gene editing

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.

A Review on Advancing Stem Cell Therapy and Tissue Engineering to Regenerate Damaged Tissues and Organs

In the field of regenerative medicine, developing stem cell therapy and tissue engineering has become a game-changing strategy that holds promise for replacing and repairing damaged organs and tissues. Innovative biological interventions are required since traditional therapy approaches frequently fail to restore total...

Recent Advances in Joubert Syndrome: Genetic Insights, Clinical Management, and Emerging Therapeutic Strategies

Joubert syndrome (JS) is a rare, genetically heterogeneous autosomal recessive ciliopathy characterized by the pathognomonic molar tooth sign (MTS) on brain magnetic resonance imaging, resulting from cerebellar vermis hypoplasia or aplasia and abnormal superior cerebellar peduncles. Since its first description by Marie...

The Role of Comparative Genomic Bioinformatics in Predicting and Managing Wheat Rust Disease in Wheat Crops: A Multi-Omics Approach for Pathogen Genomics and Host Resistance Identification

The yield results of wheat (Triticum aestivum L.) suffer critical losses annually from rust diseases including yellow rust, leaf rust and stem rust. The study explores how comparative genomic bioinformatics supports wheat rust disease prevention and control by examining multiple-omic data approaches. The research demon...

Recent Advances in Non-Viral Vectors for Gene Therapy and Gene Delivery: From Lipid Nanoparticles to Engineered Extracellular Vesicles

This review summarizes recent advances in non-viral gene-delivery platforms, compares their strengths and limitations, and discusses future directions in cell-selective delivery, endosomal escape, transient delivery of genome-editing machinery, engineered EVs, hybrid vectors, and manufacturing-oriented development.

Development of a multi - Targeted Combinatorial Nano therapeutic Cascade Using CRISPR Intrabodies ,and BDNF Growth Factors For Huntington 's Disease Neuro Regeneration "

वैज्ञानिक अनुसंधान प्रस्ताव (Research Article Proposal)शीर्षक (Title): हंटिंगटन रोग के न्यूरो-रीजनरेशन के लिए मल्टी-टार्गेटेड जीन-एंटीबॉडी नैनोथेराप्यूटिक कास्केड का कार्यान्वयनआविष्कारक का नाम (Inventor Name): बंटी कुमावत (Banti Kumawat)वर्ष (Year): 2026🔬 1. प्रस्तावना (Introduction)हंटिंगटन रोग (Huntington's Disease...