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Unleashing the potential of catalytic RNAs to combat mis-spliced transcripts

Human transcriptome can undergo RNA mis-splicing due to spliceopathies contributing to the increasing number of genetic diseases including muscular dystrophy (MD), Alzheimer disease (AD), Huntington disease (HD), myelodysplastic syndromes (MDS). Intron retention (IR) is a major inducer of spliceopat...

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Autores principales: Khalifah, Bashayer A., Alghamdi, Shareefa A., Alhasan, Ali H.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10690607/
https://www.ncbi.nlm.nih.gov/pubmed/38047291
http://dx.doi.org/10.3389/fbioe.2023.1244377
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author Khalifah, Bashayer A.
Alghamdi, Shareefa A.
Alhasan, Ali H.
author_facet Khalifah, Bashayer A.
Alghamdi, Shareefa A.
Alhasan, Ali H.
author_sort Khalifah, Bashayer A.
collection PubMed
description Human transcriptome can undergo RNA mis-splicing due to spliceopathies contributing to the increasing number of genetic diseases including muscular dystrophy (MD), Alzheimer disease (AD), Huntington disease (HD), myelodysplastic syndromes (MDS). Intron retention (IR) is a major inducer of spliceopathies where two or more introns remain in the final mature mRNA and account for many intronic expansion diseases. Potential removal of such introns for therapeutic purposes can be feasible when utilizing bioinformatics, catalytic RNAs, and nano-drug delivery systems. Overcoming delivery challenges of catalytic RNAs was discussed in this review as a future perspective highlighting the significance of utilizing synthetic biology in addition to high throughput deep sequencing and computational approaches for the treatment of mis-spliced transcripts.
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spelling pubmed-106906072023-12-02 Unleashing the potential of catalytic RNAs to combat mis-spliced transcripts Khalifah, Bashayer A. Alghamdi, Shareefa A. Alhasan, Ali H. Front Bioeng Biotechnol Bioengineering and Biotechnology Human transcriptome can undergo RNA mis-splicing due to spliceopathies contributing to the increasing number of genetic diseases including muscular dystrophy (MD), Alzheimer disease (AD), Huntington disease (HD), myelodysplastic syndromes (MDS). Intron retention (IR) is a major inducer of spliceopathies where two or more introns remain in the final mature mRNA and account for many intronic expansion diseases. Potential removal of such introns for therapeutic purposes can be feasible when utilizing bioinformatics, catalytic RNAs, and nano-drug delivery systems. Overcoming delivery challenges of catalytic RNAs was discussed in this review as a future perspective highlighting the significance of utilizing synthetic biology in addition to high throughput deep sequencing and computational approaches for the treatment of mis-spliced transcripts. Frontiers Media S.A. 2023-11-16 /pmc/articles/PMC10690607/ /pubmed/38047291 http://dx.doi.org/10.3389/fbioe.2023.1244377 Text en Copyright © 2023 Khalifah, Alghamdi and Alhasan. https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Bioengineering and Biotechnology
Khalifah, Bashayer A.
Alghamdi, Shareefa A.
Alhasan, Ali H.
Unleashing the potential of catalytic RNAs to combat mis-spliced transcripts
title Unleashing the potential of catalytic RNAs to combat mis-spliced transcripts
title_full Unleashing the potential of catalytic RNAs to combat mis-spliced transcripts
title_fullStr Unleashing the potential of catalytic RNAs to combat mis-spliced transcripts
title_full_unstemmed Unleashing the potential of catalytic RNAs to combat mis-spliced transcripts
title_short Unleashing the potential of catalytic RNAs to combat mis-spliced transcripts
title_sort unleashing the potential of catalytic rnas to combat mis-spliced transcripts
topic Bioengineering and Biotechnology
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10690607/
https://www.ncbi.nlm.nih.gov/pubmed/38047291
http://dx.doi.org/10.3389/fbioe.2023.1244377
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