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Aguirre, M.

Publications and source records attributed to Aguirre, M..

14 recordsLinked to original sources

Systematic assessment of machine learning-based variant annotation methods for rare variant association testing

Machine learning-based annotation methods are increasingly used to assess the pathogenicity of genetic variants, but their performance at prioritizing variants for gene-level association testing remains poorly characterized. Here, we systematically benchmark five annotation methods -- CADD v1.6, CADD v1.7, AlphaMissense, ESM-1b, and GPN-MSA -- using four primary gene-based tests and six annotation-level aggregation tests across 14 quantitative traits measured in up to 350,377 UK Biobank participants. Using a novel framework based on Wasserstein dis-tances, we quantify how annotation choice affects test calibration and power. Tests using CADD annotations achieve the highest signal separation, while tests using AlphaMissense annotations exhibit systematically lower calibration. All combinations of methods produced significant re-sults that were enriched (1.8-5.8-fold) for loss-of-function intolerant genes, though tests using GPN-MSA annotations displayed the highest such enrichment. Replication across symmetric phenotypes and loss-of-function burden tests was generally similar across methods. Our anal-ysis provides practical guidance for annotation method selection in rare variant studies and establishes a distributional framework for calibration assessment.

bioinformatics↗

Regulatory network topology and the genetic architecture of gene expression

In human populations, most of the genetic variance in gene expression can be attributed to trans-acting expression quantitative trait loci (eQTLs) spread across the genome. However, in practice it is difficult to discover these eQTLs, and their cumulative effects on gene expression and complex traits are yet to be fully understood. Here, we assess how properties of the genetic architecture of gene expression constrain the space of plausible gene regulatory networks. We describe a structured causal model of gene expression regulation and consider how it interacts with biologically relevant properties of the gene regulatory network to alter the genomic distribution of expression heritability. Under our model, we find that the genetic architecture of gene expression is shaped in large part by local network motifs and by hub regulators that shorten paths through the network and act as key sources of trans-acting variance. Further, simulated networks with an enrichment of motifs and hub regulators best recapitulate the distribution of cis and trans heritability of gene expression as measured in a recent twin study. Taken together, our results suggest that the architecture of gene expression is sparser and more pleiotropic across genes than would be suggested by naive models of regulatory networks, which has important implications for future studies of complex traits.

genetics↗

Gene regulatory network structure informs the distribution of perturbation effects

Gene regulatory networks (GRNs) govern many core developmental and biological processes underlying human complex traits. Even with broad-scale efforts to characterize the effects of molecular perturbations and interpret gene coexpression, it remains challenging to infer the architecture of gene regulation in a precise and efficient manner. Key properties of GRNs, like hierarchical structure, modular organization, and sparsity, provide both challenges and opportunities for this objective. Here, we seek to better understand properties of GRNs using a new approach to simulate their structure and model their function. We produce realistic network structures with a novel generating algorithm based on insights from small-world network theory, and we model gene expression regulation using stochastic differential equations formulated to accommodate modeling molecular perturbations. With these tools, we systematically describe the effects of gene knockouts within and across GRNs, finding a subset of networks that recapitulate features of a recent genome-scale perturbation study. With deeper analysis of these exemplar networks, we consider future avenues to map the architecture of gene expression regulation using data from cells in perturbed and unperturbed states, finding that while perturbation data are critical to discover specific regulatory interactions, data from unperturbed cells may be sufficient to reveal regulatory programs.

genetics↗

Transcriptomics and chromatin accessibility in multiple African population samples

Mapping the functional human genome and impact of genetic variants is often limited to European-descendent population samples. To aid in overcoming this limitation, we measured gene expression using RNA sequencing in lymphoblastoid cell lines (LCLs) from 599 individuals from six African populations to identify novel transcripts including those not represented in the hg38 reference genome. We used whole genomes from the 1000 Genomes Project and 164 Maasai individuals to identify 8,881 expression and 6,949 splicing quantitative trait loci (eQTLs/sQTLs), and 2,611 structural variants associated with gene expression (SV-eQTLs). We further profiled chromatin accessibility using ATAC-Seq in a subset of 100 representative individuals, to identity chromatin accessibility quantitative trait loci (caQTLs) and allele-specific chromatin accessibility, and provide predictions for the functional effect of 78.9 million variants on chromatin accessibility. Using this map of eQTLs and caQTLs we fine-mapped GWAS signals for a range of complex diseases. Combined, this work expands global functional genomic data to identify novel transcripts, functional elements and variants, understand population genetic history of molecular quantitative trait loci, and further resolve the genetic basis of multiple human traits and disease.

genomics↗

Learning epistatic polygenic phenotypes with Boolean interactions

Detecting epistatic drivers of human phenotypes is a considerable challenge. Traditional approaches use regression to sequentially test multiplicative interaction terms involving pairs of genetic variants. For higher-order interactions and genome-wide large-scale data, this strategy is computationally intractable. Moreover, multiplicative terms used in regression modeling may not capture the form of biological interactions. Building on the Predictability, Computability, Stability (PCS) framework, we introduce the epiTree pipeline to extract higher-order interactions from genomic data using tree-based models. The epiTree pipeline first selects a set of variants derived from tissue-specific estimates of gene expression. Next, it uses iterative random forests (iRF) to search training data for candidate Boolean interactions (pairwise and higher-order). We derive significance tests for interactions, based on a stabilized likelihood ratio test, by simulating Boolean tree-structured null (no epistasis) and alternative (epistasis) distributions on hold-out test data. Finally, our pipeline computes PCS epistasis p-values that probabilisticly quantify improvement in prediction accuracy via bootstrap sampling on the test set. We validate the epiTree pipeline in two case studies using data from the UK Biobank: predicting red hair and multiple sclerosis (MS). In the case of predicting red hair, epiTree recovers known epistatic interactions surrounding MC1R and novel interactions, representing non-linearities not captured by logistic regression models. In the case of predicting MS, a more complex phenotype than red hair, epiTree rankings prioritize novel interactions surrounding HLA-DRB1, a variant previously associated with MS in several populations. Taken together, these results highlight the potential for epiTree rankings to help reduce the design space for follow up experiments.

bioinformatics↗

CRIMSON: An Open-Source Software Framework for Cardiovascular Integrated Modelling and Simulation

In this work, we describe the CRIMSON (CardiovasculaR Integrated Modelling and SimulatiON) software environment. CRIMSON provides a powerful, customizable and user-friendly system for performing three-dimensional and reduced-order computational haemodynamics studies via a pipeline which involves: 1) segmenting vascular structures from medical images; 2) constructing analytic arterial and venous geometric models; 3) performing finite element mesh generation; 4) designing, and 5) applying boundary conditions; 6) running incompressible Navier-Stokes simulations of blood flow with fluid-structure interaction capabilities; and 7) post-processing and visualizing the results, including velocity, pressure and wall shear stress fields. A key aim of CRIMSON is to create a software environment that makes powerful computational haemodynamics tools accessible to a wide audience, including clinicians and students, both within our research laboratories and throughout the community. The overall philosophy is to leverage best-in-class open source standards for medical image processing, parallel flow computation, geometric solid modelling, data assimilation, and mesh generation. It is actively used by researchers in Europe, North and South America, Asia, and Australia. It has been applied to numerous clinical problems; we illustrate applications of CRIMSON to real-world problems using examples ranging from pre-operative surgical planning to medical device design optimization. CRIMSON binaries for Microsoft Windows 10, documentation and example input files are freely available for download from www.crimson.software, and the source code with compilation instructions is available on GitHub https://github.com/carthurs/CRIMSONFlowsolver (CRIMSON Flowsolver) under the GPL v3.0 license, and https://github.com/carthurs/CRIMSONGUI (CRIMSON GUI), under the AGPL v3.0 license. Support is available on the CRIMSON Google Groups forum, located at https://groups.google.com/forum/#!forum/crimson-users.

bioengineering↗

Polygenic risk modeling with latent trait-related genetic components

Polygenic risk models have led to significant advances in understanding complex diseases and their clinical presentation. While models like polygenic risk scores (PRS) can effectively predict outcomes, they do not generally account for disease subtypes or pathways which underlie within-trait diversity. Here, we introduce a latent factor model of genetic risk based on components from Decomposition of Genetic Associations (DeGAs), which we call the DeGAs polygenic risk score (dPRS). We compute DeGAs using genetic associations for 977 traits in the UK Biobank and find that dPRS performs comparably to standard PRS while offering greater interpretability. We show how to decompose an individuals genetic risk for a trait across DeGAs components, highlighting specific results for body mass index (BMI), myocardial infarction (heart attack), and gout in 337,151 white British individuals, with replication in a further set of 25,486 non-British white individuals from the Biobank. We find that BMI polygenic risk factorizes into components relating to fat-free mass, fat mass, and overall health indicators like physical activity measures. Most individuals with high dPRS for BMI have strong contributions from both a fat mass component and a fat-free mass component, whereas a few outlier individuals have strong contributions from only one of the two components. Overall, our method enables fine-scale interpretation of the drivers of genetic risk for complex traits.

genomics↗

Assessing digital phenotyping to enhance genetic studies of human diseases

Population-scale biobanks that combine genetic data and high-dimensional phenotyping for a large number of participants provide an exciting opportunity to perform genome-wide association studies (GWAS) to identify genetic variants associated with diverse quantitative traits and diseases. A major challenge for GWAS in population biobanks is ascertaining disease cases from heterogeneous data sources such as hospital records, digital questionnaire responses, or interviews. In this study, we use genetic parameters including genetic correlation to evaluate whether GWAS performed using cases in the UK Biobank ascertained from hospital records, questionnaire responses, and family history of diseases implicate similar disease genetics across a range of effect sizes. We find that hospital record and questionnaire GWAS largely identify similar genetic effects for many complex phenotypes and that combining together both phenotyping methods improves power to detect genetic associations. We also show that family GWAS using cases ascertained on family history of disease agrees with combined hospital record/questionnaire GWAS and that family history GWAS has better power to detect genetic associations for some phenotypes. Overall, this work demonstrates that digital phenotyping and unstructured phenotype data can be combined with structured data such as hospital records to identify cases for GWAS in biobanks and improve the ability of such studies to identify genetic associations.

genetics↗

A Fast and Flexible Algorithm for Solving the Lasso in Large-scale and Ultrahigh-dimensional Problems

The UK Biobank (Bycroft et al., 2018) is a very large, prospective population-based cohort study across the United Kingdom. It provides unprecedented opportunities for researchers to investigate the relationship between genotypic information and phenotypes of interest. Multiple regression methods, compared with GWAS, have already been showed to greatly improve the prediction performance for a variety of phenotypes. In the high-dimensional settings, the lasso (Tibshirani, 1996), since its first proposal in statistics, has been proved to be an effective method for simultaneous variable selection and estimation. However, the large scale and ultrahigh dimension seen in the UK Biobank pose new challenges for applying the lasso method, as many existing algorithms and their implementations are not scalable to large applications. In this paper, we propose a computational framework called batch screening iterative lasso (BASIL) that can take advantage of any existing lasso solver and easily build a scalable solution for very large data, including those that are larger than the memory size. We introduce snpnet, an R package that implements the proposed algorithm on top of glmnet (Friedman et al., 2010a) and optimizes for single nucleotide polymorphism (SNP) datasets. It currently supports{ell} 1-penalized linear model, logistic regression, Cox model, and also extends to the elastic net with{ell} 1/{ell}2 penalty. We demonstrate results on the UK Biobank dataset, where we achieve superior predictive performance on quantitative and qualitative traits including height, body mass index, asthma and high cholesterol. Author SummaryWith the advent and evolution of large-scale and comprehensive biobanks, there come up unprecedented opportunities for researchers to further uncover the complex landscape of human genetics. One major direction that attracts long-standing interest is the investigation of the relationships between genotypes and phenotypes. This includes but doesnt limit to the identification of genotypes that are significantly associated with the phenotypes, and the prediction of phenotypic values based on the genotypic information. Genome-wide association studies (GWAS) is a very powerful and widely used framework for the former task, having produced a number of very impactful discoveries. However, when it comes to the latter, its performance is fairly limited by the univariate nature. To address this, multiple regression methods have been suggested to fill in the gap. That said, challenges emerge as the dimension and the size of datasets both become large nowadays. In this paper, we present a novel computational framework that enables us to solve efficiently the entire lasso or elastic-net solution path on large-scale and ultrahigh-dimensional data, and therefore make simultaneous variable selection and prediction. Our approach can build on any existing lasso solver for small or moderate-sized problems, scale it up to a big-data solution, and incorporate other extensions easily. We provide a package snpnet that extends the glmnet package in R and optimizes for large phenotype-genotype data. On the UK Biobank, we observe improved prediction performance on height, body mass index (BMI), asthma and high cholesterol by the lasso over other univariate and multiple regression methods. That said, the scope of our approach goes beyond genetic studies. It can be applied to general sparse regression problems and build scalable solution for a variety of distribution families based on existing solvers.

bioinformatics↗

Phenome-wide burden of copy number variation in UK Biobank

Copy number variations (CNV) represent a significant proportion of the genetic differences between individuals and many CNVs associate causally with syndromic disease and clinical outcomes. Here, we characterize the landscape of copy number variation and their phenome-wide effects in a sample of 472,228 array-genotyped individuals from the UK Biobank. In addition to population-level selection effects against genic loci conferring high-mortality, we describe genetic burden from syndromic and previously uncharacterized CNV loci across nearly 2,000 quantitative and dichotomous traits, with separate analyses for common and rare classes of variation. Specifically, we highlight the effects of CNVs at two well-known syndromic loci 16p11.2 and 22q11.2, as well as novel associations at 9p23, in the context of acute coronary artery disease and high body mass index. Our data constitute a deeply contextualized portrait of population-wide burden of copy number variation, as well as a series of known and novel dosage-mediated genic associations across the medical phenome.

genomics↗

Non-CG methylation and multiple epigenetic layers associate child abuse with immune and small GTPase dysregulation

Early-life adversity (ELA) is a major predictor of psychopathology, and is thought to increase lifetime risk by epigenetically regulating the genome. Here, focusing on the lateral amygdala, a major brain site for emotional homeostasis, we described molecular cross-talk among multiple epigenetic mechanisms, including 6 histone marks, DNA methylation and the transcriptome, in subjects with a history of ELA and controls. We first uncovered, in the healthy brain, previously unknown interactions among epigenetic layers, in particular related to non-CG methylation in the CAC context. We then showed that ELA associates with methylomic changes that are as frequent in the CAC as in the canonical CG context, while these two forms of plasticity occur in sharply distinct genomic regions, features, and chromatin states. Combining these multiple data indicated that immune-related and small GTPase signaling pathways are most consistently impaired in the amygdala of ELA individuals. Overall, this work provides new insights into epigenetic brain regulation as a function of early-life experience.

neuroscience↗

Components of genetic associations across 2,138 phenotypes in the UK Biobank highlight novel adipocyte biology

To characterize latent components of genetic associations, we applied truncated singular value decomposition (DeGAs) to matrices of summary statistics derived from genome-wide association analyses across 2,138 phenotypes measured in 337,199 White British individuals in the UK Biobank study. We systematically identified key components of genetic associations and the contributions of variants, genes, and phenotypes to each component. As an illustration of the utility of the approach to inform downstream experiments, we report putative loss of function variants, rs114285050 (GPR151) and rs150090666 (PDE3B), that substantially contribute to obesity-related traits, and experimentally demonstrate the role of these genes in adipocyte biology. Our approach to dissect components of genetic associations across human phenotypes will accelerate biomedical hypothesis generation by providing insights on previously unexplored latent structures.

genetics↗

Global Biobank Engine: enabling genotype-phenotype browsing for biobank summary statistics

Large biobanks linking phenotype to genotype have led to an explosion of genetic association studies across a wide range of phenotypes. Sharing the knowledge generated by these resources with the scientific community remains a challenge due to patient privacy and the vast amount of data. Here we present Global Biobank Engine (GBE), a web-based tool that enables the exploration of the relationship between genotype and phenotype in large biobank cohorts, such as the UK Biobank. GBE supports browsing for results from genome-wide association studies, phenome-wide association studies, gene-based tests, and genetic correlation between phenotypes. We envision GBE as a platform that facilitates the dissemination of summary statistics from biobanks to the scientific and clinical communities. GBE currently hosts data from the UK Biobank and can be found freely available at biobankengine.stanford.edu.

bioinformatics↗

Bayesian model comparison for rare variant association studies of multiple phenotypes

Whole genome sequencing studies applied to large populations or biobanks with extensive phenotyping raise new analytic challenges. The need to consider many variants at a locus or group of genes simultaneously and the potential to study many correlated phenotypes with shared genetic architecture provide opportunities for discovery and inference that are not addressed by the traditional one variant, one phenotype association study. Here, we introduce a Bayesian model comparison approach that we refer to as MRP (Multiple Rare-variants and Phenotypes) for rare-variant association studies that considers correlation, scale, and direction of genetic effects across a group of genetic variants, phenotypes, and studies. The approach requires only summary statistic data. To demonstrate the efficacy of MRP, we apply our method to exome sequencing data (N = 184,698) across 2,019 traits from the UK Biobank, aggregating signals in genes. MRP demonstrates an ability to recover previously-verified signals such as associations between PCSK9 and LDL cholesterol levels. We additionally find MRP effective in conducting meta-analyses in exome data. Notable non-biomarker findings include associations between MC1R and red hair color and skin color, IL17RA and monocyte count, IQGAP2 and mean platelet volume, and JAK2 and platelet count and crit (mass). Finally, we apply MRP in a multi-phenotype setting; after clustering the 35 biomarker phenotypes based on genetic correlation estimates into four clusters, we find that joint analysis of these phenotypes results in substantial power gains for gene-trait associations, such as in TNFRSF13B in one of the clusters containing diabetes and lipid-related traits. Overall, we show that the MRP model comparison approach is able to improve upon useful features from widely-used meta-analysis approaches for rare variant association analyses and prioritize protective modifiers of disease risk.

genetics↗