2019
DOI: 10.1111/cts.12624
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Targeting RNA: A Transformative Therapeutic Strategy

Abstract: The therapeutic pathways that modulate transcription mechanisms currently include gene knockdown and splicing modulation. However, additional mechanisms may come into play as more understanding of molecular biology and disease etiology emerge. Building on advances in chemistry and delivery technology, oligonucleotide therapeutics is emerging as an established, validated class of drugs that can modulate a multitude of genetic targets. These targets include over 10,000 proteins in the human genome that have hith… Show more

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Cited by 117 publications
(114 citation statements)
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References 91 publications
(369 reference statements)
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“…In the cell, these oligonucleotides can hybridize to target RNA sequences, including mRNA and non-coding (nc)RNA to inhibit their expression and thereby regulate the availability of specific proteins. Different chemical modifications of synthesized oligonucleotides have been made to increase their nuclease stability, decrease non-specific effects and to improve their cellular uptake (Karaki et al, 2019;Yin and Rogge, 2019). These include phosphorothioates (PS) and derivatives of, phosphorodiamidate morphilino oligomers, peptide nucleic acids (PNAs), and chimeric 2 ′ -O-methyl/phosphorothioate ASOs (Crooke, 2017).…”
Section: Antisense Oligonucleotidesmentioning
confidence: 99%
“…In the cell, these oligonucleotides can hybridize to target RNA sequences, including mRNA and non-coding (nc)RNA to inhibit their expression and thereby regulate the availability of specific proteins. Different chemical modifications of synthesized oligonucleotides have been made to increase their nuclease stability, decrease non-specific effects and to improve their cellular uptake (Karaki et al, 2019;Yin and Rogge, 2019). These include phosphorothioates (PS) and derivatives of, phosphorodiamidate morphilino oligomers, peptide nucleic acids (PNAs), and chimeric 2 ′ -O-methyl/phosphorothioate ASOs (Crooke, 2017).…”
Section: Antisense Oligonucleotidesmentioning
confidence: 99%
“…RNAi, including antisense oligonucleotides, gapmeRs and siRNAs, are effective tools for gene therapy due to their ease of screening and upscaling. Current RNAi-based therapeutics have been developed with some success, with several RNAi-based therapies approved by the Federal Drug Administration (FDA) [97]. However, as reviewed by Win and Rogge et al (2019), toxicity profiles of these modalities need to be improved [97].…”
Section: Lncrnas As Therapeutic Targets For Hiv-1mentioning
confidence: 99%
“…Current RNAi-based therapeutics have been developed with some success, with several RNAi-based therapies approved by the Federal Drug Administration (FDA) [97]. However, as reviewed by Win and Rogge et al (2019), toxicity profiles of these modalities need to be improved [97]. Of promise are the recent clinical trials using conjugated siRNAs to treat cholesterol, whereby a single dose of the targeted siRNA molecules had lasting effects on in vivo with minimal side effects reported [98,99].…”
Section: Lncrnas As Therapeutic Targets For Hiv-1mentioning
confidence: 99%
“…From the editorial viewpoint, the current theme of From Molecule to Patient has captured emerging concepts of new drugs and platforms. Examples of nonsmall molecule drugs are not only therapeutic antibodies, which have already been widely evaluated and utilized in the clinic but therapeutic RNAs and cell‐based therapies . In the era of precision medicine, drugs are designed to hit specific therapeutic targets on a molecular basis, and new modalities are expected to pick the fruits that no one was able to reach in the past.…”
mentioning
confidence: 99%
“…Examples of nonsmall molecule drugs are not only therapeutic antibodies, which have already been widely evaluated and utilized in the clinic 1 but therapeutic RNAs and cell-based therapies. 2 In the era of precision medicine, drugs are designed to hit specific therapeutic targets on a molecular basis, and new modalities are expected to pick the fruits that no one was able to reach in the past. This is certainly an exciting change for all the researchers and healthcare providers in the field of clinical pharmacology.…”
mentioning
confidence: 99%