Correction of Aberrant Pre-mRNA Splicing by Antisense Oligonucleotides in β-Thalassemia Egyptian Patients With IVSI-110 Mutation-Reference-Cited by-同舟云学术

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Correction of Aberrant Pre-mRNA Splicing by Antisense Oligonucleotides in β-Thalassemia Egyptian Patients With IVSI-110 Mutation

Published:2008-04 Issue:4 Volume:30 Page:281-284
ISSN:1077-4114
Container-title:Journal of Pediatric Hematology/Oncology
language:en
Short-container-title:

Author:

El-Beshlawy Amal,Mostafa Azza,Youssry Ilham,Gabr Hala,Mansour Iman M.,El-Tablawy Manar,Aziz Mona,Hussein Ibtissam R.

Publisher

Ovid Technologies (Wolters Kluwer Health)

Subject

Oncology,Hematology,Pediatrics, Perinatology and Child Health

Reference22 articles.

1. Phenotype-genotype relationships in monogenic disease: lessons from the thalassemias;Weatherall;Nat Rev Genet,2001

2. Thalassemia in Iran: epidemiology, prevention, and management;Abolghasemi;J Pediatr Hematol Oncol,2007

3. The role of fetal hemoglobin-enhancing agents in thalassemia;Lal;Semin Hematol,2004

4. Therapeutic options for patients with severe beta-thalassemia: the need for globin gene therapy;Sadelain;Hum Gene Ther,2007

5. Molecular therapies in beta-thalassaemia;Quek;Br J Haematol,2007

Cited by 17 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Precise correction of a spectrum of β-thalassemia mutations in coding and non-coding regions by base editors;Molecular Therapy - Nucleic Acids;2024-06

2. Context base editing for splice correction of IVSI-110 β-thalassemia;Molecular Therapy - Nucleic Acids;2024-06

3. 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

4. Genetic Manipulation Strategies for β-Thalassemia: A Review;Frontiers in Pediatrics;2022-06-15

5. Gene Editing-Based Technologies for Beta-hemoglobinopathies Treatment;Biology;2022-06-04

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