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

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Diosgenin enhances liposome-enabled nucleic acid delivery and CRISPR/Cas9-mediated gene editing by modulating endocytic pathways ανά Brijesh Lohchania, Brijesh Lohchania, Abisha Crystal Christopher, Abisha Crystal Christopher, Porkizhi Arjunan, Porkizhi Arjunan, Gokulnath Mahalingam, Durga Kathirvelu, Aishwarya Prasannan, Vigneshwaran Venkatesan, Vigneshwaran Venkatesan, Pankaj Taneja, Mohan Kumar KM, Saravanabhavan Thangavel, Srujan Marepally

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Enhanced fetal hemoglobin production via dual-beneficial mutation editing of the HBG promoter in hematopoietic stem and progenitor cells for β-hemoglobinopathies ανά Prathibha Babu Chandraprabha, Manoj Kumar K. Azhagiri, Vigneshwaran Venkatesan, Wendy Magis, Kirti Prasad, Sevanthy Suresh, Aswin Anand Pai, Srujan Marepally, Alok Srivastava, Kumarasamypet Murugesan Mohankumar, David I. K. Martin, Saravanabhavan Thangavel

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Erythroid lineage-specific lentiviral RNAi vectors suitable for molecular functional studies and therapeutic applications ανά Abhirup Bagchi, Nivedhitha Devaraju, Karthik Chambayil, Vignesh Rajendiran, Vigneshwaran Venkatesan, Nilofer Sayed, Aswin Anand Pai, Aneesha Nath, Ernest David, Yukio Nakamura, Poonkuzhali Balasubramanian, Alok Srivastava, Saravanabhavan Thangavel, Kumarasamypet M. Mohankumar, Shaji R. Velayudhan

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Direct Generation of Immortalized Erythroid Progenitor Cell Lines from Peripheral Blood Mononuclear Cells ανά Abhirup Bagchi, Aneesha Nath, Vasanth Thamodaran, Smitha Ijee, Dhavapriya Palani, Vignesh Rajendiran, Vigneshwaran Venkatesan, Phaneendra Datari, Aswin Anand Pai, Nancy Beryl Janet, Poonkuzhali Balasubramanian, Yukio Nakamura, Alok Srivastava, Kumarasamypet Murugesan Mohankumar, Saravanabhavan Thangavel, Shaji R. Velayudhan

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Precise correction of a spectrum of β-thalassemia mutations in coding and non-coding regions by base editors ανά Kirti Prasad, Nivedhitha Devaraju, Anila George, Nithin Sam Ravi, Joshua Paul, Gokulnath Mahalingam, Vignesh Rajendiran, Lokesh Panigrahi, Vigneshwaran Venkatesan, Kartik Lakhotiya, Yogapriya Periyasami, Aswin Anand Pai, Yukio Nakamura, Ryo Kurita, Poonkuzhali Balasubramanian, Saravanabhavan Thangavel, Shaji R. Velayudhan, Gregory A. Newby, Srujan Marepally, Alok Srivastava, Kumarasamypet M. Mohankumar

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