Accès ouvert
2026
article
OpenAlex
Kjell Sandstrom, Grace N. Hagedorn, Sean C. Robinson, Julia E. Katz et autres
Natural killer (NK) cells are key innate effectors during antiviral immune responses, with their activity regulated in part by interactions between polymorphic killer-cell immunoglobulin-like receptors (KIRs) on NK cells and their major histocompatibility complex class I (MHC I) ligands on target cells. …
us, it
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Accès ouvert
2026
article
OpenAlex
Fang Wan, Jinchen He, Hong Ma, Debora PiresFerreira et autres
Abstract Adeno-associated virus (AAV)-mediated gene silencing offers a promising strategy for achieving durable therapeutic effects with a single administration. Mutations in the human superoxide dismutase 1 ( h SOD1) gene, inherited in an autosomal dominant manner, lead to motor neuron degeneration in …
us
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Accès ouvert
2026
article
OpenAlex
Aijaz Ahmad John, Yeon-Suk Yang, Jun Xie, Sachin Chaugule et autres
Chondrodysplasia is a genetic disorder characterized by impaired cartilage development and bone growth. Dysregulation of the endoplasmic reticulum (ER) stress is associated with chondrodysplasia. Here, we demonstrate a critical role for the ER stress regulator PPP1R15B in chondrocyte development. PPP1R15B is a …
us, fr, kr
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Accès ouvert
2026
article
OpenAlex
Jung-Min Kim, Yeon-Suk Yang, XIE Jun, Chan Mi Kang et autres
Osteoblast development must be precisely regulated, as insufficient bone formation results in low bone mass and skeletal fragility, whereas excessive osteogenesis drives heterotopic ossification (HO), the ectopic formation of bone in soft tissues. Here, we identify the deubiquitinating enzyme ubiquitin-specific peptidase 24 …
kr, us
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Accès ouvert
2026
article
OpenAlex
Peiyi Guo, Elisabet C. Mandon, Jie Wang, Morgan Mooney et autres
Oligodendrocytes are central to myelination and are key targets in myelin disease research and gene therapy. We optimized magnetic isolation of O4 + oligodendrocytes from adult mouse brain and PDGFRα + oligodendrocyte precursor cells from neonatal brain, and evaluated purity, viability, maturation, …
us, cn
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Accès ouvert
2026
article
OpenAlex
Siping Zhu, Hong Li, Chihuan Li, Xintong Zhu et autres
Background: Infectious bovine rhinotracheitis virus (IBRV) is a primary pathogen causing bovine respiratory disease syndrome. This virus can cause rhinotracheitis and vaginitis in cattle, resulting in high mortality and posing a serious threat to bovine production. MicroRNAs (miRNAs), a class of regulatory …
cn
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Accès ouvert
2025
article
OpenAlex
Philippe Godin, Nicholas G. Nagykery, Natalie Sicher, Julie L. Barnes et autres
The uncontrolled reproduction of free-roaming domestic cats exacerbates their welfare challenges and the ecological pressure they exert on wildlife populations. Because of logistic and economic constraints, surgical sterilization alone cannot scale to control the reproduction of the hundreds of millions of intact …
us
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Accès ouvert
2025
preprint
OpenAlex
Denise Zhong, Ioanna Stavrakaki, Tiffany DeSouza, Sabine Linskey et autres
gene, which encodes the protein seipin. However, how seipin loss causes adipose tissue failure remains unclear. Using human adipocyte progenitor cells capable of robust differentiation in vitro and in vivo, we reveal two unexpected findings that redefine CGL2 pathogenesis. First, seipin is …
us, in, ca
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Accès ouvert
2025
article
OpenAlex
Sebastian P. Fuchs, P.G. Mondragon, Rachel Zabizhin, Shallu Tomer et autres
Long-term delivery of broadly neutralizing antibodies (bnAbs) using adeno-associated virus (AAV) vector is a promising approach for both the prevention and treatment of HIV infection. However, host anti-drug antibody (ADA) responses severely limit the continuous delivery of these anti-HIV bnAbs and have …
us
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Accès ouvert
2025
article
OpenAlex
Toloo Taghian, Jillian Gallagher, Stephanie Bertrand, William C. Baker et autres
Tay-Sachs disease (TSD) and Sandhoff disease are fatal neurodegenerative diseases without an effective therapy that are caused by mutations in the HEXA and HEXB genes, respectively. Together they encode the heterodimeric isozyme of hexosaminidase, hexosaminidase A (HexA), that degrades GM2 ganglioside. This …
us
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Accès ouvert
2025
article
OpenAlex
Ruxiao Xing, Mengyao Xu, Darcy Reil, Mengtian Cui et autres
AAV capsid serotypes isolated from nature have been widely used in gene delivery and gene therapy. Recently, more than 1,000 distinct AAV capsids were identified from human clinical samples by high-throughput, long-read DNA sequencing. In this study, we tap into this broad …
us, cn
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Accès ouvert
2025
article
OpenAlex
Yeon-Suk Yang, Mi‐Jeong Kim, Sachin R. Chaugule, Ngoc Ho DeSouza et autres
The interleukin (IL)-1 pathway is a key mediator of inflammation and innate immune responses. Its dysregulation contributes to rheumatoid arthritis (RA) and autoinflammatory diseases (AIDs). In this study, we develop a recombinant adeno-associated virus (rAAV)-based gene therapy to deliver an inflammation-inducible, secreted …
us, kr
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