Direct Identification and Quantification of Recombinant Adeno-Associated Virus in Crude Cell Lysate and Conditioned Medium by Mass Photometry. [PDF]
Yamaguchi Y+8 more
europepmc +1 more source
High-Titer, Wild-Type Free Recombinant Adeno-Associated Virus Vector Production Using Intron-Containing Helper Plasmids [PDF]
Lei Cao+3 more
openalex +1 more source
Precision therapies for genetic epilepsies in 2025: Promises and pitfalls
Abstract By targeting the underlying etiology, precision therapies offer an exciting paradigm shift to improve the stagnant outcomes of drug‐resistant epilepsies, including developmental and epileptic encephalopathies. Unlike conventional antiseizure medications (ASMs) which only treat the symptoms (seizures) but have no effect on the underlying ...
Shuyu Wang+3 more
wiley +1 more source
Adeno-associated virus vector modification based on directed evolution technology for gene therapy targeting head and neck squamous cell carcinoma. [PDF]
Zhu Y, Ji W, Zhang Q, Dong Y, Liu L.
europepmc +1 more source
Saikosaponin b1 attenuates liver fibrosis by competitively disrupting the interaction between signal transducer and activator of transcription 3 (STAT3) and glioma‐associated oncogene‐1 (Gli1) in the activated hepatic stellate cells (HSCs). The blocked interaction of STAT3 and Gli1 promotes the degradation of Gli1, inhibits Gli1 transcriptional ...
Meiyu Shao+15 more
wiley +1 more source
Erratum: Assessment of adeno-associated virus purity by capillary electrophoresis-based western. [PDF]
Acevedo J, Bi Y, Gee J, Khatwani SL.
europepmc +1 more source
Gene Therapy Vectors Based on Adeno-Associated Virus Type 1
Weidong Xiao+5 more
openalex +1 more source
This review provides a detailed description of gene mutations and epigenetic mechanisms in central nervous system (CNS) disease, strategies for gene therapy, challenges for CNS gene therapy and gene‐drug nanocarriers. This review also summarizes the applications of nanogenetic therapy platforms in CNS diseases (brain tumors, neurodegenerative diseases,
Fuming Liang+4 more
wiley +1 more source
Harnessing cell aggregates for enhanced adeno-associated virus manufacturing: Cultivation strategies and scale-up considerations. [PDF]
Ladd B, Gräslund T, Chotteau V.
europepmc +1 more source