Abstract Trypanosoma cruzi, the causative agent of Chagas disease, relies on complex gene regulatory mechanisms to adapt to its diverse host environments. In recent years, it has been established that epigenetics plays an essential role in these mechanisms via the regulation of chromatin structure. Bromodomain-containi...
Victoria Boselli Raposo, Virginia Perdomo, Florencia Pacini et al.· ACS Infectious Diseases· 0 citations
Key translational challenges are discussed, focusing on the imperative to optimize delivery vectors for widespread central nervous system distribution, the necessity of advanced human-derived neuronal models to validate functional recovery, and the critical need to prevent iatrogenic Angelman Syndrome caused by UBE3A i...
S. Alberti, Irina Cutei, Giacomo Costa Broccardi et al.· Molecular Therapy· 0 citations
Ex vivo genome editing of hematopoietic stem and progenitor cells (HSPCs) holds significant therapeutic potential but remains constrained by genotoxic risks associated with nuclease-induced DNA double-strand breaks, DNA donor template delivery and sensing, and proliferation-induced stress during ex vivo manipulation. T...
Roberta Vacca, Lucrezia della Volpe, Nicolò Gualandi et al.· Molecular Therapy· 0 citations
ABSTRACT Life sciences represent one of the most dynamic and transformative scientific domains in the modern world. Rapid technological progress, interdisciplinary research integration, and molecular-level understanding of biological systems have revolutionized healthcare, agriculture, environmental protection, and bio...
Adi Reddy Rakkireddy· The American Journal of Scie...· 0 citations
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Lipid nanoparticles (LNPs) are the leading non-viral platform for nucleic acid delivery, validated clinically by their use in mRNA vaccines and the siRNA drug patisiran. Nevertheless, their wider use in protein-replacement and gene-editing therapeutics need repeated doses is limited by inflammatory side effects caused...
Combined with CRISPR-based gene editing and phenotypic assays, this integrative approach positions zebrafish as a cornerstone of modern pharmacology and a transformative platform for CNS therapeutics.
Yida Huang, Xiang Liu, Guang-Yi Fan et al.· British Journal of Pharmacol...· 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
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.
The results demonstrate that common genetic variants can influence disease risk through post-transcriptional mechanisms that alter lipid homeostasis in astrocytes, a cell type increasingly recognized as central to ALS progression.
Alan Guo, S. Lessard, Li-Lu Guo et al.· bioRxiv· 0 citations
Advances in mesenchymal stem cells, extracellular vesicles, CRISPR-based gene editing, and emerging precision medicine strategies including multi-omics profiling, machine learning-based patient stratification, and adaptive trial design are discussed.
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
Zero-knowledge proofs can verify computing results without rerunning workloads or exposing sensitive data. Magnetar helps bring them into the mainstream with support for standard programming languages, major speed gains, and quantum-resistant security. The post Proof-based general-purpose computing with Magnetar appeared first on Microsoft Research.
