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Rosen, J. D.

Publications and source records attributed to Rosen, J. D..

7 recordsLinked to original sources

Uniform processing and analysis of IGVF massively parallel reporter assay data with MPRAsnakeflow

As researchers and clinicians seek to identify human genomic alterations relevant to traits and disorders, identifying and aggregating evidence providing mechanistic support for associations between alterations and phenotypes remains challenging. In particular, the study of non-coding genomic variation remains a major challenge due to the lack of accurate functional annotation for activity in a given context and across alleles. Experimental evidence is critical for prioritizing and interpreting functional effects of genetic alterations. Massively Parallel Reporter Assays (MPRAs) have emerged as a powerful high-throughput approach, enabling quantification of regulatory element activity and allelic effects, and systematic dissection of gene regulatory logic and variant effects across different contexts. However, the diversity of MPRA designs, lack of standardized formats, and many potential processing parameters hamper data integration, reproducibility, and meta-analyses across studies. To address these challenges, the Impact of Genomic Variation on Function (IGVF) Consortium established an MPRA focus group to develop community standards, including harmonized file formats, and robust analysis pipelines for a wide range of library types and experimental designs. Here, we present these formats and comprehensive computational tools, MPRAlib and MPRAsnakeflow, for uniform processing from raw sequencing reads to counts, processing and visualization. Using diverse MPRA datasets, we characterize technical variability sources including barcode sequence bias, outlier barcodes, and delivery method (episomal vs. lentiviral). Our results establish best practices for MPRA data generation and analysis, facilitating robust, reproducible research and large-scale integration. The presented tools and standards are publicly available, providing a foundation for future collaborative efforts in regulatory genomics.

genomics↗

Higher eQTL power reveals signals that boost GWAS colocalization

Expression quantitative trait locus (eQTL) studies in human cohorts typically detect at least one regulatory signal per gene, and have been proposed as a way to explain mechanisms of genetic liability for other traits, as discovered in genome-wide association studies (GWAS). In particular, eQTL signals may colocalize with GWAS signals, suggesting gene expression as a possible mediator. However, recent studies have noted colocalization occurs infrequently, even when expression is measured in biologically relevant tissues. Most eQTL studies to date include only hundreds of individuals, and are underpowered to discover distal regulatory signals explaining smaller fractions of gene expression variance. We integrate evidence from recent eQTL studies and demonstrate that limited statistical power due to sample size skews the detection of eQTL signals identified at various signal strengths. We estimate that a sample size of 500 detects <0.1 to 60% of eQTL for a range of signal strengths and that a sample size of 2,000 would detect 36.8% of all eQTL. We show that eQTL signals that can only be discovered in larger studies exhibit characteristics more similar to those of GWAS signals, including greater distance to the regulated gene and higher probability of loss intolerance. Finally, using results from recent eQTL studies and meta-analyses, we observe a large increase in detected colocalizations with GWAS signals compared to previous studies. These findings caution against overinterpreting the absence of colocalization in underpowered studies and provide guidance for designing future eQTL experiments, to improve power and complement perturbation-based approaches in characterizing gene-trait mechanisms.

genetics↗

Leveraging TOPMed Imputation Server and Constructing a Cohort-Specific Imputation Reference Panel to Enhance Genotype Imputation among Cystic Fibrosis Patients

Cystic fibrosis (CF) is a severe genetic disorder that can cause multiple comorbidities affecting the lungs, the pancreas, the luminal digestive system and beyond. In our previous genome-wide association studies (GWAS), we genotyped [~]8,000 CF samples using a mixture of different genotyping platforms. More recently, the Cystic Fibrosis Genome Project (CFGP) performed deep ([~]30x) whole genome sequencing (WGS) of 5,095 samples to better understand the genetic mechanisms underlying clinical heterogeneity among CF patients. For mixtures of GWAS array and WGS data, genotype imputation has proven effective in increasing effective sample size. Therefore, we first performed imputation for the [~]8,000 CF samples with GWAS array genotype using the TOPMed freeze 8 reference panel. Our results demonstrate that TOPMed can provide high-quality imputation for CF patients, boosting genomic coverage from [~]0.3 - 4.2 million genotyped markers to [~]11 - 43 million well-imputed markers, and significantly improving Polygenic Risk Score (PRS) prediction accuracy. Furthermore, we built a CF-specific CFGP reference panel based on WGS data of CF patients. We demonstrate that despite having [~]3% the sample size of TOPMed, our CFGP reference panel can still outperform TOPMed when imputing some CF disease-causing variants, likely due to allele and haplotype differences between CF patients and general populations. We anticipate our imputed data for 4,656 samples without WGS data will benefit our subsequent genetic association studies, and the CFGP reference panel built from CF WGS samples will benefit other investigators studying CF.

genetics↗

Transcriptome-wide association study in UK Biobank Europeans identifies associations with blood cell traits.

Previous genome-wide association studies (GWAS) of hematological traits have identified over 10,000 distinct trait-specific risk loci, but the underlying causal mechanisms at these loci remain incompletely characterized. We performed a transcriptome-wide association study (TWAS) of 29 hematological traits in 399,835 UK Biobank (UKB) participants of European ancestry using gene expression prediction models trained from whole blood RNA-seq data in 922 individuals. We discovered 557 TWAS signals associated with hematological traits distinct from previously discovered GWAS variants, including 10 completely novel gene-trait pairs corresponding to 9 unique genes. Among the 557 associations, 301 were available for replication in a cohort of 141,286 participants of European ancestry from the Million Veteran Program (MVP). Of these 301 associations, 199 replicated at a nominal threshold ( = 0.05) and 108 replicated at a strict Bonferroni adjusted threshold ( = 0.05/301). Using our TWAS results, we systematically assigned 4,261 out of 16,900 previously identified hematological trait GWAS variants to putative target genes. Compared to coloc, our TWAS results show reduced specificity and increased sensitivity to assign variants to target genes.

genetics↗

A large-scale transcriptome-wide association study (TWAS) of ten blood cell phenotypes reveals complexities of TWAS fine-mapping

Hematological measures are important intermediate clinical phenotypes for many acute and chronic diseases. Hematological measures are highly heritable, and although genome-wide association studies (GWAS) have identified thousands of loci containing trait-associated variants, the causal genes underlying these associations are often uncertain. To better understand the underlying genetic regulatory mechanisms, we performed a transcriptome-wide association study (TWAS) using PrediXcan to systematically investigate the association between genetically-predicted gene expression and hematological measures in 54,542 individuals of European ancestry from the Genetic Epidemiology Research on Adult Health and Aging (GERA) cohort. We found 239 significant gene-trait associations with hematological measures. Among this set of 239 associations, we replicated 71 at p < 0.05 with same direction of effect for the blood cell trait in a meta-analysis of TWAS results consisting of up to 35,900 European ancestry individuals from the Womens Health Initiative (WHI), the Atherosclerosis Risk in Communities Study (ARIC), and BioMe Biobank. We further attempted to refine this list of candidate genes by performing conditional analyses, adjusting for individual variants previously associated with these hematological measures, and performed further fine-mapping of TWAS loci. To assist with the interpretation of TWAS findings, we designed an R Shiny application to interactively visualize TWAS results, one genomic locus at a time, by integrating our TWAS results with additional genetic data sources (GWAS, TWAS from other gene expression reference panels, conditional analyses, known GWAS variants, etc.). Our results and R Shiny application highlight frequently overlooked challenges with TWAS and illustrate the complexity of TWAS fine-mapping efforts. Author SummaryTranscriptome-wide association studies (TWAS) have shown great promise in furthering our understanding of the genetic regulatory mechanisms underlying complex trait variation. However, interpreting TWAS results can be incredibly complex, especially in large-scale analyses where hundreds of signals appear throughout the genome, with multiple genes often identified in a single chromosomal region. Our research demonstrates this complexity through real data examples from our analysis of hematological traits, and we provide a useful web application to visualize TWAS results in a broadly approachable format. Together, our results and web application illustrate the importance of interpreting TWAS studies in context and highlight the need to carefully examine results in a region-wide context to draw reasonable conclusions and formulate mechanistic hypotheses.

genetics↗

HPRep: Quantifying reproducibility in HiChIP and PLAC-seq datasets

HiChIP and PLAC-seq are emerging technologies for studying genome-wide long-range chromatin interactions mediated by protein of interest, enabling more sensitive and cost-efficient interrogation of protein-centric chromatin conformation. However, due to the unbalanced read distribution introduced by protein immunoprecipitation, existing reproducibility measures developed for Hi-C data are not appropriate for the analysis of HiChIP and PLAC-seq data. Here, we present HPRep, a stratified and weighted correlation metric derived from normalized contact counts, to quantify reproducibility in HiChIP and PLAC-seq data. We applied HPRep to multiple real datasets and demonstrate that HPRep outperforms existing reproducibility measures developed for Hi-C data. Specifically, we applied HPRep to H3K4me3 PLAC-seq data from mouse embryonic stem cells and mouse brain tissues, as well as H3K27ac HiChIP data from human lymphoblastoid cell line GM12878 and leukemia cell line K562, showing that HPRep can more clearly separate among pseudo-replicates, real replicates, and non-replicates. Furthermore, in an H3K4me3 PLAC-seq dataset consisting of 11 samples from four human brain cell types, HPRep demonstrates expected clustering of data which could not be achieved by existing methods developed for Hi-C data, highlighting the need of a reproducibility metric tailored to HiChIP and PLAC-seq data.

genomics↗

Robust Hi-C chromatin loop maps in human neurogenesis and brain tissues at high-resolution

Genome-wide mapping of chromatin interactions at high resolution remains experimentally and computationally challenging. Here we used a low-input \"easy Hi-C\" (eHi-C) protocol to map the 3D genome architecture in neurogenesis and brain tissues, and also developed an improved Hi-C bias-correction pipeline (HiCorr) enabling better identification of enhancer loops or aggregates at sub-TAD level. We compared ultra-deep 3D genome maps from 10 human tissue- or cell types, with a focus on stem cells and neural development. We found several large loci in skin-derived human iPSC lines showing recurrent 3D compartmental memory of somatic heterochromatin. Chromatin loop interactions, but not genome compartments, are hallmarks of neural differentiation. Interestingly, we observed many cell type- or differentiation-specific enhancer aggregates spanning large neighborhoods, supporting a phase-separation mechanism that stabilizes enhancer contacts during development. Finally, we demonstrated that chromatin loop outperforms eQTL in explaining neurological GWAS results, revealing a unique value of high-resolution 3D genome maps in elucidating the disease etiology.\n\nHighlightsO_LILow input \"easy Hi-C\" protocol compatible with 50-100K cells\nC_LIO_LIImproved Hi-C bias correction allows direct observation and accurate identification of sub-TAD chromatin loops and enhancer aggregates\nC_LIO_LIRecurrent architectural memory of somatic heterochromatin at compartment level in skin-derived hiPSCs\nC_LIO_LIChromatin loop, but not genome compartment, marks neural differentiation\nC_LIO_LIChromatin loop outperforms eQTL in defining brain GWAS target genes\nC_LI

genomics↗