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Biology subjects

Perez-Jurado, L. A.

Publications and source records attributed to Perez-Jurado, L. A..

6 recordsLinked to original sources

A humanized yeast model for studying TRAPP complex mutations; proof-of-concept using variants from an individual with a TRAPPC1-associated neurodevelopmental syndrome

Variants in membrane trafficking proteins are known to cause rare disorders with severe symptoms. The highly conserved transport protein particle (TRAPP) complexes are key membrane trafficking regulators that are also involved in autophagy. Pathogenic genetic variants in specific TRAPP subunits are linked to neurological disorders, muscular dystrophies, and skeletal dysplasias. Characterizing these variants and their phenotypes is important for understanding general and specialized roles of TRAPP subunits as well as for patient diagnosis. Patient-derived cells are not always available, which poses a limitation for the study of these diseases. Therefore, other systems, like the yeast Saccharomyces cerevisiae, can be used to dissect the mechanisms at the intracellular level underlying these disorders. The development of CRISPR/Cas9 technology in yeast has enabled a scar-less editing method that creates an efficient humanized yeast model. In this study, core yeast subunits were humanized by replacing their human orthologs, and TRAPPC1, TRAPPC2, TRAPPC2L, TRAPPC6A, and TRAPPC6B were found to successfully replace their yeast counterparts. This system was used for studying the first reported individual with an autosomal recessive disorder caused by biallelic TRAPPC1 variants, a girl with a severe neurodevelopmental disorder and myopathy. We show that the maternal variant (TRAPPC1 p.(Val121Alafs*3)) is non-functional while the paternal variant (TRAPPC1 p.(His22_Lys24del)) is conditional-lethal and affects secretion and non-selective autophagy in yeast. This parallels defects seen in fibroblasts derived from this individual which also showed membrane trafficking defects and altered Golgi morphology, all of which were rescued in the human system by wild type TRAPPC1. This study suggests that humanized yeast can be an efficient means to study TRAPP subunit variants in the absence of human cells, and can assign significance to variants of unknown significance (VUS). This study lays the foundation for characterizing further TRAPP variants through this system, rapidly contributing to disease diagnosis.

genetics↗

NetActivity enhances transcriptional signals by combining gene expression into robust gene set activity scores through interpretable autoencoders

Grouping gene expression into gene set activity scores (GSAS) provides better biological insights than studying individual genes. However, existing gene set projection methods cannot return representative, robust, and interpretable GSAS. We developed NetActivity, a framework based on a sparsely-connected autoencoder and a three-tier training that yields robust and interpretable GSAS. NetActivity was trained with 1,518 well-known gene sets and all GTEx samples, returning GSAS representative of the original transcriptome and assigning higher importance to more biologically relevant genes. Moreover, NetActivity returns GSAS with a more consistent definition than GSVA and hipathia, state-of-the-art gene set projection methods. Finally, NetActivity enables combining bulk RNA-seq and microarray datasets in a meta-analysis of prostate cancer progression, highlighting gene sets related to cell division. When applied to metastatic prostate cancer, gene sets associated with cancer progression were also altered due to drug resistance, while a classical enrichment analysis identified gene sets irrelevant to the phenotype.

bioinformatics↗

Curcumin combined with verapamil improve cardiovascular phenotype of a Williams-Beuren Syndrome mice model reducing oxidative stress

Williams-Beuren syndrome (WBS) is a rare neurodevelopmental disorder with hallmarks in the cardiovascular manifestations and no well-defined therapeutic strategies. We investigated the progression of cardiovascular phenotype present in CD (complete deletion) mice, a murine model of WBS, after chronic treatment with curcumin and verapamil, both compounds with positive effects on related pathologies. Treatment was administered orally dissolved in drinking water. After measuring "in vivo" systolic blood pressure, aortic and left ventricular myocardium were histological and molecular analysed to determine the effects of treatment and its underlying mechanism in CD mice. We observed upregulated xanthine oxidoreductase (XOR) expression in both aortic and left ventricular myocardium of CD mice. This overexpression is concomitant with increased levels of nitrated proteins as example of byproduct-mediated oxidative stress damage, indicating of XOR-generated oxidative stress impact on the pathophysiology of cardiovascular manifestations in WBS, and suggesting that the inhibition of XOR and/or oxidative stress damage could help to ameliorate the severe cardiovascular injuries presented in WBS.

genetics↗

Verapamil/Curcumin treatment attenuates the behavioral alterations observed in Williams Syndrome mice by regulation of MAPK pathway and Microglia overexpression

Williams-Beuren syndrome (WBS) is a rare neurodevelopmental disorder characterized by a distinctive cognitive phenotype for which there currently are not any effective treatments. We investigated the progression of behavioral deficits present in CD (complete deletion) mice, a rodent model of WBS, after chronic treatment with curcumin, verapamil and a combination of both. These compounds have been proven to have beneficial effects over different cognitive aspects of various murine models and thus, may have neuroprotective effects in WBS. Treatment was administered orally dissolved in drinking water. A set of behavioral tests demonstrated the efficiency of combinatorial treatment. Some histological and molecular analyses were performed to analyze the effects of treatment and its underlying mechanism in CD mice. Behavioral improvement correlates with the molecular recovery of several affected pathways regarding MAPK signaling, in tight relation with the control of synaptic transmission. Moreover, CD mice showed an increased activated microglia density in different brain regions, which was prevented by treatment. Therefore, results show that treatment prevented behavioral deficits by recovering altered gene expression in cortex of CD mice, reducing activated microglia and normalizing Bdnf expression levels. These findings unravel the mechanisms underlying the beneficial effects of this novel treatment on behavioral deficits observed in CD mice, and suggest that the combination of curcumin and verapamil could be a potential candidate to treat the cognitive impairments in WBS patients.

animal behavior and cognition↗

A robust estimation of mosaic loss of chromosome Y from genotype-array-intensity data to improve disease risk associations and transcriptional effects

Accurate protocols and methods to robustly detect the mosaic loss of chromosome Y (mLOY) are needed given its reported role in cancer, several age-related disorders and overall male mortality. Intensity SNP-array data have been used to infer mLOY status and to determine its prominent role in male disease. However, discrepancies of reported findings can be due to the uncertainty and variability of the methods used for mLOY detection and to the differences in the tissue-matrix used. We proposed MADloy, the first publicly available software tool that incorporates previous methods and includes a new robust approach, allowing efficient calling in large studies and comparisons between methods. The new method implemented in MADloy optimizes mLOY calling by correctly modeling the underlying reference population with no-mLOY status and incorporating B-deviation information. We observed improvements in the calling accuracy with respect to previous methods, using experimentally validated samples, and an increment in the statistical power to detect associations with disease and mortality, using simulation studies and real dataset analyses. We applied MADloy to detect the increment of mLOY cellularity in blood on 18 individuals after 3 years, and to confirm that its detection in saliva was sub-optimal (41%). We illustrate the use of MADloy to detect the down-regulation genes in the chromosome Y in kidney and bladder tumors with mLOY, and to perform pathway analyses for the detection of mLOY in blood. MADloy is a new software tool implemented in R for easy and robust calling of mLOY status in men aimed to facilitate its study in large epidemiological studies.

genomics↗

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↗