Engineered U7 Small Nuclear RNA Restores Correct β-Globin Pre-mRNA Splicing in Mouse βIVS2-654-Thalassemic Erythroid Progenitor Cells-Reference-Cited by-同舟云学术

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Engineered U7 Small Nuclear RNA Restores Correct β-Globin Pre-mRNA Splicing in Mouse βIVS2-654-Thalassemic Erythroid Progenitor Cells

Published:2021-05-01 Issue:9-10 Volume:32 Page:473-480
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

d'Arqom Annette¹²³,Nualkaew Tiwaporn²,Jearawiriyapaisarn Natee²,Kole Ryszard⁴,Svasti Saovaros²⁵

Affiliation:

1. Graduate Program in Molecular Medicine

2. Thalassemia Research Center, Institute of Molecular Biosciences, Mahidol University, Nakhon Pathom, Thailand

3. Department of Pharmacology, Faculty of Medicine, Universitas Airlangga, Surabaya, Indonesia

4. Department of Pharmacology, University of North Carolina, Chapel Hill, North Carolina, USA.

5. Department of Biochemistry; Faculty of Science, Mahidol University, Bangkok, Thailand

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2020.145

Reference22 articles.

1. Thalassaemia

2. Thalassemia 2016: Modern medicine battles an ancient disease

3. Innovative Curative Treatment of Beta Thalassemia: Cost-Efficacy Analysis of Gene Therapy Versus Allogenic Hematopoietic Stem-Cell Transplantation

4. Gene therapy of hemoglobinopathies: progress and future challenges

5. beta-Thalassemia in Chinese: use of in vivo RNA analysis and oligonucleotide hybridization in systematic characterization of molecular defects.

Cited by 5 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. RNA-Based Therapeutic Technology;International Journal of Molecular Sciences;2023-10-16

2. Increased autophagy leads to decreased apoptosis during β-thalassaemic mouse and patient erythropoiesis;Scientific Reports;2022-11-03

3. Medical student acceptance on gene therapy to increase children's well-being with genetic diseases: a study in Indonesia;Future Science OA;2022-07

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

5. U7 snRNA: A tool for gene therapy;The Journal of Gene Medicine;2021-02-23

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