Results 91 to 100 of about 89,643 (298)
AAV and Insertional Mutagenesis
In this issue of Molecular Therapy, David Russell provides an invited commentary on data presented at the tenth annual meeting of the American Society of Gene Therapy that strongly suggested that adenovirus-associated virus (AAV) vector insertion could in certain models be linked to the development of cancer.1 A short report describing the data that ...
openaire +2 more sources
Evaluation of AAV-DJ vector for retinal gene therapy [PDF]
Purpose The most common virus vector used in gene therapy research for ophthalmologic diseases is the adeno-associated virus (AAV) vector, which has been used successfully in a number of preclinical and clinical studies. It is important to evaluate novel
Kenta Kobayashi +3 more
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Glucocorticoids transcriptionally activate LCN2 expression via GR nuclear translocation. Secreted LCN2 binds MMP9 to degrade skeletal muscle ECM collagen, blocks integrin‐mediated mechanotransduction, bidirectionally disrupts muscle protein homeostasis, and reveals a novel target for steroid‐induced muscle atrophy.
Hongwei Shi +11 more
wiley +1 more source
Proceedings of the Twenty-First Annual Meeting of the Berkeley Linguistics Society: General Session and Parasession on Historical Issues in Sociolinguistics/Social Issues in Historical Linguistics (1995)
Patricia Cukor-Avila, Guy Bailey
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Fzd7 Restrains Pink1‐Dependent Mitophagy in Suture Stem Cells to Maintain Cranial Suture Patency
How suture stem cells fail to preserve cranial suture patency remains incompletely understood. Integrated single‐cell and high‐resolution spatial transcriptomic analyses identify reduced Fzd7 expression in Prrx1+ suture stem cells as an early feature of craniosynostosis.
Xinyan Chen +10 more
wiley +1 more source
Viral vector-mediated RNA interference in the retina [PDF]
RNA interference (RNAi) is a highly conserved post-transcriptional gene silencing process triggered by double-stranded RNA (dsRNA) in eukaryotic cells.
Georgiadis, A.
core
Disturbed flow promotes the formation of TRIM21‐rich biomolecular droplets, which concentrate TRIM21 and PTPN14 and facilitate their SPRY‐FERM interaction (illustrated by the TRIM21 D355‐PTPN14 R132 salt bridge). This condensate‐driven proximity enables TRIM21 to catalyze K48‐linked polyubiquitination of PTPN14 at lysine 956, leading to proteasome ...
Xue He +10 more
wiley +1 more source
(a–d) Quantitative analysis of synapse-related genes in DG microdissected from rat hippocampus transfected with AAV-hGATA1 or AAV-hGATA2. Rab4b, AAV-hGATA1: F1,4 = 10.78, P = 0.03; AAV-hGATA2: F1,4 = 46.2, P = 0.002; PSD95, AAV-hGATA1: F1,4 = 31.73, P ...
Yong-Seok Kim (648731) +8 more
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Evading the AAV Immune Response in Mucopolysaccharidoses
The humoral immune response elicited by adeno-associated virus (AAV)-mediated gene therapy for the treatment of mucopolysaccharidoses (MPS) poses a significant challenge to achieving therapeutic levels of transgene expression.
Yasuhiko Ago +11 more
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Compact 9dBEs Enable Efficient and Precise Genome Editing in Mammalian Cells and In Vivo
As a compact type II‐D system, the Cas9d‐based platform holds great potential for in vivo applications. Through rational engineering, its derived base editors (9dBEs) enable efficient disease modeling while facilitating single‐vector AAV delivery for in vivo genome editing. These miniature tools offer a robust strategy for basic research and biomedical
Qingquan Xiao +12 more
wiley +1 more source

