perturbation response prediction
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1,000+ papers
- BiophysicsbioRxivPreprint
DrugGym: A testbed for the economics of autonomous drug discovery
Michael R. Retchin, Yuanqing Wang, Kenichiro Takaba, John D. Chodera
DrugGym simulates drug discovery processes to evaluate how different strategies affect success rates and costs in achieving target candidate profiles.
11 citations - Cancer BiologyCancer DiscoveryPeer-reviewed
Targeting Acute Myeloid Leukemia Stem Cells Through Perturbation of Mitochondrial Calcium
Anagha Inguva Sheth, Krysta L. Engel, Hunter Tolison…·Craig T. Jordan
This study identifies mitochondrial calcium utilization as a metabolic requirement of venetoclax-resistant acute myeloid leukemia stem cells and shows that targeting mitochondrial calcium uptake can eradicate these cells.
61 citationstop 1% - Molecular BiologyApplied and Environmental MicrobiologyPeer-reviewed
Mobile-CRISPRi as a powerful tool for modulating Vibrio gene expression
Logan J. Geyman, Madeline P. Tanner, Natalia Rosario-Meléndez…·Julia C. van Kessel
The paper introduces Mobile-CRISPRi, a system for gene knockdown in Vibrio species, showing it effectively modulates gene expression and phenotypes.
10 citations - NeuroscienceCellPeer-reviewed
Massively parallel in vivo Perturb-seq reveals cell type-specific transcriptional networks in cortical development
Xinhe Zheng, Boli Wu, Yuejia Liu…·Xin Jin
This study develops a high-throughput in vivo Perturb-seq platform to analyze gene function across diverse cell types in the brain and peripheral nervous system, identifying cell type-specific transcriptional networks.
- GeneticsNature CommunicationsPeer-reviewed
Genome-scale analysis of interactions between genetic perturbations and natural variation
Joseph J. Hale, Takeshi Matsui, Ilan Goldstein…·Ian M. Ehrenreich
The paper identifies gene-perturbation interactions across genetic backgrounds in yeast using CRISPRi and combinatorial DNA barcode sequencing, revealing 460 genes with variable effects and 234 interacting loci.
6 citations - GenomicsNature BiotechnologyPeer-reviewed
Bidirectional epigenetic editing reveals hierarchies in gene regulation
Naomi M. Pacalin, Zachary Steinhart, Quanming Shi…·Howard Y. Chang
This study introduces CRISPRai, a bidirectional epigenetic editing system, to investigate gene regulation hierarchies and interactions in T cells and hematopoietic lineage transcription factors.
46 citationstop 2% - Synthetic BiologybioRxivPreprint
Programming CRISPRi-dCas9 to control the lifecycle of bacteriophage T7
This study uses CRISPRi-dCas9 to control the lifecycle and infectivity of bacteriophage T7 by targeting host-dependent promoters, showing increased time to lysis and reduced plaque size.
1 citation - Synthetic BiologybioRxivPreprint
Engineered lysins to modulate human gut microbiome communities
Juan R. Diaz Rodriguez, Wenbo Lu, James M. Papadopoulos…·Philip A. Romero
This study shows engineered lysins can target and remodel synthetic human gut microbiomes by altering species preferences through domain recombination.
2 citations - Cell BiologybioRxivPreprint
Proteasomal control of anti-CRISPRs for the regulation of CRISPR/Cas9 activity using Cas9-ACROBAT
Timothy D. Martin, Emma V. Watson, Mei Yuk Choi…·Stephen Joseph Elledge
The paper describes a method to control CRISPR/Cas9 activity using proteasomal degradation of an anti-CRISPR protein fused to a degradation or shutoff tag.
DOI 10.1101/2024.05.13.593596 - BioinformaticsBioinformaticsPeer-reviewed
From Cell-Lines to Cancer Patients: Personalized Drug Synergy Prediction
Halil İbrahim Kuru, A. Ercüment Çiçek, Öznur Taştan
This paper introduces PDSP, a deep learning framework that uses patient-specific single-drug response data to predict personalized drug synergy, improving accuracy by 27% in leukemia patients.
14 citations - GeneticsNeuronPeer-reviewed
A systems biology-based identification and in vivo functional screening of Alzheimer's disease risk genes reveals modulators of memory function
Adam D. Hudgins, Shiyi Zhou, Rachel N. Arey…·Yousin Suh
The paper identifies LOAD risk genes through systems biology and shows their impact on memory function via neuronal endosomal dysfunction in C. elegans.
3 citations - Molecular BiologybioRxivPreprint
MOSAIC enables in situ saturation mutagenesis of genes and CRISPR prime editing guide RNA optimization in human cells
Jonathan Y. Hsu, Kin Chung Lam, Justine Shih…·J. Keith Joung
MOSAIC enables high-throughput in situ saturation mutagenesis and CRISPR prime editing guide RNA optimization in human cells, identifying optimized pegRNAs and drug resistance variants.
1 citation - Molecular BiologybioRxivPreprint
Allele-specific targeting of mutant TOR1A by the compact CRISPR/NmCas9 system in DYT1 dystonia with high fidelity
This study evaluates allele-specific targeting of the mutant TOR1A{Delta}E allele using NmCas9, showing high specificity and efficacy in reducing ubiquitin accumulation in DYT1 fibroblasts.
1 citation - BioengineeringJournal of Chemical Information and ModelingPeer-reviewed
Protein Engineering with Lightweight Graph Denoising Neural Networks
Bozitao Zhong, Lirong Zheng, Banghao Wu…·Liang Hong
This study introduces a lightweight graph neural network for protein engineering that predicts fitness and guides the design of effective mutants with improved physicochemical properties.
39 citationstop 4% - BioinformaticsBioinformaticsPeer-reviewed
CODEX: COunterfactual Deep learning for the in-silico EXploration of cancer cell line perturbations
Stefan Schrod, Helena U. Zacharias, Tim Beißbarth…·Michael Altenbuchinger
CODEX models causal effects of perturbations on cancer cell lines using counterfactual reasoning, enabling in-silico exploration of drug combinations and genetic modifications.
9 citations - BioinformaticsbioRxivPreprint
Seed sequences mediate off-target activity in the CRISPR-interference (CRISPRi) system
Neha Rohatgi, JP Fortin, Ted Lau…·Rohit Reja
The paper investigates off-target activity in CRISPRi by analyzing a genome-wide sgRNA library and finds that seed sequences mediate most off-target effects.
2 citations - Cancer BiologyNaturePeer-reviewed
Tumor-selective effects of active RAS inhibition in pancreatic ductal adenocarcinoma
Urszula N. Wasko, Jingjing Jiang, Tanner C. Dalton…·Kenneth P. Olive
This study evaluates the therapeutic potential of RMC-7977, a broad-spectrum RAS inhibitor, in pancreatic ductal adenocarcinoma models, showing strong anti-tumor effects with minimal impact on normal tissues.
- Synthetic BiologySmallPeer-reviewed
Engineering Material Properties of Transcription Factor Condensates to Control Gene Expression in Mammalian Cells and Mice
Alexandra Fischer, Hanah B. Robertson, Deqiang Kong…·Wilfried Weber
This study tunes the material properties of transcription factor condensates to show their impact on gene expression levels in mammalian cells and mice.
- Synthetic BiologyNucleic Acids ResearchPeer-reviewed
CRISPR-Cas tools for simultaneous transcription & translation control in bacteria
Ryan A. L. Cardiff, Ian Faulkner, Juliana G Beall…·Jesse G. Zalatan
The paper develops CRISPR-Cas tools to independently control transcription and translation in E. coli, showing reduced polar effects with dCas13 and enabling multiplexed gene regulation for biosynthesis.
24 citations - NeurosciencebioRxivPreprint
Genetic editing of primary human dorsal root ganglion neurons using CRISPR-Cas9 with functional confirmation
Seph M. Palomino, Katherin A. Gabriel, Juliet M. Mwirigi…·Amol M. Patwardhan
This paper describes a non-viral CRISPR-Cas9 transfection method for editing primary human dorsal root ganglion neurons and confirms edits at genomic, protein, and functional levels.
3 citations - Cancer BiologyProceedings of the National Academy of SciencesPeer-reviewed
Development of an orally bioavailable mSWI/SNF ATPase degrader and acquired mechanisms of resistance in prostate cancer
Tongchen He, Caleb Cheng, Yuanyuan Qiao…·Arul M. Chinnaiyan
This paper develops an orally bioavailable PROTAC degrader of mSWI/SNF ATPases for prostate cancer and identifies resistance mechanisms through long-term treatment.
- GenomicsFEBS Open BioPeer-reviewed
Signature reversion of three disease-associated gene signatures prioritizes cancer drug repurposing candidates
Jennifer L. Fisher, Elizabeth J. Wilk, Vishal H. Oza…·Brittany N. Lasseigne
The paper identifies drug repurposing candidates for low-survival cancers by prioritizing drugs that reverse disease-associated gene signatures using three methods.
6 citations - NeurosciencebioRxivPreprint
Effects of early life adversity and adolescent basolateral amygdala inhibition on corticolimbic connectivity and anxiety behaviors
Caitlyn R. Cody, Emilce Artur de la Villarmois, Anabel M. M. Miguelez Fernández…·Heather C. Brenhouse
This study investigates how early life adversity and adolescent BLA inhibition affect corticolimbic connectivity and anxiety behaviors in animals.
- Molecular BiologyEMBO ReportsPeer-reviewed
Transcript-specific induction of stop codon readthrough using CRISPR-dCas13 system
Lekha E. Manjunath, Anumeha Singh, Sangeetha Devi Kumar…·Sandeep M. Eswarappa
This paper describes a CRISPR-dCas13 system to induce stop codon readthrough in specific transcripts, showing enhanced readthrough in AGO1, VEGFA, and HBB mRNAs.
- GenomicsbioRxivPreprint
CROPseq-multi: a universal solution for multiplexed perturbation in high-content pooled CRISPR screens
Russell T. Walton, Yue Qin, Bryce Kirby…·Paul C. Blainey
CROPseq-multi enables multiplexed CRISPR perturbations across various screening methods with improved barcode detection and compatibility.
24 citations - NeurosciencebioRxivPreprint
An arrayed CRISPR/Cas9 screen identifies mTORC1 as a regulator of lipid droplet accumulation in APOE E3 and APOE KO iPSC-derived microglia
Sonja Meier, Anne Sofie G. Larsen, Florian Wanke…·Ravi Jagasia
DOI 10.1101/2024.03.16.585330 - GenomicsCell GenomicsPeer-reviewed
Modular vector assembly enables rapid assessment of emerging CRISPR technologies
Abby V. McGee, Yanjing V. Liu, Audrey L Griffith…·John G. Doench
The paper introduces a modular CRISPR vector assembly system called Fragmid, enabling rapid creation and testing of CRISPR technologies through combinatorial design and pooled screening.
12 citations - Systems BiologybioRxivPreprint
A Targeted Genome-scale Overexpression Platform for Proteobacteria
Amy B. Banta, Ashley N. Hall, Kevin S. Myers…·Jason M. Peters
The paper develops CRISPRtOE, a genome-scale overexpression platform for Proteobacteria, demonstrating its use in Escherichia coli and Zymomonas mobilis to identify antibiotic and engineering targets.
12 citations - NeurosciencebioRxivPreprint
A Massively Parallel CRISPR-Based Screening Platform for Modifiers of Neuronal Activity
Steven C. Boggess, Vaidehi Gandhi, Ming‐Chi Tsai…·Martin Kampmann
The paper develops a CRISPRi-based screening platform to identify genes affecting neuronal excitability in human iPSC-derived neurons, revealing new links to neurodegenerative and neurodevelopmental disorders.
6 citations