into ambiguous latent factors, and typically fail to explicitly create separate latent representations for disentangled covariates. Moreover, continuous covariates introduce an additional layer of complexity; yet discretizing them artificially imposes arbitrary boundaries, obscuring subtle transitions and hindering accurate capture of biological gradients. Therefore, preserving the continuous nature of such covariates in disentangled representations is critical, as it maintains their intrinsic ordering and enables researchers to discern nuanced biological shifts--such as identifying thresholds in dose-response relationships or characterizing gradual developmental transitions--in a naturally interpretable manner. The key idea in this paper is to devise a tailored deep generative model for systematically separating both categorical and continuous covariates into independent latent dimensions, while still ensuring coherent integration of the underlying gene-expression data. By explicitly targeting these covariates and preserving continuous variables as smooth, ordered latent axes, our approach clarifies complex interactions and uncovers nuanced patterns that remain concealed under standard analyses. The resulting disentangled representations can then support robust out-of-distribution generalizations, refined differential analyses, and more principled hypotheses about how diverse factors interact to drive cellular variation.

bioinformatics↗

Protection against Clostridioides difficile disease by a naturally avirulent C. difficile strain

Clostridioides difficile (C. difficile) strains belonging to the epidemic BI/NAP1/027 (RT027) group have been associated with increased transmissibility and disease severity. In addition to the major toxin A and toxin B virulence factors, RT027 strains also encode the CDT binary toxin. Our lab previously identified a toxigenic RT027 isolate, ST1-75, that is avirulent in mice despite densely colonizing the colon. Here, we show that co-infecting mice with the avirulent ST1-75 and virulent R20291 strains protects mice from colitis due to rapid clearance of the virulent strain and persistence of the avirulent strain. Although avirulence of ST1-75 is due to a mutation in the cdtR gene, which encodes a response regulator that modulates the production of all three C. difficile toxins, the ability of ST1-75 to protect against acute colitis is not directly attributable to the cdtR mutation. Metabolomic analyses indicate that the ST1-75 strain depletes amino acids more rapidly than the R20291 strain and supplementation with amino acids ablates ST1-75s competitive advantage, suggesting that the ST1-75 strain limits the growth of virulent R20291 bacteria by amino acid depletion. Since the germination kinetics and sensitivity to the co-germinant glycine are similar for the ST1-75 and R20291 strains, our results identify the rapidity of in vivo nutrient depletion as a mechanism providing strain-specific, virulence-independent competitive advantages to different BI/NAP1/027 strains. They also suggest that the ST1-75 strain may, as a biotherapeutic agent, enhance resistance to CDI in high-risk patients. ImportanceClostridioides difficile infections (CDI) are prevalent in healthcare settings and are associated with high recurrence rates. Therapies to prevent CDI, including recent FDA-approved live biotherapeutic products, are costly and have not been used to prevent primary infections. While a nontoxigenic C. difficile strain (NTCD-M3) protects against virulent CDI in animals and reduced CDI recurrence in a phase 2 clinical trial, protection against CDI recurrence in humans was variable and required high doses of the nontoxigenic strain. Here we show that an avirulent C. difficile isolate, ST1-75, efficiently outcompetes virulent C. difficile strains in mice when co-infected at a 1:1 ratio. Our data suggest that inter-strain competition results from ST1-75s more rapid depletion of amino acids than the virulent R20291 strain. Our study identifies inter-strain nutrient depletion as a potentially exploitable mechanism to reduce the incidence of CDI.

microbiology↗

Comprehensive analyses of a large human gut Bacteroidales culture collection reveal species and strain level diversity and evolution

Species of the Bacteroidales order are among the most abundant and stable bacterial members of the human gut microbiome with diverse impacts on human health. While Bacteroidales strains and species are genomically and functionally diverse, order-wide comparative analyses are lacking. We cultured and sequenced the genomes of 408 Bacteroidales isolates from healthy human donors representing nine genera and 35 species and performed comparative genomic, gene-specific, mobile gene, and metabolomic analyses. Families, genera, and species could be grouped based on many distinctive features. However, we also show extensive DNA transfer between diverse families, allowing for shared traits and strain evolution. Inter- and intra-specific diversity is also apparent in the metabolomic profiling studies. This highly characterized and diverse Bacteroidales culture collection with strain-resolved genomic and metabolomic analyses can serve as a resource to facilitate informed selection of strains for microbiome reconstitution.

microbiology↗

A human prenatal skin cell atlas reveals immune cell regulation of skin morphogenesis

Human prenatal skin is populated by innate immune cells including macrophages, and whether they act solely in immunity or have additional functions in morphogenesis is unclear. We assembled the first comprehensive multi-omic reference atlas of prenatal human skin (7-16 post-conception weeks), combining single cell and spatial transcriptomic data, to characterise the skins microenvironmental cellular organisation. This revealed that crosstalk between non-immune and immune cells underpins formation of hair follicles, has implications for scarless wound healing, and is critical for skin angiogenesis. We benchmarked a skin organoid model, derived from human embryonic stem (ES) and induced pluripotent stem (iPS) cells, against prenatal and adult skin, demonstrating close recapitulation of the epidermal and dermal skin components during hair follicle development. Notably, the skin organoid lacked immune cells and had markedly diminished endothelial cell heterogeneity and quantity. From our in vivo skin cell atlas data, we found that macrophages and macrophage-derived growth factors play a key role in driving endothelial development prenatally. Indeed, vascular network formation was enhanced following transfer of autologous iPS-derived macrophages into both endothelial cell angiogenesis assays and skin organoid cultures. In summary, innate immune cells moonlight as key players in skin morphogenesis beyond their conventional immune roles, a function they achieve via extensive crosstalk with non-immune cells. Finally, we leveraged our human prenatal skin cell atlas to further our understanding of the pathogenesis of genetic hair and skin disorders.

developmental biology↗

Targeting DNA2 Overcomes Metabolic Reprogramming in Multiple Myeloma

DNA damage resistance is a major barrier to effective DNA-damaging therapy in multiple myeloma (MM). To discover novel mechanisms through which MM cells overcome DNA damage, we investigated how MM cells become resistant to antisense oligonucleotide (ASO) therapy targeting ILF2, a DNA damage regulator that is overexpressed in 70% of MM patients whose disease has progressed after standard therapies have failed. Here, we show that MM cells undergo an adaptive metabolic rewiring and rely on oxidative phosphorylation to restore energy balance and promote survival in response to DNA damage activation. Using a CRISPR/Cas9 screening strategy, we identified the mitochondrial DNA repair protein DNA2, whose loss of function suppresses MM cells ability to overcome ILF2 ASO-induced DNA damage, as being essential to counteracting oxidative DNA damage and maintaining mitochondrial respiration. Our study revealed a novel vulnerability of MM cells that have an increased demand for mitochondrial metabolism upon DNA damage activation. STATEMENT OF SIGNIFICANCEMetabolic reprogramming is a mechanism through which cancer cells maintain survival and become resistant to DNA-damaging therapy. Here, we show that targeting DNA2 is synthetically lethal in myeloma cells that undergo metabolic adaptation and rely on oxidative phosphorylation to maintain survival after DNA damage activation.

cancer biology↗

Discovery and characterization of LNCSOX17 as an essential regulator in human endoderm formation

Long non-coding RNAs (lncRNAs) have emerged as fundamental regulators in various biological processes, including embryonic development and cellular differentiation. Despite much progress over the past decade, the genome-wide annotation of lncRNAs remains incomplete and many known non-coding loci are still poorly characterized. Here, we report the discovery of a previously not annotated lncRNA that is transcribed upstream of the SOX17 gene and located within the same topologically associating domain. We termed it LNCSOX17 and show that it is induced following SOX17 activation but its expression is more tightly restricted to early definitive endoderm. Loss of LNCSOX17 affects crucial functions independent of SOX17 and leads to an aberrant endodermal transcriptome, signaling pathway deregulation and epithelial to mesenchymal transition defects. Consequently, cells lacking the lncRNA cannot further differentiate into more mature endodermal cell types. We identified and characterized LNCSOX17 as an essential new actor in early human endoderm, thereby further expanding the list of functionally important non-coding regulators.

genomics↗

Multi-organ functions of yolk sac during human early development

The yolk sac (YS) represents an evolutionarily-conserved extraembryonic structure that ensures timely delivery of nutritional support and oxygen to the developing embryo. However, the YS remains ill-defined in humans. We therefore assemble a complete single cell 3D map of human YS from 3-8 post conception weeks by integrating multiomic protein and gene expression data. We reveal the YS as a site of primitive and definitive haematopoiesis including a YS-specific accelerated route to macrophage production, a source of nutritional/metabolic support and a regulator of oxygen-carrying capacity. We reconstruct the emergence of primitive haematopoietic stem and progenitor cells from YS hemogenic endothelium and their decline upon stromal support modulation as intraembryonic organs specialise to assume these functions. The YS therefore functions as three organs in one revealing a multifaceted relay of vital organismal functions as pregnancy proceeds. One Sentence SummaryHuman yolk sac is a key staging post in a relay of vital organismal functions during human pregnancy.

developmental biology↗

Ancient herpes simplex 1 genomes reveal recent viral structure in Eurasia

Human herpes simplex virus 1 (HSV-1), a life-long infection spread by oral contact, today infects a majority of adults globally1, yet no ancient HSV-1 genomes have yet been published. Phylogeographic clustering of sampled diversity into European, pan-Eurasian, and African groups2, 3 has suggested that the virus co-diverged with anatomically modern humans migrating out of Africa4, although a much younger origin has also been proposed5. The lack of ancient HSV-1 genomes, high rates of recombination, and high mobility of humans in the modern era have impeded the understanding of HSV-1s evolutionary history. Here we present three full ancient European HSV-1 genomes and one partial genome, dating to between the 3rd and 17th century CE, sequenced to up to 9.5x with paired human genomes up to 10.16x. These HSV-1 strains fall within modern Eurasian diversity. We estimate a mean mutation rate of 7.6 x 10-7 - 1.13 x 10-6 for non-African diversity leading to an estimated age of sampled modern Eurasian diversity to 4.68 (3.87 - 5.65) kya. Extrapolation of these rates indicate the age of sampled HSV-1 to 5.29 (4.60-6.12 kya, suggesting lineage replacement coinciding with late Neolithisation and implicating Bronze Age migrations6 in the distribution of HSV-1 through Eurasia.

genomics↗

Liver x receptor alpha drives chemoresistance in response to side-chain hydroxycholesterols in triple negative breast cancer

Triple negative breast cancer (TNBC) is challenging to treat successfully because targeted therapies do not exist. Instead, systemic therapy is typically restricted to cytotoxic chemotherapy, which fails more often in patients with elevated circulating cholesterol. Liver x receptors are ligand-dependent transcription factors that are homeostatic regulators of cholesterol, and are linked to regulation of broad-affinity xenobiotic transporter activity in non-tumor tissues. We show that LXR ligands confer chemotherapy resistance in TNBC cell lines and xenografts, and that LXRalpha is necessary and sufficient to mediate this resistance. Furthermore, in TNBC patients who had cancer recurrences, LXRalpha and ligands were independent markers of poor prognosis and correlated with P-glycoprotein expression. However, in patients who survived their disease, LXRalpha signaling and P-glycoprotein were decoupled. These data reveal a novel chemotherapy resistance mechanism in this poor prognosis subtype of breast cancer. We conclude that systemic chemotherapy failure in some TNBC patients is caused by co-opting the LXRalpha:P-glycoprotein axis, a pathway highly targetable by therapies that are already used for prevention and treatment of other diseases.

cancer biology↗

Assessing the co-variability of DNA methylation across peripheral cells and tissues: implications for the interpretation of findings in epigenetic epidemiology

Summary/AbstractO_ST_ABSBackgroundC_ST_ABSMost epigenome-wide association studies (EWAS) quantify DNA methylation (DNAm) in peripheral tissues such as whole blood to identify positions in the genome where variation is statistically associated with a trait or exposure. As whole blood comprises a mix of cell types, it is unclear whether trait-associated variation is specific to an individual cellular population. MethodsWe collected three peripheral tissues (whole blood, buccal and nasal epithelial cells) from thirty individuals. Whole blood samples were subsequently processed using fluorescence-activated cell sorting (FACS) to purify five constituent cell-types (monocytes, granulocytes, CD4+ T cells, CD8+ T cells, and B cells). DNAm was profiled in all eight sample-types from each individual using the Illumina EPIC array. ResultsWe identified significant differences in both the level and variability of DNAm between different tissues and cell types, and DNAm data-derived estimates of age and smoking were found to differ dramatically across sample types from the same individual. We found that for the majority of loci variation in DNAm in individual blood cell types was only weakly predictive of variance in DNAm measured in whole blood, however, the proportion of variance explained was greater than that explained by either buccal or nasal tissues. Instead we observe that DNAm variation in whole blood is additively influenced by a combination of the major blood cell types. For a subset of sites variable DNAm detected in whole blood can be attributed to variation in a single blood cell type providing potential mechanistic insight. ConclusionsWe identified major differences in DNAm between blood cell types and peripheral tissues, with each sample type being characterized by a unique DNAm signature across multiple loci. Our results suggest that associations between whole blood DNAm and traits or exposures reflect differences in multiple cell types and provide important insights for the interpretation of EWAS performed in whole blood. Key MessagesO_LIWe identified major differences in DNA methylation between blood cell types and peripheral tissues, with each sample type being characterized by a unique DNA methylation signature across multiple loci. C_LIO_LIEstimates of DNAmAge and tobacco smoking from DNA methylation data can be highly variable across different sample types collected from the same individual at the same time. C_LIO_LIWhile individual blood cell types did predict more of the variation in whole blood compared to buccal epithelial and nasal epithelial cells, the percentage of variance explained was still small. C_LIO_LIInstead our data indicate that at the majority of sites, variation in multiple blood cell types additively combines to drive variation in DNA methylation in whole blood. C_LIO_LIThere are subset of sites where variable DNA methylation detected in whole blood can be attributed to variation in a single blood cell type. C_LI

genomics↗

Modelling erythropoiesis in congenital dyserythropoietic anaemia type I (CDA-I)

We employ and extensively characterise an ex vivo culture system to study terminal erythroid maturation of CD34+ progenitors from the peripheral blood of normal individuals and patients with Congenital Dyserythropoietic Anaemia type 1 (CDA-I). Using morphological analysis, FACS analysis and the proteomic approach CyTOF, we analysed patient-derived erythroblasts stage-matched with those from healthy donors during the expansion phase and into early differentiation. In patient cells, aspects of disordered erythropoiesis manifest midway through differentiation, including increased proliferation and changes in the DNA accessibility profile. We also show that cultured erythroblasts from CDA-I patients recapitulate the pathognomic feature of this erythroid disorder with up to 40% of the cells having abnormal spongy chromatin morphology by electron microscopy, as well as upregulation of GDF15, a marker of ineffective erythropoiesis. In the tertiary phase of culture, patient cells show significantly less enucleation and there is persistence of earlier erythroid precursors. Furthermore, the enucleation defect appears to be more severe in patients with mutations in C15orf41, as compared to the other known causative gene CDAN1, indicating a genotype/phenotype correlation in CDA-I. Such erythroblasts are a valuable resource for investigating the pathogenesis of this disease and provide the opportunity for streamlining diagnosis for CDA-I patients and ultimately other forms of unexplained anaemia.

genetics↗