Editing the core region in HPFH deletions alters fetal and adult globin expression for treatment of β-hemoglobinopathies
通过编辑HPFH缺失的核心区域，可以改变胎儿和成人珠蛋白的表达，用于治疗β-血红蛋白病。
期刊:Molecular Therapy-Nucleic Acids
影响因子:6.5
doi:10.1016/j.omtn.2023.04.024
Vigneshwaran Venkatesan,Abisha Crystal Christopher,Manuel Rhiel,Manoj Kumar K Azhagiri,Prathibha Babu,Kaivalya Walavalkar,Bharath Saravanan,Geoffroy Andrieux,Sumathi Rangaraj,Saranya Srinivasan,Karthik V Karuppusamy,Annlin Jacob,Abhirup Bagchi,Aswin Anand Pai,Yukio Nakamura,Ryo Kurita,Poonkuzhali Balasubramanian,Rekha Pai,Srujan Kumar Marepally,Kumarasamypet Murugesan Mohankumar,Shaji R Velayudhan,Melanie Boerries,Dimple Notani,Toni Cathomen,Alok Srivastava,Saravanabhavan Thangavel
