gene editing

Engineering RAG Systems for Real-World Applications: Design, Development, and Evaluation

Five domain-specific RAG applications developed for real-world scenarios across governance, cybersecurity, agriculture, industrial research, and medical diagnostics are presented, highlighting technical, operational, and ethical challenges affecting the reliability and usability of RAG systems in practice.

Anomaly detection in smart power grids with graph-regularized MS-SVDD: a multimodal subspace learning approach

A generalized Multimodal Subspace Support Vector Data Description model with graph-embedded regularization is proposed, illustrating how relational and structural information can be systematically embedded into one-class models, enabling robust learning under complex, high-dimensional, and multimodal conditions.

Bridging Humans and LLMs: Investigating Human-AI Collaboration in Multi-agent Requirements Analysis for Organizational AI Adoption

LLM-based multi-agent systems can support strategic AI planning by enabling iterative refinement with human experts by supporting structured and collaborative Requirements Engineering processes for AI adoption planning.

Genetically Modified MSCs for Targeted Regeneration: Balancing Efficacy, Biosafety, and GMP Standardization

Development of safe-harbor integration and B2M/CIITA-based hypoimmunogenic designs as strategies to reduce engineering-related batch variability and HLA-dependent donor variability support a transition from empirical optimized MSC preparations toward molecularly defined cellular medicines with predefined genotype, expr...

Mitochondria in health and disease: cellular powerhouses, signaling centers, and drivers of dysfunction

Recent studies demonstrate promising therapeutic targets for mitochondrial dysfunction and highlight the need for research in mitochondrial function to change the therapeutic landscape in the management of mitochondrial dysfunction-associated diseases.

Clean Attacks: Formalizing Semantically Valid Adversarial Behavior in Autonomous AI Agent Systems

A previously unstated class of adversarial input called a clean attack - an input that is syntactically correct, semantically consistent with the declared task context, consistent with all observable policy constraints and still has the goal of misguiding the agent away from the original operator goal - is identified a...

REFINE: A Multi-Agent LLM Approach for Evidence-Guided Code Refactoring

This work introduces REFINE (Refactoring with Evidence-aware Flow for Integrated ageNtic Execution), a tool-agnostic, evidence-aware multi-agent approach for generating Java file-level refactoring candidates that achieves a higher median code-smell reduction with smaller edits and fewer public-method removals.

Unlocking the Curative Potential of Gene Transfer and Editing for Hematopoietic Disorders.

Recent developments and refinements in gene transfer and editing technologies for HSPCs are reviewed, while also discussing the critical limitations and hurdles to clinical translation, as recently presented at the New Investigator Committee Gene Therapy webinar.

Elucidate the structural role of helper lipids in modulating hepatic expression following repeated intravenous administration of mRNA-LNPs

Lipid nanoparticles (LNPs) are the most advanced non-viral platform for hepatic mRNA delivery, yet the functional role of helper phospholipids, particularly under repeated dosing regimens required for chronic protein replacement therapies, remains poorly understood. Here, we systematically investigated six helper phosp...

Emerging Nucleic Acid-Based Therapies for Hypercholesterolemia with Focus on a New Modality, Liver-Directed miR-30c Analog C2

This review summarizes recent advances, mechanisms of action, clinical progress, and remaining challenges across antisense oligonucleotides, siRNAs, gene therapy, genome editing, and emerging miRNA-based therapeutics for hypercholesterolemia.

Genome-wide association studies and genomics-assisted breeding in pearl millet (Pennisetum glaucum): Advancing climate resilience, nutritional security, and sustainable production

Exploring the Potential of CRISPR-Cas9 Gene Editing for Sickle Cell Disease and Malaria Resistance: A Systematic Review

Current evidence supports separate translational pathways in SCD and malaria applications remaining predominantly preclinical, with clinical progress established in SCD and malaria applications remaining predominantly preclinical.