This review explores the molecular mechanisms regulating exon 10 splicing, focusing on how exonic and intronic splicing mutations destabilise mRNA structures or alter splicing factor binding, and discusses how these changes can cause disease symptoms in experimental models.
Abstract RNA editing is a major post-transcriptional process in plant organelles that frequently alters coding sequences and can influence mitochondrial gene function. However, organ-resolved RNA editing in mitochondrial nad transcripts remains poorly characterized in Catharanthus roseus (L.) G.Don, despite the biologi...
A. A. Alnufaei· Russian journal of plant phy...· 0 citations
Plant organelle genomes are important for evolutionary biology research, yet remain poorly characterized in genus Terminalia (Combretaceae). Here, we systematically analyzed the mitochondrial genomes (mtDNA) and chloroplast genomes (cpDNA) of three Terminalia species ( Terminalia chebula , Terminalia franchetii , Termi...
Xingyu Liu, Yingmin Zhang, Pengrui Luo et al.· BMC Genomics· 0 citations
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
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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
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.
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.