bioRxiv Science⌕ Search

Biology subjects

Matheson, J.

Publications and source records attributed to Matheson, J..

5 recordsLinked to original sources

Rare variants drive high variance in human ancestral fitness at mutation-selection-drift balance

It is an open question whether variation in the genetic load of unconditionally deleterious mutations contributes substantially to the variability in human disease. Here, we solve for mutation-selection-drift balance and predict variation in genetic load given a realistic human genome-wide deleterious mutation rate, U, and a distribution of fitness effects (DFE). Empirical estimates of U come from sequence constraint, which fails to count slightly deleterious mutations that nevertheless fix. We use the inferred DFE to correct for this and conclude that total human U>3.8. Two humans typically differ in ancestral fitness by 17-33% given uncertainty in U, or by 6-49% when we consider a broad range of alternative DFEs. Results are similar for other species with larger mean selection coefficients, such as other mammals. Most variation in load comes from rare variants with frequencies below 1%, with a substantial fraction coming from ultra-rare variants below 0.01%. This could help explain why some of the heritability observed in pedigree studies is missing from genome-wide association studies. Accounting for rare and ultra-rare variants, e.g., via variant-effect prediction of unique mutations from whole-genome sequencing rather than via polygenic risk scores, could help identify individuals at high risk of disease. SignificanceMany human mutations mildly disrupt molecular function, e.g., by destabilizing proteins. Having too many of these mutations would have reduced fitness in ancestral human environments and might contribute to disease today. Here, we mathematically derive how much variation in fitness such mutations cause, using estimated human parameter values. Rare variants with larger fitness effects contribute the most. Identifying individuals with high disease risk likely requires methods capable of scoring rare variants.

evolutionary biology↗

Extinction vortices are driven more by a shortage of beneficial mutations than by deleterious mutation accumulation

Habitat loss contributes to extinction risk in multiple ways. Genetically, small populations can face an "extinction vortex" -- a positive feedback loop between declining fitness and declining population size. Two distinct genetic mechanisms can drive a long-term extinction vortex: i) ineffective selection in small populations allows deleterious mutations to fix, driving "mutational meltdown", and ii) smaller populations generate fewer beneficial mutations essential for long-term adaptation, a mechanism we term "mutational drought". To determine their relative importance, we ask whether, for a population near its critical size for persistence, changes in population size have a larger effect on the beneficial vs. deleterious component of fitness flux. In stable environments, we find that mutational drought is nearly as significant as mutational meltdown. Drought is more important than meltdown when populations must also adapt to a changing environment, unless the beneficial mutation rate is extremely high. Linkage disequilibria from background selection under realistically high deleterious mutation rates modestly increase the importance of mutational drought. Long-term conservation efforts should consider adaptive potential, not just deleterious load.

evolutionary biology↗

Id1, Spp1 and Pak3 are biomarkers of Smad4 and TGF-β1 dependency in conditional intestinal adenoma, organoids and colorectal cancer.

TGF-{beta} ligand activation suppresses cell growth yet can paradoxically and potently promote cancer invasion and metastasis depending on downstream pathway mutational context. Here, we evaluated the basis of this observation in conditional murine intestinal adenoma models with and without loss of Mothers against decapentaplegic homolog 4 (Smad4), with the aim of identifying TGF-{beta}-BMP-SMAD4 pathway dependent gene expression biomarkers for translational application. Conditional Lgr5-CreERT2 activation in Apcfl/flSmad4fl/flresulted in adenoma formation with recombined homozygote floxed alleles (Apc{Delta}/{Delta}Smad4{Delta}/{Delta}). The adenoma phenotype was discordant, with a reduced small intestinal adenoma burden yet development of large non-metastatic caecal adenoma with nuclear localisation of phospho-Smad2/3. Derived Apc{Delta}/{Delta}Smad4{Delta}/{Delta} adenoma organoids resisted TGF-{beta}1 dose dependent growth arrest and cell death (IC50 534pM) compared to Apc{Delta}/{Delta}Smad4+/+ (IC 24pM). TGF-{beta}1 (390pM) modified adenoma mRNA expression (bulk RNA-Seq) most significantly for Id1low and Spp1high in Apc{Delta}/{Delta}Smad4{Delta}/{Delta}. Single cell RNAseq of caecal adenoma identified expansion of Lgr5low, Pak3high and Id1low progenitor populations in Apc{Delta}/{Delta}Smad4{Delta}/{Delta}. Of the 76 Smad4 and TGF-{beta}1 dependent genes identified in adenoma organoids, 7 human equivalent genes were also significantly differentially expressed in colorectal cancer, including ID1low, SPP1high and PAK3high that also correlated with poorer survival (TCGA cohorts). Murine conditional models identified Smad4 loss of function mRNA expression biomarkers that require further evaluation as functional classifiers of colorectal cancer subtypes.

cancer biology↗

Extended blood stage sensitivity profiles of Plasmodium vivax to doxycycline and tafenoquine using Plasmodium cynomolgi as a model

Testing Plasmodium vivax antimicrobial sensitivity is limited to ex vivo schizont maturation assays, which precludes determining the IC50s of delayed action antimalarials such as doxycycline. Using Plasmodium cynomolgi as a model for P. vivax, we determined the physiologically significant delayed death effect induced by doxycycline (IC50(96h), 1401 {+/-} 607 nM). As expected, IC50(96 h) to chloroquine (20.4 nM), piperaquine (12.6 {micro}M) and tafenoquine (1424 nM) were not affected by extended exposure.

microbiology↗

A Drug Repurposing Approach Reveals Targetable Epigenetic Pathways in Plasmodium vivax Hypnozoites

Radical cure of Plasmodium vivax malaria must include elimination of quiescent hypnozoite forms in the liver; however, the only FDA-approved treatments are contraindicated in many vulnerable populations. To identify new drugs and drug targets for hypnozoites, we screened the Repurposing, Focused Rescue, and Accelerated Medchem (ReFRAME) library and a collection of epigenetic inhibitors against P. vivax liver stages. From both libraries, we identified inhibitors targeting epigenetics pathways as selectively active against P. vivax and P. cynomolgi hypnozoites. These include DNA methyltransferase (DNMT) inhibitors as well as several inhibitors targeting histone post-translational modifications. Immunofluorescence staining of Plasmodium liver forms showed strong nuclear 5-methylcystosine signal, indicating liver stage parasite DNA is methylated. Using bisulfite sequencing, we mapped genomic DNA methylation in sporozoites, revealing DNA methylation signals in most coding genes. We also demonstrated that methylation level in proximal promoter regions as well as in the first exon of the genes may affect, at least partially, gene expression in P. vivax. The importance of selective inhibitors targeting epigenetic features on hypnozoites was validated using MMV019721, an acetyl-CoA synthetase inhibitor that affects histone acetylation and was previously reported as active against P. falciparum blood stages. In summary, our data indicate that several epigenetic mechanisms are likely modulating hypnozoite formation or persistence and provide an avenue for the discovery and development of improved radical cure antimalarials. One-Sentence SummaryDrug repurposing screens reveal several epigenetic inhibitors as active against P. vivax hypnozoites demonstrating that epigenetic pathways play a central role in hypnozoite quiescence.

cell biology↗