Data related to Figure 5g in the manuscript "High-throughput optimized prime editing-mediated endogenous protein tagging for pooled imaging of protein localization".Representative 10-cell albums for all confidently tagged genes (SupplementaryData2-3) and tagging spacers (SupplementaryData4-7). Single cell images were r...
Henry Sanchez, Deepak C. Mani, Tomer Lapidot et al.· Figshare· 0 citations
Data related to Figure 5g in the manuscript "High-throughput optimized prime editing-mediated endogenous protein tagging for pooled imaging of protein localization".Representative 10-cell albums for all confidently tagged genes (SupplementaryData2-3) and tagging spacers (SupplementaryData4-7). Single cell images were r...
Henry Sanchez, Deepak C. Mani, Tomer Lapidot et al.· Figshare· 0 citations
A multidomain framework is proposed that distinguishes target engagement, molecular correction, downstream pharmacodynamic responses, organ function, and whole-animal or patient-relevant benefit without assuming that effective treatments must improve every domain sequentially.
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease with ∼90% of cases being sporadic. Although genome-wide association studies have identified numerous genetic risk loci, these variants account for only a portion of ALS heritability, suggesting that additional genetic and regulatory mechanisms con...
Alan Guo, S. Lessard, Li-Lu Guo et al.· bioRxiv· 0 citations
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Heart failure (HF) is one of the most urgent unmet challenges in cardiovascular medicine. Despite advances in guideline-directed medical therapy (GDMT), substantial morbidity and mortality remain high in patients with advanced disease. This suggests a need for disease-modifying interventions that address u...
Russell G. Saltzman, K. Asensi, Arnab Chakraborty et al.· Expert Review of Cardiovascu...· 0 citations
Findings indicate that the Cas12a system does not yield locus excision at the efficiency required for sensitive negative selection assays, and showed no meaningful correlation between gRNA efficiency, orientation, GC content, or cut site geometry and the success of locus excision.
N. Anvar, Zsofia M. Szegletes, R. Steger et al.· bioRxiv· 0 citations
Heterozygous loss-of-function variants causing gene dosage reduction underlie many human genetic disorders, yet preclinical mouse models frequently fail to recapitulate human disease phenotypes due to post-translational compensation. Here, we present a generalizable framework to modulate gene dosage by alternative spli...
Eric D. Smith, Joshua K. Meisner, Abbey Bullard et al.· bioRxiv· 0 citations
Mutations in the epigenetic regulator ASXL1 are common in myeloid malignancies and portend a near-universally poor prognosis. While multiple mechanisms for mutant ASXL1-dependent oncogenesis have been proposed, none have been functionally validated in the context of the human hematopoietic stem cell, where these mutati...
Madison L. Hall, Aliya Quintal, Sonia N. Acharya et al.· Blood· 0 citations
It is demonstrated that technology CRISPR–Cas9 gene editing holds significant promise as a therapeutic strategy for TP53-mutant CLL by enabling precise gene correction of functional replacement.
A. Udristioiu, Manole Cojocaru· Cancer Research· 0 citations
Roles for certain genetic variants in CHEK2 in NB are supported, as well as potential mechanistic targets for high-risk or therapy-resistant NB, and preclinical information is provided to inform diagnostics and potential therapeutic strategies for high-risk NB.
Xue-Ting Xiong, Meagan Beffort, L. Kee et al.· Cancer Research· 0 citations
This dataset was generated as part of a research project investigating how transfer RNA (tRNA) abundance and tRNA modification regulate codon-dependent translation in hepatocellular carcinoma (HCC). The data cover two complementary aspects of tRNA-mediated translational regulation.The first project examines the relatio...
Environmental particulate exposure contributes to the severity and persistence of numerous acute and chronic lung diseases, including asthma, viral pneumonia, acute respiratory distress syndrome, and pulmonary fibrosis. Chitin, a highly abundant environmental polysaccharide present in fungi, insects, and mites, is cont...
Yilin Wang· Washington University in St....· 0 citations
Exploring how generative AI could make machine vision more accessible to businesses. The post GenEye in a Box: Making Machine Vision Something You Can Just Ask For appeared first on GPT-Lab.