Short-hairpin RNA against aberrant HBBIVSI-110(G>A) mRNA restores β-globin levels in a novel cell model and acts as mono- and combination therapy for β-thalassemia in primary hematopoietic stem cells

Ижил төстэй зүйлс

• Short-hairpin RNA against aberrant HBBIVSI-110(G>A) mRNA restores β-globin levels in a novel cell model and acts as mono- and combination therapy for β-thalassemia in primary hematopoietic stem cells
  -н: Petros Patsali, зэрэг
  Хэвлэсэн: (2018-09-01)
• Context base editing for splice correction of IVSI-110 β-thalassemia
  -н: Basma Naiisseh, зэрэг
  Хэвлэсэн: (2024-06-01)
• Evaluation of Mono- and Bi-Functional GLOBE-Based Vectors for Therapy of β-Thalassemia by <i>HBB^AS3</i> Gene Addition and Mutation-Specific RNA Interference
  -н: Lola Koniali, зэрэг
  Хэвлэсэн: (2023-12-01)
• Functional correction and genome integrity with duplex base editing of β-thalassemic hematopoietic stem cells
  -н: Nikoletta Y. Papaioannou, зэрэг
  Хэвлэсэн: (2026-02-01)
• Unravelling the Complexity of the +33 C>G [HBB:c.-18C>G] Variant in Beta Thalassemia
  -н: Coralea Stephanou, зэрэг
  Хэвлэсэн: (2024-01-01)