Results 181 to 190 of about 326,770 (247)
T Cell‐Mediated Targeting of Interneurons in Mice Shapes Hippocampal Remodeling and Epilepsy
Objective Autoimmune encephalitis (AE) is associated with autoantibodies targeting distinct neuronal populations. In AE, antibodies against glutamate decarboxylase 65 (GAD65), expressed in GABAergic interneurons, are frequently detected. In GAD65‐AE, hippocampal biopsies often show infiltrates of CD8+ cytotoxic T cells (CTLs), suggesting a prominent T ...
Daniel S. Galvis‐Montes +6 more
wiley +1 more source
Engineering Biology Beyond Single Genes: Advances and Challenges in Multiplex Genome Editing
Multiplex genome editing is transforming genome engineering from single‐gene perturbation to network‐level control, yet its broader application remains limited by challenges in gRNA array engineering, delivery technologies, and safety management. Emerging AI‐driven approaches are accelerating guide RNA design and CRISPR effector optimization for ...
Linli Wang, Yongbin Liu, Hongbing Han
wiley +1 more source
Early Synovial Regulatory T Cell Engagement in Posttraumatic Osteoarthritis
Objective Posttraumatic osteoarthritis (PTOA) develops despite restoration of joint biomechanics, suggesting that early postinjury biologic processes within the synovium shape disease trajectories. We investigated whether synovial Treg cells are engaged during the early phase following joint injury and whether experimental enrichment of this ...
Xueyou Zhang +5 more
wiley +1 more source
Precision medicine in paediatrics: Progress and priorities
Precision medicine is revolutionizing personalized healthcare, advancing both diagnostics and therapeutics at an unprecedented pace. Reviewing the paediatric applications of pharmacometrics, pharmacogenomics and advanced therapy medicinal products highlights not only the relevance of these exciting innovations to frontline care but also the significant
Nicola Husain +3 more
wiley +1 more source
What Makes an “Ideal” Cell Line for Recombinant Adeno‐Associated Virus Production?
Several host cell types have been used to produce rAAVs to date. Cell line‐specific traits that are beneficial in the context of rAAV manufacturing are reviewed here, with the goal of developing a consensus on the ‘ideal’ characteristics that an rAAV production host should possess (created with Biorender.com).
James Conheady +6 more
wiley +1 more source
Enhancing Adeno‐Associated Virus 2 Capsid Engineering Through Co‐Evolutionary Coupling
ABSTRACT Adeno‐associated virus (AAV) has emerged as a leading platform for gene therapy. However, to unlock the full potential, their manufacturing yields, stability, and efficacy must all be improved. Rational design is limited by poor predictability and the potential impact of mutations on multiple important vector properties.
Sirimar Laosinwattana +4 more
wiley +1 more source
ABSTRACT Generation of CAR‐T cell therapies relies predominantly on lentiviral vectors (LVs). As in vivo CAR‐T trials progress, demand for larger quantities of higher‐potency LV is expected. However, primary capture by anion‐exchange chromatography (AIEX) remains a major manufacturing bottleneck from use of adsorbents not tailored to LVs, leading to ...
George Pamenter +10 more
wiley +1 more source
Lifecycle perspective on cell and gene therapy manufacturing challenges and enabling GMP solutions. ABSTRACT Cell and gene therapies (CGTs) are revolutionizing modern medicine; however, making these advanced medicines scalable and readily available to commercial manufacturers worldwide is a major challenge. The number of approved CGT products continues
Rajath Samaga +2 more
wiley +1 more source
ABSTRACT Vaccines and gene therapies require efficient delivery of genetic material to host cells. Viral vectors leverage viruses' ability to enter host cells to achieve this. Following the SARS‐CoV‐2 vaccine and novel gene therapies that utilize adenovirus vectors, there has been an increase in the demand for large‐scale production of vectors.
Betsy Widdicombe +7 more
wiley +1 more source
ABSTRACT Adeno‐associated viruses (AAVs) are the leading vectors for in vivo gene therapy, fueling a growing demand for scalable and robust biomanufacturing processes. Yet, the impact of cell lysis conditions on the performance of midstream and downstream unit operations across serotypes remains poorly characterized.
Arianna Minzoni +20 more
wiley +1 more source

