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

A systemically deliverable lipid-conjugated siRNA targeting DUX4 as an facioscapulohumeral muscular dystrophy therapeutic.

一种可系统递送的脂质偶联 siRNA,靶向 DUX4,作为面肩肱型肌营养不良症的治疗方法

Daman Katelyn, Yan Jing, Biscans Annabelle, Echeverria Dimas, Shmushkovich Taisia, Wolfson Alexey, Alterman Julia F, Khvorova Anastasia, Emerson Charles P Jr

On-Chip Photonic Detection Techniques for Non-Invasive In Situ Characterizations at the Microfluidic Scale

用于微流控尺度非侵入式原位表征的片上光子检测技术

Kurdadze, Tamar; Lamadie, Fabrice; Nehme, Karen A; Teychené, Sébastien; Biscans, Béatrice; Rodriguez-Ruiz, Isaac

Rational design of a JAK1-selective siRNA inhibitor for the modulation of autoimmunity in the skin

合理设计JAK1选择性siRNA抑制剂以调节皮肤自身免疫

Qi Tang ,Hassan H Fakih ,Mohammad Zain Ui Abideen ,Samuel R Hildebrand ,Khashayar Afshari ,Katherine Y Gross ,Jacquelyn Sousa ,Allison S Maebius ,Christina Bartholdy ,Pia Pernille Søgaard ,Malene Jackerott ,Vignesh Hariharan ,Ashley Summers ,Xueli Fan ,Ken Okamura ,Kathryn R Monopoli ,David A Cooper ,Dimas Echeverria ,Brianna Bramato ,Nicholas McHugh ,Raymond C Furgal ,Karen Dresser ,Sarah J Winter ,Annabelle Biscans ,Jane Chuprin ,Nazgol-Sadat Haddadi ,Shany Sherman ,Ümmügülsüm Yıldız-Altay ,Mehdi Rashighi ,Jillian M Richmond ,Claire Bouix-Peter ,Carine Blanchard ,Adam Clauss ,Julia F Alterman ,Anastasia Khvorova ,John E Harris

Single intravitreal administration of a tetravalent siRNA exhibits robust and efficient gene silencing in mouse and pig photoreceptors

单次玻璃体内注射四价 siRNA 可在小鼠和猪的光感受器中表现出强大而有效的基因沉默

Shun-Yun Cheng, Jillian Caiazzi, Annabelle Biscans, Julia F Alterman, Dimas Echeverria, Nicholas McHugh, Matthew Hassler, Samson Jolly, Delaney Giguere, Joris Cipi, Anastasia Khvorova, Claudio Punzo

Therapeutic Oligonucleotides: An Outlook on Chemical Strategies to Improve Endosomal Trafficking

治疗性寡核苷酸:改善内体运输的化学策略展望

Mangla, Priyanka; Vicentini, Quentin; Biscans, Annabelle

Divalent siRNAs are bioavailable in the lung and efficiently block SARS-CoV-2 infection

二价 siRNA 在肺中具有生物利用度,可有效阻断 SARS-CoV-2 感染

Vignesh N Hariharan, Minwook Shin, Ching-Wen Chang, Daniel O'Reilly, Annabelle Biscans, Ken Yamada, Zhiru Guo, Mohan Somasundaran, Qi Tang, Kathryn Monopoli, Pranathi Meda Krishnamurthy, Gitali Devi, Nicholas McHugh, David A Cooper, Dimas Echeverria, John Cruz, Io Long Chan, Ping Liu, Sun-Young Lim,

RNAi-based modulation of IFN-γ signaling in skin

基于RNAi的皮肤IFN-γ信号通路调控

Qi Tang ,Jacquelyn Sousa ,Dimas Echeverria ,Xueli Fan ,Ying-Chao Hsueh ,Khashayar Afshari ,Nicholas MeHugh ,David A Cooper ,Lorenc Vangjeli ,Kathryn Monopoli ,Ken Okamura ,Annabelle Biscans ,Adam Clauss ,John E Harris ,Anastasia Khvorova

Engineered ionizable lipid siRNA conjugates enhance endosomal escape but induce toxicity in vivo

工程化可电离脂质 siRNA 结合物可增强内体逃逸,但会在体内引起毒性

Annabelle Biscans, Socheata Ly, Nicholas McHugh, David A Cooper, Anastasia Khvorova

Chemical optimization of siRNA for safe and efficient silencing of placental sFLT1

siRNA 的化学优化,以安全有效地沉默胎盘 sFLT1

Sarah M Davis, Vignesh N Hariharan, Agnes Lo, Anton A Turanov, Dimas Echeverria, Jacquelyn Sousa, Nicholas McHugh, Annabelle Biscans, Julia F Alterman, S Ananth Karumanchi, Melissa J Moore, Anastasia Khvorova

PK-modifying anchors significantly alter clearance kinetics, tissue distribution, and efficacy of therapeutics siRNAs

PK 修饰锚定物可显著改变 siRNA 治疗的清除动力学、组织分布和疗效

Bruno M D C Godinho, Emily G Knox, Samuel Hildebrand, James W Gilbert, Dimas Echeverria, Zachary Kennedy, Reka A Haraszti, Chantal M Ferguson, Andrew H Coles, Annabelle Biscans, Jillian Caiazzi, Julia F Alterman, Matthew R Hassler, Anastasia Khvorova