Results 31 to 40 of about 110,488 (256)

Intrabody Gene Therapy Ameliorates Motor, Cognitive, and Neuropathological Symptoms in Multiple Mouse Models of Huntington's Disease [PDF]

, 2009
Huntington's disease (HD) is an autosomal dominant neurodegenerative disease resulting from the expansion of a glutamine repeat in the huntingtin (Htt) protein.
Ko, Jan, Patterson, Paul H., Southwell, Amber L.   +2 more
core   +2 more sources

Circumventing antivector immunity: potential use of nonhuman adenoviral vectors [PDF]

, 2014
Adenoviruses are efficient gene delivery vectors based on their ability to transduce a wide variety of cell types and drive high-level transient transgene expression. While there have been advances in modifying human adenoviral (HAdV) vectors to increase
Beard C.W., Benkö M., Cauthen A.N., Crawford-Miksza L., Estrella Lopez-Gordo, Gall J., Gall J.G., Gattoni A., Ginsberg H.S., Haviv Y.S., Hofmann C., Iva I. Podgorski, Kajon A.E., Kanerva A., Kass-Eisler A., Kuriyama S., Mastrangeli A., Michou A.I., Nicholas Downes, Ramon Alemany, Rogozhin V.N., Roy S., Seiradake E., Shmarov M.M., Soudais C., Tutykhina I.L., Wang D.Y., Wohlfart C., Ying B., Zakhartchouk A.N., Zeng Q.   +30 more
core   +3 more sources

Increased risk for T cell autoreactivity to ß-cell antigens in the mice expressing the Avy obesity-associated gene. [PDF]

, 2019
There has been considerable debate as to whether obesity can act as an accelerator of type 1 diabetes (T1D). We assessed this possibility using transgenic mice (MIP-TF mice) whose ß-cells express enhanced green fluorescent protein (EGFP). Infecting these
Atkinson, Mark A, Dang, Hoa, Kaufman, Daniel L, Sun, Ren, Tian, Jide, Wu, Ting-Ting, Yong, Jing   +6 more
core   +2 more sources

Familial and genetic overlap between Sjögren’s disease and other autoimmune diseases

Frontiers in Immunology
PurposeTo evaluate familial clustering of Sjögren’s disease (SjD) with other autoimmune diseases and to characterize shared genetic architecture using an integrative genomic approach.MethodsMeta-analysis identified studies assessing autoimmune disease ...
Hitomi Ono-Minagi, Hitomi Ono-Minagi, Takuma Ohnishi, Natalie Atyeo, Kentaro Okuno, Peter D. Burbelo, John A. Chiorini   +6 more
doaj   +1 more source

Virus Binding and Internalization Assay for Adeno-associated Virus

Bio-Protocol, 2017
The binding and internalization of adeno-associated virus (AAV) is an important determinant of viral infectivity and tropism. The ability to dissect these two tightly connected cellular processes would allow better understanding and provide insight on ...
Garrett Berry, Victor Tse
doaj   +1 more source

Onasemnogene Abeparvovec in Type I Spinal Muscular Atrophy: 24‐Month Follow‐Up From the Italian Registry

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Onasemnogene abeparvovec (OA) is an AAV9‐based gene therapy for spinal muscular atrophy type I (SMA I). Real‐world outcomes show increased response variability compared to clinical trials, and follow‐up data beyond 12–18 months are limited.
Marika Pane, Giorgia Coratti, Chiara Cutrì, Antonio Varone, Riccardo Masson, Adele D'Amico, Valeria Sansone, Sonia Messina, Federica Ricci, Chiara Ticci, Claudio Bruno, Caterina Agosto, Francesca Benedetti, Antonella Pini, Sabrina Siliquini, Massimiliano Filosto, Alberto Zambon, Ilaria Bitetti, Maria Rosaria Manna, Claudia Dosi, Riccardo Zanin, Stefano Parravicini, Roberto De Sanctis, Giulia Stanca, Michela Catteruccia, Michele Tosi, Irene Mizzoni, Emilio Albamonte, Valentina Franchino, Maria Sframeli, Ilaria Cavallina, Elena Procopio, Michele Sacchini, Simone Morando, Noemi Brolatti, Federica Trucco, Gaia Scarpini, Elena Briganti, Beatrice Berti, Concetta Palermo, Daniela Leone, Stefano C. Previtali, Eugenio Mercuri, the ITASMAC working group   +43 more
wiley   +1 more source

Silencing of UTX Mitigates Aging-Associated Cardiac Fibrosis via Blocking Cardiac Fibro blaststo-Myofibroblasts Trans-Differentiation

Anatolian Journal of Cardiology, 2023
Background: Cardiac fibrosis increases with age. Fibroblast activation plays an essential role in cardiac fibrosis. Histone modifications are involved in various chromatin-dependent processes.
Chao Li, Tiantian Lin, Delin Li, Daoyuan Si, Huan Sun, Sibao Yang, Zhongfan Zhang, Qian Zhang, Kaiyao Shi   +8 more
doaj   +1 more source

A Versatile, Portable Intravital Microscopy Platform for Studying Beta-cell Biology In Vivo [PDF]

, 2019
The pancreatic islet is a complex micro-organ containing numerous cell types, including endocrine, immune, and endothelial cells. The communication of these systems is lost upon isolation of the islets, and therefore the pathogenesis of diabetes can only
Conteh, Abass M., Day, Richard N., Dunn, Kenneth W., Kamocka, Malgorzata M., Linnemann, Amelia K., Martinez, Michelle M., Mirmira, Raghavendra G., Orr, Kara S., Piñeros, Annie R., Reissaus, Christopher A., Tersey, Sarah A., Twigg, Ashley N.   +11 more
core   +1 more source

Artifact‐Minimizing Ultrathin Transparent Electrodes Fabricated via iCVD for In Vivo Optogenetic Stimulation and Neural Signal Monitoring of Primary Visual Cortex

Advanced Functional Materials, EarlyView.
We present ultrathin flexible transparent electrodes through iCVD‐enabled molecular control of 10 nm gold films on poly(dimethylaminomethylstyrene). In vivo validation demonstrated photoelectric artifact reduction vs. opaque electrodes and preservation of natural neural dynamics.
Tae Jin Mun, Minju Kim, Chaesung Kim, Yunyoung Choi, In‐Yeop Baek, Younghak Cho, Gyeongyong Lee, Yerim Jang, Min‐Ho Nam, Wonryung Lee, Maesoon Im, Hyejeong Seong   +11 more
wiley   +1 more source

Adeno-Associated Virus (AAV) Versus Immune Response

Viruses, 2019
Decades ago, Friedmann and Roblin postulated several barriers to gene therapy, including tissue targeting, delivery across the blood⁻brain barrier (BBB), and host immune responses. These issues remain pertinent till today.
Joseph Rabinowitz, Ying Kai Chan, Richard Jude Samulski   +2 more
doaj   +1 more source