Journal
JOURNAL OF IMMUNOLOGICAL METHODS
Volume 365, Issue 1-2, Pages 1-7Publisher
ELSEVIER
DOI: 10.1016/j.jim.2010.12.001
Keywords
Pre-mRNA splicing; Antisense morpholino oligonucleotides; Genetic disorders; Primary immunodeficiency diseases
Categories
Funding
- NINDS NIH HHS [R01 NS052528-04, R01 NS052528] Funding Source: Medline
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Highly complementary antisense morpholino oligonucleotides (AMOs) can bind to pre-mRNA and modulate splicing site selection. This offers a powerful tool to regulate the splicing process, such as correcting subtypes of splicing mutations and nonsense mutations and reprogramming alternative splicing processes. Therefore, AMO-mediated splicing modulation represents an attractive therapeutic strategy for genetic disorders. Primary immunodeficiency diseases (PIDs) are a heterogeneous group of genetic disorders that result from mutations in genes involved in development and maintenance of the immune system. Many of these mutations are splicing mutations and nonsense mutations that can be manipulated by AMOs. This review discusses AMO-mediated splicing modulation approaches and their potential applications in treating PIDs. (C) 2010 Elsevier B.V. All rights reserved.
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