Homology-directed gene-editing approaches for hematopoietic stem and progenitor cell gene therapy by Manoj Kumar K. Azhagiri, Prathibha Babu, Vigneshwaran Venkatesan, Saravanabhavan Thangavel

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

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