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

mRNA trans-splicing dual AAV vectors for (epi)genome editing and gene therapy

用于(表观)基因组编辑和基因治疗的 mRNA 反式剪接双 AAV 载体

Lisa Maria Riedmayr, Klara Sonnie Hinrichsmeyer, Stefan Bernhard Thalhammer, David Manuel Mittas, Nina Karguth, Dina Yehia Otify, Sybille Böhm, Valentin Johannes Weber, Michael David Bartoschek, Victoria Splith, Manuela Brümmer, Raphael Ferreira, Nanda Boon, Gabriele Maria Wögenstein, Christian Grim

A gene therapy for inherited blindness using dCas9-VPR-mediated transcriptional activation

利用 dCas9-VPR 介导的转录激活治疗遗传性失明的基因治疗

Sybille Böhm, Victoria Splith, Lisa Maria Riedmayr, René Dominik Rötzer, Gilles Gasparoni, Karl J V Nordström, Johanna Elisabeth Wagner, Klara Sonnie Hinrichsmeyer, Jörn Walter, Christian Wahl-Schott, Stefanie Fenske, Martin Biel, Stylianos Michalakis, Elvir Becirovic