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

Authors: 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

Source: Stem Cell Research & Therapy, Vol 15, Iss 1, Pp 1-19 (2024)

Subject Terms: β-hemoglobinopathies, Gene therapy, Hematopoietic stem cells, Homology-directed gene editing, Single-stranded oligonucleotides, Medicine (General), R5-920, Biochemistry, QD415-436

File Description: electronic resource

Relation: https://doaj.org/toc/1757-6512

Access URL: https://doaj.org/article/b32aa20255e7453e8753d8e3cc21811a

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

Authors: 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

Source: Molecular Therapy: Nucleic Acids, Vol 32, Iss , Pp 671-688 (2023)

Subject Terms: MT: RNA/DNA Editing, deletional HPFH, sickle cell diseases, beta-thalassemia, gene editing, gene therapy, Therapeutics. Pharmacology, RM1-950

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2162253123001105; https://doaj.org/toc/2162-2531

Access URL: https://doaj.org/article/444686798a7c40d7a355be2ff089b162

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

Authors: 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

Source: Cells, Vol 10, Iss 3, p 523 (2021)

Subject Terms: erythroid, differentiation, immortalization, peripheral blood, HPV E6/E7, Cytology, QH573-671

File Description: electronic resource

Relation: https://www.mdpi.com/2073-4409/10/3/523; https://doaj.org/toc/2073-4409

Access URL: https://doaj.org/article/cc913110f5c44221bd81e68c4de7504b