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327 papers
Hemoglobinopathies provide the first clinically validated delivery model for CRISPR therapeutics, and offers a donor-independent alternative to allogeneic hematopoietic stem-cell transplantation without graft rejection or graft-versus-host disease.
A pH-gated surface-mediated hydrogel patch for infection-responsive delivery of CRISPR-Cas9 ribonucleoprotein (RNP) complexes establishes a novel paradigm for localized, precision gene therapy and holds strong translational promise for the treatment of chronic inflammatory wounds.
The T-Editor platform enables rapid and efficient CRISPR-mediated gene editing for engineering TIL to enhance its therapeutic potency and FAM84B may represent a novel potential target for improving TIL-mediated antitumor activity.
An optimized protocol balancing high transfection/transduction efficiency with minimized cytotoxicity was developed, supporting tissue-engineered IVD constructs and other CRISPR-based regenerative therapies for DDD.
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The genetics of hearing loss is transitioning from a diagnostic modality to an interventional one, with emphasis on emerging gene-based therapies, clinical trial design, regulatory and ethical considerations, and practical implications for otolaryngologists as biologic treatment enters clinical practice.
This optimized PE system substantially enhances PE efficiency in Cucurbit crops, providing an effective solution to common challenges such as low editing efficiency and limited heritability in these species.
This review summarizes recent progress in elucidating plant virus-vector molecular interactions and their potential use in innovative strategies for virus and vector control.
An RNA-guided bridge recombinase system is engineered through rational mutagenesis and AI-assisted directed evolution, enabling programmable chromosomal rearrangements in both plant and mammalian cells and achieving up to a 29.8-fold increase in activity.
Compared with conventional homologous recombination, the CRISPR-Cas9 system substantially improved gene disruption efficiency, thereby overcoming a major limitation in the genetic manipulation of lichen-forming fungi.
This first-in-human study demonstrated the feasibility of an "off-the-shelf" base-edited CAR T cell approach and informs future multiantigen strategies against AML.
Cell-specific epigenetic editing holds very high therapeutic value for atherosclerosis, cardiomyopathy, and fibrosis, provided that delivery, specificity, and safety challenges are also addressed.
This synthesis provides a framework for translating mechanistic hormonal insights into field-applicable cultivars to ensure global food security and proposes a systems-level roadmap for developing climate-resilient rice cultivars capable of maintaining yield stability across a volatile combinatorial stress landscape.
Human-Computer Interaction and Visualization
A new algorithm learns to anticipate the unprecedented scenarios that critical infrastructure and global supply chains are least prepared for.
Generative AI
A new method for surgically removing training examples from a model reveals that as datasets grow, the link between what a model learns and what it produces dissolves.
In his latest book, Professor Eugene Fitzgerald examines the forces that turn breakthroughs into value — and why innovation resists simple formulas.
General Science
