日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

AAV-mediated exon skipping therapy for Usher syndrome, type 2A.

AAV介导的Usher综合征2A型外显子跳跃疗法。

Mauriac Stephanie A, Lee Jiyoon, Zhang Jingyuan, Jin Jiahe, Nist-Lund Carl, Martin Camille T, Von Muhlenbrock Cristobal R, O'Malley Sydney, Chaves Mary G, Madison Margot A, Foster Kate, Long Shinong, Jacobs Chris, Kumar Sripriya Ravindra, Ramirez Silvia, Ichida Justin, Koehler Karl R, Holt Jeffrey R, Géléoc Gwenaëlle S G

Spatially patterned kidney assembloids recapitulate progenitor self-assembly and enable high-fidelity in vivo disease modeling.

空间模式化的肾脏组装体重现了祖细胞的自组装过程,并实现了高保真度的体内疾病建模。

Huang Biao, Medina Pedro, He Jincan, Zeng Zipeng, Kim Sunghyun, Romo Janet, Koppitch Kari, Zhang Chennan C, Gyarmati Georgina, Park Yohan, Bohovyk Ruslan, N'Guetta Pierre-Emmanuel Yoann, Guo Jinjin, Ma Tianyi, Schreiber Megan E, Xu Cong, Pham Jessica, Parvez Riana K, Su Jackson, Xia Mateo W, Liu Zhenqing, Perin Laura, El-Nachef Danny, Murry Charles E, Kumar Sanjeev, O'Brien Lori, Zimmerman Kurt A, Ichida Justin, Lindström Nils O, Pastor-Soler Nuria M, Hallows Kenneth R, Staruschenko Alexander, Peti-Peterdi Janos, Jiang Cizhong, McMahon Andrew P, Li Zhongwei

The Ku80-p53-SIRT1 axis in DNA damage response contributes to sporadic and familial ALS and FTD.

Ku80-p53-SIRT1轴在DNA损伤反应中与散发性和家族性ALS和FTD有关。

Jun Yong-Woo, Lee Soojin, Almeida Sandra, Freude Kristine K, Ichida Justin K, Gao Fen-Biao

Human iPSC-Derived Motor Neuron Innervation Enhances the Differentiation of Muscle Bundles Engineered with Benchtop Fabrication Techniques.

人类 iPSC 衍生的运动神经元支配增强了利用台式制造技术构建的肌肉束的分化

Santoso Jeffrey W, Do Stephanie K, Verma Riya, Do Alexander V, Hendricks Eric, Ichida Justin K, McCain Megan L

Elucidating the Synergistic Effect of the PrimeC Combination for Amyotrophic Lateral Sclerosis in Human Induced Pluripotent Stem Cell-Derived Motor Neurons and Mouse Models

阐明PrimeC联合疗法对人诱导多能干细胞衍生运动神经元和鼠模型中肌萎缩侧索硬化症的协同作用

Salomon-Zimri, Shiran; Kerem, Nitai; Linares, Gabriel R; Russek-Blum, Niva; Ichida, Justin K; Tracik, Ferenc

Elevated tripeptidyl-peptidase 1 corrects multiple disease phenotypes in a mouse model of juvenile neuronal ceroid lipofuscinosis

三肽基肽酶 1 水平升高可纠正幼年神经元蜡样脂褐质沉积症小鼠模型中的多种疾病表型

Banach-Petrosky, Whitney; Larrimore, Katherine E; Sleat, Elizabeth H; Bazer, Allyson; Samuels, Benjamin; Tan, Yalun; Melton, Andrew C; Ichida, Justin K; Logan, Todd P; Lobel, Peter; Sleat, David E

An ALS assembly modulator signature in peripheral blood mononuclear cells: implications for ALS pathophysiology, therapeutics, and diagnostics.

外周血单核细胞中 ALS 组装调节因子的特征:对 ALS 病理生理学、治疗和诊断的意义

Yu Shao Feng, Michon Maya, Lingappa Anuradha F, Paulvannan Kumar, Solas Dennis, Staats Kim, Ichida Justin, Dey Debendranath, Rosenfeld Jeffrey, Lingappa Vishwanath R

Protein Assembly Modulation: A New Approach to Amyotrophic Lateral Sclerosis (ALS) Therapeutics

蛋白质组装调控:肌萎缩侧索硬化症(ALS)治疗的新方法

Yu, Shao Feng; Paulvannan, Kumar; Solas, Dennis; Lingappa, Anuradha F; Moreira, Ana Raquel; Sahu, Shriya; Michon, Maya; Macieik, Amanda; Goldsmith, Danielle; DeYarman, Nicholas; Mallesh, Suguna; Prasad, M Dharma; Maios, Claudia; Ruan, Kai; Tomassy, Giulio S; Jensen, Elizabeth; McGuirk, Emma; Bader, Verian; Mueller-Schiffmann, Andreas; Reed, Jonathan C; Lingappa, Jaisri R; Asundi, Vinod; Hong, Shi; Jacobsen, Steve; Brandon, Nicholas; Ostrow, Lyle; Lloyd, Tom; Parker, J Alex; Staats, Kim A; Ichida, Justin; Dodge, James C; Dey, Debendranath; Korth, Carsten; Selvarajah, Suganya; Lingappa, Vishwanath R; Rosenfeld, Jeffrey

Human iPSC-derived motor neuron innervation enhances the differentiation of muscle bundles engineered with benchtop fabrication techniques.

人类 iPSC 衍生的运动神经元支配增强了利用台式制造技术构建的肌肉束的分化

Santoso Jeffrey W, Do Stephanie K, Verma Riya, Do Alexander V, Hendricks Eric, Ichida Justin K, McCain Megan L

Molecular Tweezers: Supramolecular Hosts with Broad-Spectrum Biological Applications

分子镊子:具有广泛生物学应用的超分子宿主

Shahpasand-Kroner, Hedieh; Siddique, Ibrar; Malik, Ravinder; Linares, Gabriel R; Ivanova, Magdalena I; Ichida, Justin; Weil, Tatjana; Münch, Jan; Sanchez-Garcia, Elsa; Klärner, Frank-Gerrit; Schrader, Thomas; Bitan, Gal