bioRxiv Science⌕ Search

Biology subjects

Zhao, Y.

Publications and source records attributed to Zhao, Y..

At least 289 records · Page 16Linked to original sources

Assemblatron: An Automated Workflow for High-Throughput Assembly of Big-DNA Libraries

The ability to synthesize 50-kb+ DNA molecules has tremendous potential in the fields of genome engineering, metabolic engineering, and synthetic regulatory genomics. Despite tremendous achievements in these fields, such as the completion of the first synthetic eukaryotic genome, assembling custom big DNAs remains slow, expensive, and laborious. In this work, we present a set of improvements to yeast-based DNA assembly methods that enable medium-to high-throughput big DNA experiments. In particular, we i) developed two easy-to-use vector systems: Jack In the Box (JIB), which deploys a split marker and split centromere strategy to reduce background, and Selection of URS Recombinational Excision (SURE) which exploits URS silencer removal from the LEU2 marker. These strategies sharply decrease the number of colonies containing an empty vector, greatly reducing the amount of screening required to find correct clones. ii) We improved yeast transformation efficiency, increasing the likelihood that all required DNA segments are co-transformed into the same cell. iii) We developed an automation pipeline for segment mixing through single-colony isolation. We also established three phenotype-driven assays that directly and rapidly report the frequency of correctly assembled large DNA molecules. Harnessing these improvements, we show a high success rate in assembling diverse big DNA libraries by combinatorial assembly and oligo-guided architecture in yeast (OLIGARCHY), a novel method that allows cost-effective reuse of segments for distinct assemblies with reduced effort and cost. Using OLIGARCHY, we designed 96 structural variants of a [~]73kb construct, and a single person was able to correctly assemble 90 of these in 7 days, for a total of 7 Mb of assembled DNA. Finally, we used the SURE vector to greatly improve existing methods to turn oligonucleotides into gene-sized double-stranded DNA segments, efficiently turning 80 overlapping 60-mers into [~]3-kb DNAs directly, without PCR amplification.

genetics↗

Global analysis of membrane protein S-acylation in the model plant Arabidopsis thaliana

Protein S-acylation is the addition of fatty acids to the cysteine residues in a protein, catalyzed by protein S-acyltransferases (PATs). Despite extensive research on protein S-acylation in animals, our understanding of this process in plants remains limited. In this study, we sought to characterize the S-acylproteome of membrane proteins in Arabidopsis and identify potential substrates for two important plant immunity-related PATs (PAT5 and PAT9). To achieve this, S-acylated membrane proteins were first enriched via our optimized acyl-biotinyl exchange strategies at both the protein-level and peptide-level. The enriched samples were then analyzed by label-free quantitative liquid chromatography-mass spectrometry. The results from the two enrichment methods demonstrated that they were complementary in identifying S-acylated proteins and S-acylation sites. Using these methods, over 2500 S-acylation sites in more than 2000 putative S-acylated proteins were identified. Proteins involved in vesicle trafficking, plant phosphorylation, immune responses, and signal transduction pathways were significantly enriched. Additionally, certain amino acid patterns surrounding the S-acylation sites were identified. Comparisons of the S-acylproteomes between the wild type and the PAT5 and PAT9 mutants revealed over 100 potential substrates for both S-acyltransferases. The high quality of our data was supported by the significant overlap with the previously reported data and successful experimental verification of selected candidate proteins. Overall, our study revealed a well-represented S-acylproteome for Arabidopsis (especially its membrane proteins) and identified potential substrates for PAT5 and PAT9. These findings will facilitate the functional characterization of S-acylated proteins in plants.

plant biology↗

Modeling Post-Gastrula Development via Bidirectional Pluripotent Stem Cells

The absence of stem cells capable of efficiently generating both trophoblast and epiblast lineages has hindered precise recapitulation of embryonic development. Through high-content chemical screening, we established an AL medium to generate mouse and human bidirectional pluripotent stem cells (BPSCs) characterized by concurrent OCT4/CDX2 expression. Mouse BPSCs demonstrated high plastic differentiation into trophoblast, epiblast and primitive endoderm lineages in vitro within 48 hours without exogenous induction factors and efficiently contributed to the embryo and extraembryonic tissues in vivo. Mechanistically, hyperactivation of the Wnt signaling pathway breaks the early lineage differentiation barrier by initiating a Lef1-dependent bypass. Remarkably, BPSCs can efficiently generated E8.5 embryoids that completed gastrulation and displayed advanced features such as brain development, a closed neural tube, a beating heart, somite formation, and primordial germ cells. These findings highlight BPSCs as a powerful tool for investigating early lineage specification and post-gastrulation embryonic development, with potential applications across multiple species.

developmental biology↗

EVscope: A Comprehensive Bioinformatics Pipeline for Accurate and Robust Analysis of Total RNA Sequencing from Extracellular Vesicles

MotivationExtracellular vesicle (EV) RNA sequencing has emerged as a powerful approach for studying RNA biomarkers and intercellular communication. Nevertheless, the extremely low abundance, fragmented nature and ubiquitous tissue origin of EV RNAs, alongside potential contamination from co-isolated materials, such as free DNA and bacterial RNA, pose substantial analytical challenges. These complexities highlight a pressing need for a standardized, computational workflow that ensures robust quality control and EV RNA characterization. ResultsHere, we present EVscope, an open-source bioinformatics pipeline designed specifically for processing EV RNA-seq datasets. EVscope employs an optimized genome-wide expectation-maximization (EM) algorithm that significantly improves multi-mapping read assignment at single-base resolution by effectively leveraging alignment scores (AS) and local read coverage, specifically tailored for fragmented and low-abundance EV RNAs. Notably, EVscope uniquely generates EM-based BigWig files for downstream analysis, a capability currently unavailable in existing EM-based BigWig quantification tools. The pipeline systematically integrates 27 major steps, including quality control, analysis of library structure, contamination assessment, read alignment, read strandedness detection, UMI-based deduplication, RNA quantification, genomic DNA (gDNA) contamination correction, cellular and tissue source inference and visualization with a comprehensive HTML report. EVscope incorporates a comprehensive, updated annotation covering 19 distinct RNA biotypes, encompassing protein-coding genes, lncRNAs, miRNAs, piRNAs, retrotransposons (LINEs, SINEs, ERVs), and additional non-coding RNAs (tRNAs, rRNAs, snoRNAs). Furthermore, it leverages two highly balanced circRNA detection algorithms for robust circular RNA identification. Notably, a downstream module enables the inference of the tissue/cellular origins of EV RNAs using bulk and single-cell RNA-seq reference datasets. EVscope is implemented as a convenient, single-command Bash pipeline leveraging Conda-managed standard software packages and custom scripts, ensuring reproducibility and straightforward deployment. Availability and implementationCode, documentation, and tutorials are available at GitHub (https://github.com/TheDongLab/EVscope) and archived on Zenodo (https://zenodo.org/records/15577789).

bioinformatics↗

PEG-Free Tunable Poly(2-Oxazoline) Lipids Modulate LNP Biodistribution and Expression In Vivo after Intramuscular Administration

Over the past decade, lipid nanoparticles (LNPs) have emerged as a transformative delivery platform, particularly in the field of mRNA vaccines, by enabling the stabilization and efficient intracellular delivery of nucleic acids. Importantly, the FDA approved LNPs used in the Pfizer-BioNTech and Moderna COVID-19 vaccines, rely on polyethylene glycol (PEG) to stabilize the nanoparticle in vivo. However, recent studies revealed that anti-PEG antibodies are ubiquitous in the population and are known to be a major cause of anaphylaxis and reduced therapeutic efficacy by accelerating blood clearance of PEGylated products. In this study, we report the development of novel poly(2-oxazoline) (POx) "stealth" lipids as PEG alternatives for LNP formulation. POx polymers are known to be poorly immunogenic and mitigate accelerated blood clearance. Upon varying polymer hydrophilicity and molecular weight, we screened transfection efficiency in multiple cell lines and evaluated top candidates in vivo by intramuscular administration. Our findings demonstrate that POx-lipids incorporating shorter hydrophilic methyl- and ethyl-oxazoline POx chains enhance transfection both in vitro and in vivo, while concurrently reducing liver accumulation and improving dendritic cell uptake - key features for effective vaccine delivery. These POx-lipids boost expression in muscle tissue by up to threefold, and greatly increase accumulation in extrahepatic tissues, including lymph nodes and spleen - critical sites for immune priming in the in the context of vaccination. Altogether, this work marks a notable advancement toward replacing PEG in LNP-based therapies and provides a clear path to optimizing POx-lipids for targeted vaccine and therapeutic applications.

bioengineering↗

New alleles of Arabidopsis BIK1 reinforce its predominant role in pattern-triggered immunity and caution interpretations of other reported functions

The receptor-like cytoplasmic kinase BIK1 and its close homolog PBL1 have been widely recognized as central components of plant immunity. However, most genetic studies of BIK1 and PBL1 functions were carried out with single T-DNA insertional mutant alleles. Some phenotypes observed in these mutants, e.g. autoimmunity, have been difficult to reconcile with the proposed role of BIK1 and PBL1 in pattern-triggered immunity. In this study, we generated multiple new alleles of bik1 and pbl1 by CRISPR-Cas9-based gene editing and systematically analyzed these mutants alongside existing T-DNA insertional lines. These analyses reinforced the central role of BIK1 and PBL1 in pattern-triggered immunity mediated by both receptor kinases and receptor-like proteins. At the same time, however, we revealed several pleiotropic phenotypes associated with T-DNA insertions that are not necessarily linked to loss of BIK1 or PBL1 function. Further analyses of newly generated bik1 pbl1 double mutants uncovered an even greater contribution of these kinases to immune signaling and disease resistance than previously appreciated. These findings clarify longstanding ambiguities surrounding BIK1 and PBL1 functions.

plant biology↗

Global profiling of the proteome and acetylome in mice with abdominal aortic aneurysms

BackgroundAbdominal Aortic Aneurysm (AAA) is a life-threatening vascular condition characterized by a progressive dilation of the abdominal aorta and a high risk of rupture. Currently, no effective pharmacological therapies are available, and treatment relies mainly on surgical intervention. The lack of drug targets is largely due to limited understanding of the molecular mechanisms underlying AAA development. This study aims to identify potential therapeutic targets by performing comprehensive proteomic and acetylomic analyses in a mouse model of AAA. MethodsWe conducted global proteomic and acetylomic profiling on abdominal aortic tissues collected from an AAA mouse model. A total of 7,858 proteins and 1,790 acetylated proteins encompassing 4,581 acetylation sites were quantified. Bioinformatics analyses were performed to investigate the regulatory networks and biological pathways associated with these proteins and post-translational modifications. ResultsIntegrated analysis revealed that histone-related proteins were significantly enriched in pathways co-regulated by the proteome and acetylome. These pathways are influenced by histone acetyltransferases and deacetylases, implicating histone acetylation in AAA-related inflammatory and immune processes. Notably, the deacetylases Sirt2 and Sirt5 were identified as potential regulators of neutrophil extracellular trap (NET) formation through suppression of histone acetylation. ConclusionsThis study provides the first comprehensive proteomic and acetylomic dataset for abdominal aortic tissues in a mouse model of AAA, substantially expanding the mouse acetylation database. The results highlight the central role of histone modifications in immune dysregulation and inflammation, and propose Sirt2 and Sirt5 as candidate therapeutic targets that may inhibit NET formation and slow AAA progression. These findings suggest new avenues for drug development targeting the sirtuin family, although further experimental validation and cross-species studies are needed to confirm translational relevance. Graphic Abstract O_FIG O_LINKSMALLFIG WIDTH=200 HEIGHT=145 SRC="FIGDIR/small/657536v1_ufig1.gif" ALT="Figure 1"> View larger version (42K): org.highwire.dtl.DTLVardef@1c397a3org.highwire.dtl.DTLVardef@188180org.highwire.dtl.DTLVardef@de1a59org.highwire.dtl.DTLVardef@193e740_HPS_FORMAT_FIGEXP M_FIG C_FIG

bioinformatics↗

Gene therapies alleviate absence epilepsy associated with Scn2a deficiency in DBA/2J mice

Mutations in the voltage-gated sodium channel gene SCN2A, which encodes the NaV1.2 channel, cause severe epileptic seizures. Patients with SCN2A loss-of-function (LoF) mutations, such as protein-truncating mutations, often experience later-onset and drug-resistant epilepsy, highlighting an urgent unmet clinical need for new therapies. We previously developed a gene-trap Scn2a (Scn2agt/gt) mouse model with a global NaV1.2 reduction in the widely used C57BL/6N (B6) strain. Although these mice display multiple behavioral abnormalities, EEG recordings indicated only mild epileptiform discharges, possibly attributable to the seizure-resistant characteristics associated with the B6 strain. To enhance the epileptic phenotype, we derived congenic Scn2agt/gt mice in the seizure-susceptible DBA/2J (D2J) strain. Notably, we found that these mice exhibit prominent spontaneous absence seizures, marked by both short and long spike-wave discharges (SWDs). Restoring NaV1.2 expression in adult mice substantially reduced their SWDs, suggesting the possibility of SCN2A gene replacement therapy during adulthood. RNA sequencing revealed significant alterations in gene expression in the Scn2agt/gt mice, in particular a broad downregulation of voltage-gated potassium channel (KV) genes, including KV1.1. The reduction of KV1.1 expression was further validated in human cerebral organoids with SCN2A deficiency, highlighting KV1.1 as a promising therapeutic target for refractory seizures associated with SCN2A dysfunction. Importantly, delivery of exogenous human KV1.1 expression via adeno-associated virus (AAV) in D2J Scn2agt/gt mice substantially reduced absence seizures. Together, these findings underscore the influence of mouse strain on seizure severity and highlight the potential of targeted gene therapies for treating SCN2A deficiency-related epilepsies. Graphical abstract O_FIG O_LINKSMALLFIG WIDTH=200 HEIGHT=91 SRC="FIGDIR/small/657652v1_ufig1.gif" ALT="Figure 1"> View larger version (18K): org.highwire.dtl.DTLVardef@35374eorg.highwire.dtl.DTLVardef@1c22197org.highwire.dtl.DTLVardef@2b4582org.highwire.dtl.DTLVardef@e06e68_HPS_FORMAT_FIGEXP M_FIG C_FIG In briefScn2a deficiency leads to absence seizures in D2J mice and neuronal hyperexcitability with compensatory KV reduction; restoring NaV1.2 or introducing human KV1.1 reduces seizure burden. HighlightsO_LIScn2a deficiency induces robust absence seizures in the DBA/2J but not the C57BL/6N strain. C_LIO_LICortical neurons in adult DBA/2J mice with Scn2a deficiency exhibit intrinsic hyperexcitability. C_LIO_LISevere Scn2a deficiency leads to downregulation of multiple potassium channel genes. C_LIO_LIGenetic restoration of NaV1.2 expression alleviates spike-wave discharges (SWDs). C_LIO_LIAAV-mediated human KV1.1 delivery substantially reduced absence seizures, demonstrating the therapeutic potential of targeted gene therapy. C_LI

neuroscience↗

Novel Body-Selective Regions Responsive to Bodies Away from the Center of Gaze

We report the existence of two previously undescribed body-selective visual regions in the human brain, which we term the Ventromedial Body Area (VMBA) and the Medial Body Area (MBA). We localize these regions based on high-signal localizer contrasts and characterize selectivity in them with encoding model analysis of BOLD fMRI responses to thousands of naturalistic images. These regions respond to images of bodies away from the center of gaze.

neuroscience↗

Macrophage-derived reactive oxygen species promote Salmonella aggresome formation contributing to bacterial antibiotic persistence

In this study, we reveal that macrophage-derived reactive oxygen species (ROS) can trigger the rapid formation of Salmonella aggresomes, which substantially contribute to the increased frequency of persisters induced by phagocytosis. Salmonella containing aggresomes exhibited a dormant phenotype characterized by reduced adenosine triphosphate (ATP) levels and decreased metabolic activity. Furthermore, these dormant bacteria showed upregulated expression of Salmonella pathogenicity island 1 (SPI-1)-encoded type III secretion system (T3SS)-related genes, followed by later expression of SPI-2 T3SS-related genes when macrophages ROS production declined. Our results demonstrate that Salmonella containing aggresomes can enter a dormant state to escape antibiotic attack, while crucially maintaining the ability to resuscitate when the stress environment is improved. Research on bacterial aggresomes could potentially provide therapeutic strategies to combat bacterial antibiotic persistence. O_FIG O_LINKSMALLFIG WIDTH=200 HEIGHT=122 SRC="FIGDIR/small/657582v1_ufig1.gif" ALT="Figure 1"> View larger version (55K): org.highwire.dtl.DTLVardef@7e5f1forg.highwire.dtl.DTLVardef@970a39org.highwire.dtl.DTLVardef@204449org.highwire.dtl.DTLVardef@632c47_HPS_FORMAT_FIGEXP M_FIG C_FIG

biophysics↗

Human microglia in brain assembloids display region-specific diversity and respond to hyperexcitable neurons carrying SCN2A mutation

Microglia critically shape neuronal circuit development and function, yet their region-specific properties and roles in distinct circuits of the human brain remain poorly understood. In this study, we generated region-specific brain organoids (cortical, striatal, and midbrain), each integrated with human microglia, to fill this critical gap. Single-cell RNA sequencing uncovered six distinct microglial subtypes exhibiting unique regional signatures, including a subtype highly enriched for the GABAB receptor gene within striatal organoids. To investigate the contributions of microglia to neural circuitry, we created microglia-incorporated midbrain-striatal assembloids, modeling a core circuit node for many neuropsychiatric disorders including autism. Using chemogenetics to activate this midbrain-striatal circuit, we observed increased calcium signaling in microglia involving GABAB receptors. Leveraging this model, we examined microglial responses within neural circuits harboring an SCN2A nonsense (C959X) mutation associated with profound autism. Remarkably, microglia displayed heightened calcium responses to SCN2A mutation-mediated neuronal hyperactivity, and engaged in excessive synaptic pruning. These pathological effects were reversed by pharmacological inhibition of microglial GABAB receptors. Collectively, our findings establish an advanced platform to dissect human neuroimmune interactions in sub-cortical regions, highlighting the important role of microglia in shaping critical circuitry related to neuropsychiatric disorders. Graphical abstract O_FIG O_LINKSMALLFIG WIDTH=200 HEIGHT=188 SRC="FIGDIR/small/657874v1_ufig1.gif" ALT="Figure 1"> View larger version (72K): org.highwire.dtl.DTLVardef@125b36eorg.highwire.dtl.DTLVardef@270079org.highwire.dtl.DTLVardef@903525org.highwire.dtl.DTLVardef@19145c4_HPS_FORMAT_FIGEXP M_FIG C_FIG TeaserModeling regional microglial diversity in sub-cortical regions is challenging. We generated human organoid and assembloid models containing microglia that acquire region-specific heterogeneity. Our work shows dynamic responses of microglia when exposed to hyperexcitable midbrain-striatal circuits, providing an exciting platform to study neuroimmune interactions in human brain development and neuropsychiatric disorders, including SCN2A mutation-mediated monogenic autism. HighlightsO_LI* Single-cell RNA sequencing analyses reveal six distinct microglial subtypes that spontaneously attain unique specialization in human cortical, striatal, and midbrain organoids. C_LIO_LI* Microglia facilitate axonal projections across regional organoids, promoting assembloid formation. C_LIO_LI* Microglia respond to hyperexcitable neurons via calcium signaling and exhibit excessive pruning of neuronal synapses. C_LIO_LI* Blocking microglial GABAB receptors normalizes calcium activity and reduces synaptic pruning, suggesting a potential targeting strategy for synaptic deficits. C_LI

neuroscience↗

Autism-associated SCN2A deficiency disrupts cortico-striatal circuitry in human brain assembloids

Profound autism spectrum disorder (ASD) is frequently attributable to single-gene mutations, with SCN2A (voltage-gated sodium channel NaV1.2) protein-truncating variants (PTVs) being one of the most penetrant. Although cortico-striatal circuitry is implicated as a key node in ASD, the impact of SCN2A deficiency on human neural circuits is unknown. Using the human cortico-striatal assembloid model, we show that the autism-causing PTV SCN2A-C959X impairs long-range cortical axonal projections, reduces striatal spine density, and attenuates excitatory cortical-striatal synaptic transmission. Surprisingly, these assembloids carrying the heterozygous SCN2A nonsense mutation exhibited pronounced network hyperexcitability, a human cell-specific phenotype not observed in Scn2a+/- mice, highlighting a human-specific circuit vulnerability. Collectively, our study unveils human circuit-specific dysfunctions of SCN2A deficiency and SCN2A-mediated ASD. HighlightsO_LIAxonal projections facilitate synapse formation and functional connectivity in human brain assembloids. C_LIO_LINaV1.2 is expressed along neuronal axons, extending to soma and dendrites in human brain assembloids. C_LIO_LISCN2A-C959X disrupts axonal projection patterns, impairs excitatory synaptic transmission, reduces spine density, and results in elevated neuronal excitability. C_LI Graphical abstract O_FIG O_LINKSMALLFIG WIDTH=200 HEIGHT=90 SRC="FIGDIR/small/657036v1_ufig1.gif" ALT="Figure 1"> View larger version (27K): org.highwire.dtl.DTLVardef@6d5b1forg.highwire.dtl.DTLVardef@1795ecdorg.highwire.dtl.DTLVardef@13f0d7eorg.highwire.dtl.DTLVardef@8ee059_HPS_FORMAT_FIGEXP M_FIG C_FIG In briefO_ST_ABSSCN2A haploinsufficiency impairs cortico-striatal circuitry.C_ST_ABSSCN2A haploinsufficiency disrupts axon initial segment (AIS) integrity, leading to hyperexcitability (red arrow), reduced axon projections, and impaired synaptic transmission (decreased sEPSCs and altered network firing). These deficits result in dysfunction within the cortico-striatal circuitry.

neuroscience↗

Joint Modeling of Longitudinal Biomarker and Survival Outcomes with the Presence of Competing Risk in Nested Case-Control Studies with Application to the TEDDY Microbiome Dataset

MotivationLarge-scale prospective cohort studies collect longitudinal biospecimens alongside time-to-event outcomes to investigate biomarker dynamics in relation to disease risk. The nested case-control (NCC) design provides a cost-effective alternative to full cohort biomarker studies while preserving statistical efficiency. Despite advances in joint modeling for longitudinal and time-to-event outcomes, few approaches address the unique challenges posed by NCC sampling, non-normally distributed biomarkers, and competing survival outcomes. ResultsMotivated by the TEDDY study, we propose "JM-NCC", a joint modeling framework designed for NCC studies with competing events. It integrates a generalized linear mixed-effects model for potentially non-normally distributed biomarkers with a cause-specific hazard model for competing risks. Two estimation methods are developed. fJM-NCC leverages NCC sub-cohort longitudinal biomarker data and full cohort survival and clinical metadata, while wJM-NCC uses only NCC sub-cohort data. Both simulation studies and an application to TEDDY microbiome dataset demonstrate the robustness and efficiency of the proposed methods.

bioinformatics↗

Ketone Bodies-mediated Cysteine Modifications Discovered by Chemical Proteomics

All the studies of ketone body-dependent post-translational modifications (PTMs), notably those mediated by ketone bodies, {beta}-hydroxybutyrate (Bhb) and acetoacetate (Acac), have focused on lysine acylations. However, given the chemically diverse and reactive nature of metabolites generated, it remains unclear whether non-lysine modifications can also happen. Here, we report the synthesis of an acetoacetate-alkyne (Acac-alkyne) chemical probe that enables efficient metabolic labeling, robust fluorescent visualization, and mass spectrometry-based identification of Acac-modified proteins. By combining chemical proteomics with open-search strategy, we showed that Acac will induce previously uncharacterized cysteine modifications in mammalian cells. Notably, cysteine S-crotonation (Ccr) is validated by employing both probe-based and standard peptide-based co-elution assays. Metabolic pathway tracing further identifies BDH1 and ECHS1 as key enzymes that generate Ccr formation. Together, these findings establish ketone metabolism as a novel source of cysteine modifications and provide an alternative mechanistic pathway to explain the profound biological effects of ketone body.

biochemistry↗

Intravenous BCG immunization drives naive CD4+ T cell to Th1 differentiation via CD4+ T cell epigenetic reprogramming

Intravenous (IV) administration of live Bacille Calmette-Guerin (BCG) boosts potent immunity through coordinated interactions of innate immune response and adaptive immune response for BCG antiviral activity. The underlying mechanisms by which BCG initiates CD4+ T response remain incompletely understood. Here, we show that IV administration of BCG drives naive CD4+ T cell differentiation to Th1 cell through epigenetic reprogramming. Single-cell transcriptomic and epigenomic analyses revealed that IV BCG immunization induced metabolic reprogramming in monocytes and increased the proportion of T cells. The immunization induced chromatin openness for transcription factors regulating naive CD4+ T cells differentiation into Th1 cells. Importantly, we observed increases in Stat1, Irf1 and interferon-stimulated genes such as Igtp in Th1 cells, leading to upregulation of interferon related gene expression for antiviral response by BCG immunization. Activated transcription factors like Irf1 in CD4+ T cells promotes Il12rb1 expression to facilitate IL-12 signaling and drive naive CD4+ T cells differentiation into Th1 cells. We showed that BCG immunization reduced respiratory syncytial virus (RSV) infection. Thus, transcription regulation through epigenetic reprogramming plays a critical role in BCG-induced CD4+ T cell differentiation and to the broad antiviral activity. SignificanceBacille Calmette-Guerin (BCG) vaccination confers nonspecific protection against heterologous pathogens. Recent work demonstrated that coordinated interactions of innate immune and persistent CD4+ T cell response plays a critical role in BCG induced antiviral activity. In our study, we investigated BCG immunization on epigenetic reprogramming of CD4+ T cells and found that BCG immunization elicited antiviral activity by provoking T cell proliferation. We observed chromatin openness favoring naive T cell differentiation to Th1 response. Importantly, we observed STAT1 and interferon regulatory factor-1 (Irf1) activation. These findings provide mechanistic insights into BCG-induced Th1 response through epigenetic reprogramming and highlight the potential of BCG in offering a long-term protection against emerging and reemerging pathogens. Graphic abstract O_FIG O_LINKSMALLFIG WIDTH=200 HEIGHT=181 SRC="FIGDIR/small/655674v1_ufig1.gif" ALT="Figure 1"> View larger version (53K): org.highwire.dtl.DTLVardef@1c3cb94org.highwire.dtl.DTLVardef@16a8e8forg.highwire.dtl.DTLVardef@b58b88org.highwire.dtl.DTLVardef@dbc994_HPS_FORMAT_FIGEXP M_FIG C_FIG HighlightsO_LIIntravenous BCG immunization drives naive CD4+ T cell differentiation to Th1 response; C_LIO_LIBCG alters chromatin accessibility for transcriptional factor expression favoring Th1 response through a Irf1-Il12rb1-IL12 feedback loop; C_LIO_LIThe immunization elicits antiviral activity against RSV infection; C_LI

immunology↗

Unveiling KRAS Mutant Structures with AlphaFold 3: New Avenues for Targeted Cancer Therapy

Despite recent advances in targeting the "undruggable" KRAS (Kirsten rat sarcoma viral oncogene homologue) protein, much of its structural information remains elusive. Utilizing a "branch-pruning" strategy, this study systematically mutated the Switch I and II regions of KRAS, predicting mutants structures with AlphaFold 3 and analyzing their structure differences with KRAS wildtype using PyMOL. The analysis of 494 mutants showed that over 64% had root-mean-square deviation (RMSD) scores below 0.200, indicating minor structural changes compared to wildtype KRAS. Notably, Switch II exhibited greater conformational variability, suggesting its potential role in oncogenic signaling. This work found that Q61 mutants showed limited structural changes, but nearby sites like E63 and M67 were more dynamic. Highly dynamic sites D38 (on Switch I) and E76/G60 (on Switch II) were identified, potentially affecting KRAS dimerization and post-translational modifications in oncogenesis. Mutants like D31I, R73C, and T74Q induced conformational changes, yet their biological functions are still unknown, possibly impacting effector binding and allosteric communication. These findings could reveal cryptic pockets for drug targeting, deepening our understanding of KRASs role in cancer.

cancer biology↗

The cryo-EM structure of mouse radial spoke 3 reveals a unique metabolic and regulatory hub in cilia

Cilia are complex, microtubule-based organelles that protrude from many eukaryotic cells and have important roles in sensing, signaling, and motility. Recent studies have revealed the atomic structures of many multi-component ciliary complexes, providing new insights into their mechanisms of action that are vital for cilias biological functions. However, little is known about the structure, proteome, and function of full-length radial spoke 3 (RS3), which is distinct from the structurally well-characterized RS1 and RS2. Radial spokes are conserved megadalton complexes that transmit mechanochemical signals from the central pair of microtubules to the dynein motors, thereby coordinating ciliary motility. Here, we combined cryo-electron microscopic single-particle reconstruction, cryo-electron tomography (cryo-ET), proteomic analysis, and computational modeling to determine the 3D structure and atomic model of RS3 from mouse respiratory cilia. Our structure reveals all protein components of RS3, including regulatory and metabolic enzymes, such as a protein kinase A subunit, adenylate kinases and malate dehydrogenases. We have confirmed the important role of adenylate kinase 7 in RS3 by cryo-ET analyses of respiratory cilia in AK7-deficient mice, which display primary ciliary dyskinesia. Our findings suggest that RS3 is an important regulatory hub and cluster of metabolic proteins that helps to maintain ATP at the levels required for sustained dynein motor activity and ciliary beating. This work advances our understanding of the structure and function of RS3 in ciliary motility and provides insights into the etiology of ciliopathies.

biophysics↗

Low-Strength Type I Interferon Signaling Promotes CAR T-Cell Treatment Efficacy

CD19-directed chimeric antigen receptor (CAR) T-cell therapy has significantly advanced the treatment landscape for relapsed/refractory diffuse large B-cell lymphoma (r/r DLBCL). However, up to 60% of patients do not achieve a complete response. To uncover determinants of therapeutic efficacy, we analyzed the infusion products of eight r/r DLBCL patients with distinct clinical responses to axicabtagene ciloleucel using single-cell transcriptomics. Compared to patients who exhibited progressive disease, infusion products of complete responders demonstrated enriched signatures of type I interferon (IFN-I) signaling. Based on these findings, we developed a novel strategy to improve CD19-directed CAR T-cell treatment efficacy by incorporating IFN-I as an enhancer during the ex vivo manufacturing process, with IFN-I removal before CAR T-cell infusion to avoid in vivo toxicities. For both CD28- and 4-1BB-costimulated second-generation CARs, we found that low-strength IFN-I signaling enhanced CAR T-cell cytotoxicity and treatment efficacy against B-cell lymphoma and leukemia. Our low-strength IFN-I-enhanced CAR T-cell ex vivo manufacturing approach leverages an existing FDA-approved pharmacologic agent, circumvents in vivo interferon-associated toxicities, and remains fully compatible with current CAR constructs and manufacturing workflows. Together, our results establish IFN-I as a potent and costimulation-independent enhancer of CAR T-cell efficacy and provide a translationally feasible approach to enhance CAR T-cell therapies.

immunology↗