In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of -thalassemia-Reference-Cited by-同舟云学术

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In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of -thalassemia

Published:2008-07-23 Issue:30 Volume:105 Page:10547-10552
ISSN:0027-8424
Container-title:Proceedings of the National Academy of Sciences
language:en
Short-container-title:Proceedings of the National Academy of Sciences

Author:

Miccio A.,Cesari R.,Lotti F.,Rossi C.,Sanvito F.,Ponzoni M.,Routledge S. J. E.,Chow C.-M.,Antoniou M. N.,Ferrari G.

Publisher

Proceedings of the National Academy of Sciences

Subject

Multidisciplinary

Reference35 articles.

1. The cure of thalassemia by bone marrow transplantation

2. Permanent and panerythroid correction of murine thalassemia by multiple lentiviral integration in hematopoietic stem cells

3. Successful correction of the human β-thalassemia major phenotype using a lentiviral vector

4. Recent advances in globin gene transfer for the treatment of beta-thalassemia and sickle cell anemia

5. Sea urchin insulator protects lentiviral vector from silencing by maintaining active chromatin structure

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1. Cellular and animal models for the investigation of β-thalassemia;Blood Cells, Molecules, and Diseases;2024-01

2. Evaluation of Mono- and Bi-Functional GLOBE-Based Vectors for Therapy of β-Thalassemia by HBBAS3 Gene Addition and Mutation-Specific RNA Interference;Cells;2023-12-15

3. Gene Therapy for Hemoglobinopathies;Human Gene Therapy;2023-09-01

4. In vivo hematopoietic stem cell modification by mRNA delivery;Science;2023-07-28

5. Precision Editing as a Therapeutic Approach for β-Hemoglobinopathies;International Journal of Molecular Sciences;2023-05-31

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