Splicing correction by peptide-conjugated morpholinos as a novel treatment for late-onset Pompe disease.
利用肽偶联吗啉代寡核苷酸进行剪接校正，作为治疗晚发型庞贝病的新方法
期刊:Molecular Therapy-Nucleic Acids
影响因子:6.1
doi:10.1016/j.omtn.2025.102524
Oliver Ryan A, Ahern Meghan E, Castaneda Perla G, Jinadasa Tushare, Bardhan Anirban, Morgan Kathy Y, Ha Kristin, Adhikari Kailash, Jungels Nino, Liberman Noa, Mitra Anindita, Greer Christopher D, Wright Alec M, Thompson Emily G, Garcia Stephanie, Copson Elena, Allu Senkara, Tan Xuyu, Callahan Alex J, Cai Bao Zhong, Guerlavais Vincent, Kim Kevin J, Malmberg Annika B
