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

Enhancement of mouse hematopoietic stem/progenitor cell function via transient gene delivery using integration-deficient lentiviral vectors

利用整合缺陷型慢病毒载体进行瞬时基因传递增强小鼠造血干细胞/祖细胞功能

Maria E Alonso-Ferrero, Niek P van Til, Kerol Bartolovic, Márcia F Mata, Gerard Wagemaker, Dale Moulding, David A Williams, Christine Kinnon, Simon N Waddington, Michael D Milsom, Steven J Howe

Eliminating HIV-1 Packaging Sequences from Lentiviral Vector Proviruses Enhances Safety and Expedites Gene Transfer for Gene Therapy

消除慢病毒载体前病毒中的 HIV-1 包装序列可提高安全性并加快基因治疗的基因转移

Conrad A Vink, John R Counsell, Dany P Perocheau, Rajvinder Karda, Suzanne M K Buckley, Martijn H Brugman, Melanie Galla, Axel Schambach, Tristan R McKay, Simon N Waddington, Steven J Howe

Targeted genome editing restores T cell differentiation in a humanized X-SCID pluripotent stem cell disease model

靶向基因组编辑在人源化 X-SCID 多能干细胞疾病模型中恢复 T 细胞分化

Jamal Alzubi, Celeste Pallant, Claudio Mussolino, Steven J Howe, Adrian J Thrasher, Toni Cathomen

Minicircle DNA Provides Enhanced and Prolonged Transgene Expression Following Airway Gene Transfer

微环 DNA 在气道基因转移后提供增强和延长的转基因表达

Mustafa M Munye, Aristides D Tagalakis, Josephine L Barnes, Rachel E Brown, Robin J McAnulty, Steven J Howe, Stephen L Hart

Systemic gene delivery following intravenous administration of AAV9 to fetal and neonatal mice and late-gestation nonhuman primates

向胎儿和新生小鼠以及妊娠晚期非人类灵长类动物静脉注射 AAV9 后进行全身基因传递

Citra N Mattar, Andrew M S Wong, Klemens Hoefer, Maria E Alonso-Ferrero, Suzanne M K Buckley, Steven J Howe, Jonathan D Cooper, Simon N Waddington, Jerry K Y Chan, Ahad A Rahim

In vivo bioimaging with tissue-specific transcription factor activated luciferase reporters

利用组织特异性转录因子激活的荧光素酶报告基因进行体内生物成像

Suzanne M K Buckley, Juliette M K M Delhove, Dany P Perocheau, Rajvinder Karda, Ahad A Rahim, Steven J Howe, Natalie J Ward, Mark A Birrell, Maria G Belvisi, Patrick Arbuthnot, Mark R Johnson, Simon N Waddington, Tristan R McKay

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

插入诱变与获得性体细胞突变相结合导致 SCID-X1 患者在接受基因治疗后出现白血病

Steven J Howe, Marc R Mansour, Kerstin Schwarzwaelder, Cynthia Bartholomae, Michael Hubank, Helena Kempski, Martijn H Brugman, Karin Pike-Overzet, Stephen J Chatters, Dick de Ridder, Kimberly C Gilmour, Stuart Adams, Susannah I Thornhill, Kathryn L Parsley, Frank J T Staal, Rosemary E Gale, David C

Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency

自灭活γ-逆转录病毒载体用于X连锁严重联合免疫缺陷症的基因治疗

Susannah I Thornhill, Axel Schambach, Steven J Howe, Meera Ulaganathan, Elke Grassman, David Williams, Bernhard Schiedlmeier, Neil J Sebire, H Bobby Gaspar, Christine Kinnon, Christopher Baum, Adrian J Thrasher

Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells

含有无增强子的普遍作用染色质开放元件 (UCOE) 的慢病毒载体可在造血细胞中提供高度可重复和稳定的转基因表达

Fang Zhang, Susannah I Thornhill, Steven J Howe, Meera Ulaganathan, Axel Schambach, Joanna Sinclair, Christine Kinnon, H Bobby Gaspar, Michael Antoniou, Adrian J Thrasher

Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo

γ 逆转录病毒介导的 SCID-X1 纠正与体内载体整合位点分布不均有关

Kerstin Schwarzwaelder, Steven J Howe, Manfred Schmidt, Martijn H Brugman, Annette Deichmann, Hanno Glimm, Sonja Schmidt, Claudia Prinz, Manuela Wissler, Douglas J S King, Fang Zhang, Kathryn L Parsley, Kimberly C Gilmour, Joanna Sinclair, Jinhua Bayford, Rachel Peraj, Karin Pike-Overzet, Frank J T