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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Main Authors: Bashayer A. Khalifah, Shareefa A. Alghamdi, Ali H. Alhasan
Format: Article
Language:English
Published: Frontiers Media S.A. 2023-11-01
Series:Frontiers in Bioengineering and Biotechnology
Subjects:
Online Access:https://www.frontiersin.org/articles/10.3389/fbioe.2023.1244377/full
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author Bashayer A. Khalifah
Bashayer A. Khalifah
Shareefa A. Alghamdi
Ali H. Alhasan
Ali H. Alhasan
author_facet Bashayer A. Khalifah
Bashayer A. Khalifah
Shareefa A. Alghamdi
Ali H. Alhasan
Ali H. Alhasan
author_sort Bashayer A. Khalifah
collection DOAJ
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 doaj.art-094194226f154c14b5e54effcf69d95d2023-11-17T08:35:02ZengFrontiers Media S.A.Frontiers in Bioengineering and Biotechnology2296-41852023-11-011110.3389/fbioe.2023.12443771244377Unleashing the potential of catalytic RNAs to combat mis-spliced transcriptsBashayer A. Khalifah0Bashayer A. Khalifah1Shareefa A. Alghamdi2Ali H. Alhasan3Ali H. Alhasan4Institute for Bioengineering, Health Sector, King Abdulaziz City for Science and Technology (KACST), Riyadh, Saudi ArabiaFaculty of Sciences, King Abdulaziz University, Jeddah, Saudi ArabiaFaculty of Sciences, King Abdulaziz University, Jeddah, Saudi ArabiaInstitute for Bioengineering, Health Sector, King Abdulaziz City for Science and Technology (KACST), Riyadh, Saudi ArabiaCollege of Science and General Studies, Alfaisal University, Riyadh, Saudi ArabiaHuman 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.https://www.frontiersin.org/articles/10.3389/fbioe.2023.1244377/fullintron retentionsynthetic biologyspliceosomespliceopathiescatalytic RNAs
spellingShingle Bashayer A. Khalifah
Bashayer A. Khalifah
Shareefa A. Alghamdi
Ali H. Alhasan
Ali H. Alhasan
Unleashing the potential of catalytic RNAs to combat mis-spliced transcripts
Frontiers in Bioengineering and Biotechnology
intron retention
synthetic biology
spliceosome
spliceopathies
catalytic RNAs
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 intron retention
synthetic biology
spliceosome
spliceopathies
catalytic RNAs
url https://www.frontiersin.org/articles/10.3389/fbioe.2023.1244377/full
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