An engineered U 1 small nuclear RNA rescues splicing‐defective coagulation F 7 gene expression in mice-Reference-Cited by-同舟云学术

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An engineered U 1 small nuclear RNA rescues splicing‐defective coagulation F 7 gene expression in mice

Published:2014-02 Issue:2 Volume:12 Page:177-185
ISSN:1538-7933
Container-title:Journal of Thrombosis and Haemostasis
language:en
Short-container-title:J Thromb Haemost

Author:

Balestra D.¹,Faella A.²,Margaritis P.²³,Cavallari N.¹,Pagani F.⁴,Bernardi F.¹,Arruda V. R.²³,Pinotti M.¹

Affiliation:

1. Department of Life Sciences and Biotechnology, and LTTA University of Ferrara Ferrara Italy

2. Department of Pediatrics The Children's Hospital of PhiladelphiaPhiladelphia PA USA

3. University of Pennsylvania Perelman School of Medicine Philadelphia PA USA

4. International Centre for Genetic Engineering and Biotechnology Trieste Italy

Funder

Telethon

Publisher

Wiley

Subject

Hematology

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1111/jth.12471

Reference32 articles.

1. Special lectures in haemophilia management

2. Mechanisms for selecting 5′ splice sites in mammalian pre-mRNA splicing

3. Pre-mRNA splicing and human disease

4. Aberrant 5′ splice sites in human disease genes: mutation pattern, nucleotide structure and comparison of computational tools that predict their utilization

5. Identification of a mutation that perturbs NF1 agene splicing using genomic DNA samples and a minigene assay

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1. Clinical, Laboratory, and Molecular Aspects of Factor VII Deficiency;Seminars in Thrombosis and Hemostasis;2024-08-29

2. Using RNA-targeting CRISPR-Cas13 and engineered U1 systems to reduceABCA4splice variants in Stargardt disease;2024-03-09

3. Development of Engineered-U1 snRNA Therapies: Current Status;International Journal of Molecular Sciences;2023-09-27

4. In Vivo Efficacy and Safety Evaluations of Therapeutic Splicing Correction Using U1 snRNA in the Mouse Retina;Cells;2023-03-21

5. Engineered U1 snRNAs to modulate alternatively spliced exons;Methods;2022-09

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