Gene Therapy for Hemoglobinopathies
Author:
Affiliation:
1. San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), San Raffaele Scientific Institute, Milan, Italy; and
2. University Vita-Salute San Raffaele, Milan, Italy.
Publisher
Mary Ann Liebert Inc
Subject
Genetics,Molecular Biology,Molecular Medicine
Link
https://www.liebertpub.com/doi/pdf/10.1089/hum.2023.138
Reference137 articles.
1. Structure and function of haemoglobin
2. Human Fetal Hemoglobin Expression Is Regulated by the Developmental Stage-Specific Repressor BCL11A
3. Transcription factors LRF and BCL11A independently repress expression of fetal hemoglobin
4. Effective therapies for sickle cell disease: are we there yet?
5. Hematopoietic stem cell function in β-thalassemia is impaired and is rescued by targeting the bone marrow niche
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1. Updates in Pediatric Sickle Cell Lung Disease;Clinics in Chest Medicine;2024-09
2. Hematopoietic Stem Cell Transplantation in Children with Sickle Cell Disease and Thalassemia Major: A National Database Study;Pediatric Hematology and Oncology;2024-07-15
3. An update review of new therapies in sickle cell disease: the prospects for drug combinations;Expert Opinion on Pharmacotherapy;2024-01-22
4. Therapeutic Effects of Hematopoietic Stem Cell Derived From Gene-Edited Mice on β654-Thalassemia;Stem Cells;2023-12-22
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