Diosgenin enhances liposome-enabled nucleic acid delivery and CRISPR/Cas9-mediated gene editing by modulating endocytic pathways by 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

Get full text

Enhanced fetal hemoglobin production via dual-beneficial mutation editing of the HBG promoter in hematopoietic stem and progenitor cells for β-hemoglobinopathies by 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

Get full text

Direct Generation of Immortalized Erythroid Progenitor Cell Lines from Peripheral Blood Mononuclear Cells by 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

Get full text

Precise correction of a spectrum of β-thalassemia mutations in coding and non-coding regions by base editors by 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

Get full text

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

Get full text