Engineering Biology Beyond Single Genes: Advances and Challenges in Multiplex Genome Editing
Animal Research and One Health, EarlyView.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
Delivery of Adeno-Associated Virus Vectors to the Central Nervous System for Correction of Single Gene Disorders. [PDF]
Int J Mol SciDaci R, Flotte TR.
europepmc +1 more source
Ocular Gene Therapy with Adeno-associated Virus Vectors: Current Outlook for Patients and Researchers. [PDF]
J Ophthalmic Vis Res, 2020 Casey GA, Papp KM, MacDonald IM.
europepmc +1 more source
Early Synovial Regulatory T Cell Engagement in Posttraumatic Osteoarthritis
Arthritis &Rheumatology, EarlyView.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
British Journal of Clinical Pharmacology, EarlyView.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
A Quantitative In Vitro Potency Assay for Adeno-Associated Virus Vectors Encoding for the UGT1A1 Transgene. [PDF]
Mol Ther Methods Clin Dev, 2020 Aronson SJ +13 more
europepmc +1 more source
What Makes an “Ideal” Cell Line for Recombinant Adeno‐Associated Virus Production?
Biotechnology and Bioengineering, EarlyView.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
Adeno-associated virus vectors and neurotoxicity-lessons from preclinical and human studies. [PDF]
Gene TherStone D, Aubert M, Jerome KR.
europepmc +1 more source
Enhancing Adeno‐Associated Virus 2 Capsid Engineering Through Co‐Evolutionary Coupling
Biotechnology and Bioengineering, EarlyView.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
Applications and Limitations of Equilibrium Density Gradient Analytical Ultracentrifugation for the Quantitative Characterization of Adeno-Associated Virus Vectors. [PDF]
Anal ChemHirohata K +15 more
europepmc +1 more source