Results 121 to 130 of about 73,809 (216)

Engineered Transformer Base Editor with Enhanced Editing Efficiency

Advanced Science, EarlyView.
A highly efficient transformer base editor system achieves robust genomic editing in a humanized mouse model. This work establishes a versatile and translatable platform, opening new avenues for precision gene therapy. ABSTRACT Canonical cytosine base editors (CBEs) achieve precise C‐to‐T conversions without inducing DNA double‐strand breaks (DSBs ...
Bowen Chen, Letong Liang, Rui Xu, Tianyi Wang, Hongyan Chen, Daru Lu   +5 more
wiley   +1 more source

Gene Therapy Strategies for Uveal Melanoma: Adeno-associated Virus Delivery Challenges and Translational Opportunities. [PDF]

BioDrugs
Wilson RJ, Ramlogan-Steel CA, Deasy WJ, Greenstein SJ, Layton CJ, Steel JC.   +5 more
europepmc   +1 more source

FTSJ3‐Mediated 2′‐O‐Methylation of HSPA5 mRNA Stabilizes BOP1 via SUMOylation to Drive DRP1‐Dependent Mitochondrial Fission and Fatty Acid Oxidation in Lung Squamous Cell Carcinoma

Advanced Science, EarlyView.
The RNA methyltransferase FTSJ3 drives lung squamous cell carcinoma progression by catalyzing 2′‐O‐methylation of HSPA5 mRNA, which stabilizes BOP1 through SUMOylation and activates DRP1/SREBP1‐mediated mitochondrial fission and fatty acid oxidation. Targeting this axis with liposomal siRNA potently suppresses tumor growth and metastasis ABSTRACT Lung ...
Qin Hu, Jiaheng Lin, Yifei Zhu, Ying Liu, Guoren Zhou   +4 more
wiley   +1 more source

Adeno-Associated Virus Vector Mediated Gene Therapy: A Promising Approach to Transform Hypertrophic Cardiomyopathy Treatment. [PDF]

Biotechnol J
Siddiqui S, Parab SB, Bhatt LK.
europepmc   +1 more source

The GPX3‐VCAM1 Axis Gates Pro‐Fibrotic Tubule Cell Fate in Hyperuricemic Nephropathy

Advanced Science, EarlyView.
Hyperuricemia induces NLRP3 inflammasome signaling activation and downregulation of GPX3 in proximal tubular epithelial cells, leading to oxidative stress–driven VCAM1 upregulation, emergence of pro‐fibrotic proximal tubule cells, macrophage recruitment, and renal fibrosis.
Yunfei Qi, Qiang Zhao, Yue Lve, Yaming Yuan, Tingting Zhou, Yue Yuan, Dexi Wu, Yifan Tang, Yan Liu, Zhigang Li, Chunping Jiang, Qiurong Ding   +11 more
wiley   +1 more source

Adeno-Associated virus-based approaches for mitochondrial diseases: advances and challenges. [PDF]

Mol Psychiatry
Corrà S, Balmaceda V, Viscomi C.
europepmc   +1 more source

CRISPR and Gene Augmentation Rescue Trabecular Meshwork Dysfunction in iPSC Models of Lowe Syndrome

Advanced Science, EarlyView.
By modeling Lowe syndrome using patient‐derived iPSCs, this study establishes a human disease model that faithfully recapitulates OCRL deficiency‐associated ciliary and cytoskeletal defects. The model enables evaluation of both mutation‐agnostic DNA augmentation and CRISPR‐mediated mutation correction strategies, both of which restore OCRL function and
Siyu Chen, Zhiquan Liu, Wenmin Wang, Qing Wang, Tia J. Kowal, Fan Zhang, Grzegorz Walkiewicz, Faruk Hossen, Herbert M. Lachman, Jinqiong Zhou, Yiting Wang, Yang Sun   +11 more
wiley   +1 more source

An Expanded Toolbox for Versatile Chemical Editing of Adeno-Associated Virus. [PDF]

Angew Chem Int Ed Engl
Pham Q, Glicksman J, Han B, Koo D, Loynd C, Singha Roy SJ, Chatterjee A.   +6 more
europepmc   +1 more source

Palmitoyl Acyltransferase Zdhhc17 Promotes Functional Recovery After Spinal Cord Injury by Targeting the Nuclear Transport Factors Kpna2 and Ipo9

Advanced Science, EarlyView.
In this study, we demonstrate that Zdhhc17, acting as a PAT, suppresses neuronal apoptosis and promotes axon regeneration after injury, thereby enhancing functional recovery following SCI. In this context, Kpna2 and Ipo9 serve as novel palmitoylation substrates of Zdhhc17, whose palmitoylation prevents the injury‐induced degradation of their proteins ...
Meixuan Chen, Huan Chen, Sining Ding, Fan Liu, Xiaofei Zheng, Yuan Wang, Ruitu Tian, Huan Li, Peilin Liu, Le Hu, Bin Liu, Limin Rong, Mangmang Li   +12 more
wiley   +1 more source

Anion-exchange chromatography separates structurally heterogeneous and low-potency particles in adeno-associated virus manufacture. [PDF]

Mol Ther Adv
Tsunaka Y, Makihira H, Kono H, Yang Z, Lyu X, Soth S, Fukuhara M, Shibuya R, Yamaguchi Y, Uchiyama S.   +9 more
europepmc   +1 more source