Editing the core region in HPFH deletions alters fetal and adult globin expression for treatment of β-hemoglobinopathies

Reactivation of fetal hemoglobin (HbF) is a commonly adapted strategy to ameliorate β-hemoglobinopathies. However, the continued production of defective adult hemoglobin (HbA) limits HbF tetramer production affecting the therapeutic benefits. Here, we evaluated deletional hereditary persistence of f...

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Detalles Bibliográficos
Autores principales: Venkatesan, Vigneshwaran, Christopher, Abisha Crystal, Rhiel, Manuel, Azhagiri, Manoj Kumar K., Babu, Prathibha, Walavalkar, Kaivalya, Saravanan, Bharath, Andrieux, Geoffroy, Rangaraj, Sumathi, Srinivasan, Saranya, Karuppusamy, Karthik V., Jacob, Annlin, Bagchi, Abhirup, Pai, Aswin Anand, Nakamura, Yukio, Kurita, Ryo, Balasubramanian, Poonkuzhali, Pai, Rekha, Marepally, Srujan Kumar, Mohankumar, Kumarasamypet Murugesan, Velayudhan, Shaji R., Boerries, Melanie, Notani, Dimple, Cathomen, Toni, Srivastava, Alok, Thangavel, Saravanabhavan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10197010/
https://www.ncbi.nlm.nih.gov/pubmed/37215154
http://dx.doi.org/10.1016/j.omtn.2023.04.024

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