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Biology subjects

Iacono, G.

Publications and source records attributed to Iacono, G..

10 recordsLinked to original sources

Generation of a novel Slc7a9G105R mutant mouse identifies new biomarkers for cystinuria

Cystinuria is a rare inherited disease characterized by increased urinary cystine levels resulting in the formation of cystine stones in the urinary tract. Mutations in the genes encoding the cystine transporter complex, SLC3A1 and SLC7A9, are the primary drivers of the disease. Current mouse models used to study cystinuria rely on gene deficiency or spontaneous mutations in mice that do not accurately reflect the pathogenic mutations found in humans. To overcome this limitation, we generated novel Slc7a9G105R mice carrying the most common pathogenic single-point mutation in the SLC7A9 gene. Both male and female Slc7a9G105R mice developed a cystinuria phenotype by 9 weeks of age, characterized by substantial cystine stone formation and increased urinary cystine, lysine, arginine, and ornithine. Slc7a9G105R mice displayed distinct serum and urinary metabolite profiles mapped to dibasic amino acid pathways. Fecal metagenomics revealed that Slc7a9G105R mice had a heterogeneous microbiota with altered functional pathways, including increased L-cysteine biosynthesis. Depletion of the microbiota with antibiotics did not impact cystine stone burden but reduced urinary tract inflammation. Prophylactic or therapeutic dietary supplementation with alpha-lipoic acid reduced stone burden and inflammation, but it also caused damage to the urothelium. Untargeted metabolomics analysis following alpha-lipoic acid supplementation identified metabolites that can increase cystine solubility, reduce inflammation, and damage epithelial cells. Correlation analysis revealed novel serum biomarkers of stone burden, including blood urea nitrogen, 2-hydroxybutyric acid, 2-amino-2-thiazoline-4-carboxylic acid, and indole-3-acetylglycine. Collectively, the Slc7a9G105R mutant mouse model offers a precise, rapid-onset, and translational platform for investigating cystinuria pathogenesis and evaluating potential therapeutic strategies. Translational statementThe development of a novel knock-in mouse model carrying the most common pathogenic point mutation in the human SLC7A9 gene provides a clinically relevant and translationally valuable platform for investigating cystinuria pathogenesis and testing emerging therapies. This model represents the closest possible approximation of human SLC7A9-mediated cystinuria, enabling rigorous preclinical evaluation of small molecules and gene therapies. It has also facilitated the identification of candidate biomarkers for cystine stone burden and treatment response, which are urgently needed to improve disease monitoring and clinical decision-making. The next critical step is to validate these biomarkers in human cystinuria cohorts to support their clinical translation.

pathology↗

Differentiating erythroblasts adapt to turbulent flow by accelerating maturation and activating cholesterol biosynthesis.

In vitro culture of erythroblasts (EBL) and production of mature erythrocytes for transfusions requires upscaling in fluidic-turbulent bioreactors, resulting in membrane shear stress. For the implementation of erythroid cultures in bioreactors, understanding the effects of mechanical stress on terminal EBL differentiation is required. To this end, we investigated the effect of orbital shaking-induced shear stress on differentiating CD49d+CD235low primary human EBL towards enucleated reticulocytes at the molecular, cellular, and functional level. Orbital shaking at the onset of EBL differentiation enhanced cell maturation increasing enucleation percentage compared to static cultures, without cell viability loss. Transcriptome analysis uncovered 505 genes differentially expressed between static and dynamic cultures, with genes involved in lipid and cholesterol biosynthesis upregulated in dynamic conditions. In line with this, cells differentiated in orbital-shakers showed increased cholesterol concentration and osmotic resistance compared to static cultures. HMGCR (3-Hydroxy-3-Methylglutaryl-CoA-Reductase), rate-limiting enzyme of the cholesterol biosynthesis pathway, showed earlier and significantly higher induction during differentiation in dynamic. The severe loss of EBL in dynamic, but not in static conditions, due to HMGCR inhibition confirmed the ability of EBL to adapt to shear stress through modulating of their transcriptional program and upregulation of cholesterol biosynthesis. This work sheds light into specific mechanisms that will assist the successful upscaling of erythroid differentiation in turbulent bioreactors. In addition, as shear-stress on hematopoietic cells is also occurring within the bone marrow niche, these results introduces a potential novel signalling axis that need to be integrated into the known transduction pathways that control erythropoiesis.

cell biology↗

Functional stress responses in Glaucophyta: evidence of ethylene and ABA functions in C. paradoxa

Glaucophytes, an enigmatic group of freshwater algae, occupy a pivotal position within the Archaeplastida, providing insights into the early evolutionary history of plastids and their host cells. These algae possess unique plastids, known as cyanelles, that retain certain ancestral features, enabling a better understanding of the plastid transition from cyanobacteria. In this study, we investigated the role of ethylene, a potent hormone used by land plants to coordinate stress responses, in the glaucophyte alga Cyanophora paradoxa. We demonstrate that C. paradoxa produces gaseous ethylene when supplied with exogenous 1-aminocyclopropane-1-carboxylic acid (ACC), the ethylene precursor in land plants. In addition, we show that cells produce ethylene natively in response to abiotic stress, and that another plant hormone, abscisic acid (ABA), interferes with ethylene synthesis from exogenously supplied ACC, while positively regulating reactive oxygen species (ROS) accumulation. ROS synthesis also occurred following abiotic stress and ACC treatment, possibly acting as a second-messenger in stress responses. A physiological response of C. paradoxa to ACC treatment is growth inhibition. Using transcriptomics, we reveal that ACC treatment induces the upregulation of senescence-associated proteases, consistent with the observation of growth inhibition. This is the first report of hormone usage in a glaucophyte alga, extending our understanding of hormone-mediated stress response coordination into the Glaucophyta, with implications for the evolution of signaling modalities across Archaeplastida.

evolutionary biology↗

Expansion and differentiation of ex vivo cultured erythroblasts in scalable stirred bioreactors

Transfusion of donor-derived red blood cells (RBCs) is the most common form of cell therapy. Production of transfusion-ready cultured RBCs (cRBCs) is a promising replacement for the current fully donor-dependent therapy. However, very large number of cells are required for transfusion. Here we scale-up cRBC production from static cultures to 0.5 L stirred tank bioreactors, and identify the effect of operating conditions on the efficiency of the process. Oxygen requirement of proliferating erythroblasts (0.55-2.01 pg/cell/h) required sparging of air to maintain the dissolved oxygen concentration at the tested setpoint (2.88 mg O2/L). Erythroblasts could be cultured at dissolved oxygen concentrations as low as 0.7 O2 mg/mL without negative impact on proliferation, viability or differentiation dynamics. Stirring speeds of up to 600 rpm supported erythroblast proliferation, while 1800 rpm led to a transient halt in growth and accelerated differentiation followed by a recovery after 5 days of culture. Erythroblasts could also be differentiated in bioreactors, with final enucleation levels and hemoglobin content similar to parallel cultures under static conditions. After defining optimal mixing and aeration strategies, erythroblast proliferation cultures were successfully scaled up to 3 L bioreactors.

bioengineering↗

Sampling artifacts in single-cell genomics cohort studies

Robust protocols and automation now enable large-scale single-cell RNA and ATAC sequencing experiments and their application on biobank and clinical cohorts. However, technical biases introduced during sample acquisition can hinder solid, reproducible results and a systematic benchmarking is required before entering large-scale data production. Here, we report the existence and extent of gene expression and chromatin accessibility artifacts introduced during sampling and identify experimental and computational solutions for their prevention.

genomics↗

A TBX5 dosage-sensitive gene regulatory network for human congenital heart disease

Haploinsufficiency of transcriptional regulators causes human congenital heart disease (CHD). However, underlying CHD gene regulatory network (GRN) imbalances are unknown. Here, we define transcriptional consequences of reduced dosage of the CHD-linked transcription factor, TBX5, in individual cells during cardiomyocyte differentiation from human induced pluripotent stem cells (iPSCs). We discovered highly sensitive dysregulation of TBX5-dependent pathways-- including lineage decisions and genes associated with cardiomyocyte function and CHD genetics--in discrete subpopulations of cardiomyocytes. GRN analysis identified vulnerable nodes enriched for CHD genes, indicating that cardiac network stability is sensitive to TBX5 dosage. A GRN-predicted genetic interaction between Tbx5 and Mef2c was validated in mouse, manifesting as ventricular septation defects. These results demonstrate exquisite sensitivity to TBX5 dosage by diverse transcriptional responses in heterogeneous subsets of iPSC-derived cardiomyocytes. This predicts candidate GRNs for human CHDs, with implications for quantitative transcriptional regulation in disease.

developmental biology↗

A Human Accelerated Region participates in early human forebrain patterning and expansion

The expansion of the mammalian brain is associated with specific developmental processes; however, not much is known about how evolutionary changes participated in the acquisition of human brain traits during early developmental stages. Here we investigated whether enhancers active during the phylotypic stage show human-specific genomic divergence which could contribute to the evolutionary expansion of the forebrain. Notably, we identified an active enhancer containing a human accelerated region (HAR) located in the Chromosome 14q12, a region enriched with neurodevelopmental genes, such as Foxg1, Nkx2.1 and Nova1. Reporter analysis revealed that the human variant is active in the forebrain in transgenic mice and that it has stronger enhancer activity than the mouse or chimpanzee versions. Humanization of the mouse enhancer variant in transgenic mice and in mouse organoids resulted in an expansion of Foxg1 expressing domains in the forebrain early neural progenitors with a bias towards dorsal identities. Overall, our results suggest that human-specific mutations in critical regulatory elements controlling early brain development impact the expansion and patterning of the forebrain.

developmental biology↗

Single-Cell Transcriptomics Unveils Gene Regulatory Network Plasticity

SUMMARYSingle-cell RNA sequencing (scRNA-seq) plays a pivotal role in our understanding of cellular heterogeneity. Current analytical workflows are driven by categorizing principles that consider cells as individual entities and classify them into complex taxonomies. We have devised a conceptually different computational framework based on a holistic view, where single-cell datasets are used to infer global, large-scale regulatory networks. We developed correlation metrics that are specifically tailored to single-cell data, and then generated, validated and interpreted single-cell-derived regulatory networks from organs and perturbed systems, such as diabetes and Alzheimers disease. Using advanced tools from graph theory, we computed an unbiased quantification of a genes biological relevance, and accurately pinpointed key players in organ function and drivers of diseases. Our approach detected multiple latent regulatory changes that are invisible to single-cell workflows based on clustering or differential expression analysis. In summary, we have established the feasibility and value of regulatory network analysis using scRNA-seq datasets, which significantly broadens the biological insights that can be obtained with this leading technology.

genomics↗

matchSCore: Matching Single-Cell Phenotypes Across Tools and Experiments

Single-cell transcriptomics allows the identification of cellular types, subtypes and states through cell clustering. In this process, similar cells are grouped before determining co-expressed marker genes for phenotype inference. The performance of computational tools is directly associated to their marker identification accuracy, but the lack of an optimal solution challenges a systematic method comparison. Moreover, phenotypes from different studies are challenging to integrate, due to varying resolution, methodology and experimental design. In this work we introduce matchSCore (https://github.com/elimereu/matchSCore), an approach to match cell populations fast across tools, experiments and technologies. We compared 14 computational methods and evaluated their accuracy in clustering and gene marker identification in simulated data sets. We further used matchSCore to project cell type identities across mouse and human cell atlas projects. Despite originating from different technologies, cell populations could be matched across data sets, allowing the assignment of clusters to reference maps and their annotation.

bioinformatics↗

bigSCale: An Analytical Framework for Big-Scale Single-Cell Data

Single-cell RNA sequencing significantly deepened our insights into complex tissues and latest techniques are capable processing ten-thousands of cells simultaneously. With bigSCale, we provide an analytical framework being scalable to analyze millions of cells, addressing challenges of future large datasets. Unlike previous methods, bigSCale does not constrain data to fit an a priori-defined distribution and instead uses an accurate numerical model of noise. We evaluated the performance of bigSCale using a biological model of aberrant gene expression in patient derived neuronal progenitor cells and simulated datasets, which underlined its speed and accuracy in differential expression analysis. We further applied bigSCale to analyze 1.3 million cells from the mouse developing forebrain. Herein, we identified rare populations, such as Reelin positive Cajal-Retzius neurons, for which we determined a previously not recognized heterogeneity associated to distinct differentiation stages, spatial organization and cellular function. Together, bigSCale presents a perfect solution to address future challenges of large single-cell datasets.\n\nExtended AbstractSingle-cell RNA sequencing (scRNAseq) significantly deepened our insights into complex tissues by providing high-resolution phenotypes for individual cells. Recent microfluidic-based methods are scalable to ten-thousands of cells, enabling an unbiased sampling and comprehensive characterization without prior knowledge. Increasing cell numbers, however, generates extremely big datasets, which extends processing time and challenges computing resources. Current scRNAseq analysis tools are not designed to analyze datasets larger than from thousands of cells and often lack sensitivity and specificity to identify marker genes for cell populations or experimental conditions. With bigSCale, we provide an analytical framework for the sensitive detection of population markers and differentially expressed genes, being scalable to analyze millions of single cells. Unlike other methods that use simple or mixture probabilistic models with negative binomial, gamma or Poisson distributions to handle the noise and sparsity of scRNAseq data, bigSCale does not constrain the data to fit an a priori-defined distribution. Instead, bigSCale uses large sample sizes to estimate a highly accurate and comprehensive numerical model of noise and gene expression. The framework further includes modules for differential expression (DE) analysis, cell clustering and population marker identification. Moreover, a directed convolution strategy allows processing of extremely large data sets, while preserving the transcript information from individual cells.\n\nWe evaluate the performance of bigSCale using a biological model for reduced or elevated gene expression levels. Specifically, we perform scRNAseq of 1,920 patient derived neuronal progenitor cells from Williams-Beuren and 7q11.23 microduplication syndrome patients, harboring a deletion or duplication of 7q11.23, respectively. The affected region contains 28 genes whose transcriptional levels vary in line with their allele frequency. BigSCale detects expression changes with respect to cells from a healthy donor and outperforms other methods for single-cell DE analysis in sensitivity. Simulated data sets, underline the performance of bigSCale in DE analysis as it is faster and more sensitive and specific than other methods. The probabilistic model of cell-distances within bigSCale is further suitable for unsupervised clustering and the identification of cell types and subpopulations. Using bigSCale, we identify all major cell types of the somatosensory cortex and hippocampus analyzing 3,005 cells from adult mouse brains. Remarkably, we increase the number of cell population specific marker genes 4-6-fold compared to the original analysis and, moreover, define markers of higher order cell types. These include CD90 (Thy1), a neuronal surface receptor, potentially suitable for isolating intact neurons from complex brain samples.\n\nTo test its applicability for large data sets, we apply bigSCale on scRNAseq data from 1.3 million cells derived from the pallium of the mouse developing forebrain (E18, 10x Genomics). Our directed down-sampling strategy accumulates transcript counts from cells with similar transcriptional profiles into index cell transcriptomes, thereby defining cellular clusters with improved resolution. Accordingly, index cell clusters provide a rich resource of marker genes for the main brain cell types and less frequent subpopulations. Our analysis of rare populations includes poorly characterized developmental cell types, such as neuron progenitors from the subventricular zone and neocortical Reelin positive neurons known as Cajal-Retzius (CR) cells. The latter represent a transient population which regulates the laminar formation of the developing neocortex and whose malfunctioning causes major neurodevelopmental disorders like autism or schizophrenia. Most importantly, index cell cluster can be deconvoluted to individual cell level for targeted analysis of populations of interest. Through decomposition of Reelin positive neurons, we determined a previously not recognized heterogeneity among CR cells, which we could associate to distinct differentiation stages as well as spatial and functional differences in the developing mouse brain. Specifically, subtypes of CR cells identified by bigSCale express different compositions of NMDA, AMPA and glycine receptor subunits, pointing to subpopulations with distinct membrane properties. Furthermore, we found Cxcl12, a chemokine secreted by the meninges and regulating the tangential migration of CR cells, to be also expressed in CR cells located in the marginal zone of the neocortex, indicating a self-regulated migration capacity.\n\nTogether, bigSCale presents a perfect solution for the processing and analysis of scRNAseq data from millions of single cells. Its speed and sensitivity makes it suitable to the address future challenges of large single-cell data sets.

genomics↗