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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.
Chandraprabha PB, Azhagiri MKK, Venkatesan V, Magis W, Prasad K, Suresh S, Pai AA, Marepally S, Srivastava A, Mohankumar KM, Martin DIK, Thangavel S. Chandraprabha PB, et al. Among authors: azhagiri mkk. Stem Cell Res Ther. 2024 Dec 31;15(1):504. doi: 10.1186/s13287-024-04117-0. Stem Cell Res Ther. 2024. PMID: 39736768 Free PMC article.
Editing the core region in HPFH deletions alters fetal and adult globin expression for treatment of β-hemoglobinopathies.
Venkatesan V, Christopher AC, Rhiel M, Azhagiri MKK, Babu P, Walavalkar K, Saravanan B, Andrieux G, Rangaraj S, Srinivasan S, Karuppusamy KV, Jacob A, Bagchi A, Pai AA, Nakamura Y, Kurita R, Balasubramanian P, Pai R, Marepally SK, Mohankumar KM, Velayudhan SR, Boerries M, Notani D, Cathomen T, Srivastava A, Thangavel S. Venkatesan V, et al. Among authors: azhagiri mkk. Mol Ther Nucleic Acids. 2023 Apr 26;32:671-688. doi: 10.1016/j.omtn.2023.04.024. eCollection 2023 Jun 13. Mol Ther Nucleic Acids. 2023. PMID: 37215154 Free PMC article.
Preferential Expansion of Human CD34+CD133+CD90+ Hematopoietic Stem Cells Enhances Gene-Modified Cell Frequency for Gene Therapy.
Christopher AC, Venkatesan V, Karuppusamy KV, Srinivasan S, Babu P, Azhagiri MKK, Chambayil K, Bagchi A, Rajendiran V, Ravi NS, Kumar S, Marepally SK, Mohankumar KM, Srivastava A, Velayudhan SR, Thangavel S. Christopher AC, et al. Among authors: azhagiri mkk. Hum Gene Ther. 2022 Feb;33(3-4):188-201. doi: 10.1089/hum.2021.089. Epub 2022 Jan 7. Hum Gene Ther. 2022. PMID: 34486377