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Molecular Therapy. Nucleic Acids|March 10, 2025
Potential ASO-based personalized treatment for Charcot-Marie-Tooth disease type 2SSandra Smieszek, Bartlomiej Przychodzen, Christina Tyner, et al.
Molecular Therapy. Nucleic Acids|December 6, 2024
Efficient and in situ correction of hemoglobin Constant Spring mutation by prime editing in human hematopoietic cellsCongwen Shao, Qing Liu, Jinchao Xu, et al.
Molecular Therapy. Nucleic Acids|December 6, 2024
Discovery of peptides for ligand-mediated delivery of mRNA lipid nanoparticles to cystic fibrosis lung epitheliaMelissa R Soto, Mae M Lewis, Jasmim Leal, et al.
Molecular Therapy. Nucleic Acids|December 17, 2024
AAV-regulated Serpine2 overexpression promotes hair cell regenerationQiuhan Sun, Fangzhi Tan, Xinlin Wang, et al.
Molecular Therapy. Nucleic Acids|December 24, 2024
CHD6 eviction of promoter nucleosomes maintains housekeeping transcriptional program in prostate cancerLina Bu, Shaodong Huang, Ziyan Rao, et al.
Molecular Therapy. Nucleic Acids|December 10, 2024
A novel hyperactive variant of the Sleeping Beauty transposase facilitates non-viral genome engineeringMatthias Thomas Ochmann, Csaba Miskey, Lacramioara Botezatu, et al.
Molecular Therapy. Nucleic Acids|November 19, 2024
Rationally designed Campylobacter jejuni Cas9 enables efficient gene activation and base editingYuxi Chen, Rui Kang, Yuanling Jiang, et al.
Molecular Therapy. Nucleic Acids|November 19, 2024
Erratum: Inhaled aptamer therapy: A hopeful therapy for lung diseaseM Elena Martin
Molecular Therapy. Nucleic Acids|November 18, 2024
Profiling patent compounds in lipid nanoparticle formulations of siRNAYunfeng Han, Mengyang Wang, Yu Chen, et al.
Molecular Therapy. Nucleic Acids|November 4, 2024
Lipopeptide-mediated Cas9 RNP delivery: A promising broad therapeutic strategy for safely removing deep-intronic variants in ABCA4Irene Vázquez-Domínguez, Mert Öktem, Florian A Winkelaar, et al.
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