Recent studies have verified that non-coding RNAs (ncRNAs) [include intronic RNAs, microRNAs (miRNAs), long non-coding RNAs (lncRNA), circular RNAs (circRNA), and extracellular RNAs］are not non-functional substances. Although ncRNAs lack the potential to translate to proteins, they can affect the expression or activities of other genes and proteins through a variety of mechanisms. The ability of ncRNA to control gene expression may affect normal gene expression and disease progression, making it a new hotspot in drug research. Therefore, the synthesis of nucleic acid has become a promising starting point for drug development. From the trend of ncRNA research, in the near future, the research focus is on the binding mechanism or impact of the splicing function of the oligonucleotides or compounds to miRNA. In the long run, the understanding of the regulatory mechanism of lncRNA and other ncRNAs on target genes/proteins may increase.
Since the active mechanism of ncRNAs is non-traditional and is not been fully uncovered yet, there are some controversies. Many possible mechanisms for manipulating expression using lncRNA are just beginning to be understood. These unknown or uncertain pharmacological mechanisms make the identification and development of lead compounds more challenging. Although the therapeutic potential of oligonucleotides has been recognized for more than 40 years, few drugs have been approved so far. Part of the reason is that the specificity of ncRNA is difficult to control because of its few thousand Daltons mass of oligonucleotides which contains many hydrogen bond donors, receptors, and multiple negative charges. The RNA-targeting oligonucleotides must enter the cell to be active. One miRNA can usually regulate multi genes, thus, blocking a single miRNA may manipulate multiple signal pathways to produce different effects. lncRNA sequences are less conserved between human and animal models. It makes it difficult to study the physiological/pathological activities of lncRNA in humans. This research topic will focus on all research results of ncRNA in pharmacological mechanisms, hoping to make breakthroughs in the above problems.
To promote the research on genetic modification in pharmacology, we create this research topic. It is encouraged to publish any research reports on gene modification related to disease foundations and clinics. It also provides an ideal platform for the academic exchange of gene editing in genetic study. This research topic will include but not limit to the following research fields:
1. Genes and target proteins regulation by miRNA, intronic RNA, repetitive RNA, and lncRNA.
2. Emerging classes of RNA drug targets, including miRNAs, intron/exon junctions, repetitive RNAs, and lncRNAs.
3. The pharmacological mechanism of miRNA inhibitors and mimetics.
4. Role of antisense oligonucleotides (ASO) and double-stranded RNA in ncRNA regulation.
5. Off-target effects and mechanism of RNAi.
6. Pharmacological regulation mechanism of genasense or phosphorothioate DNA (PS-DNA) on apoptosis and other cell pathology.
7. Novel gene modification techniques.
Recent studies have verified that non-coding RNAs (ncRNAs) [include intronic RNAs, microRNAs (miRNAs), long non-coding RNAs (lncRNA), circular RNAs (circRNA), and extracellular RNAs］are not non-functional substances. Although ncRNAs lack the potential to translate to proteins, they can affect the expression or activities of other genes and proteins through a variety of mechanisms. The ability of ncRNA to control gene expression may affect normal gene expression and disease progression, making it a new hotspot in drug research. Therefore, the synthesis of nucleic acid has become a promising starting point for drug development. From the trend of ncRNA research, in the near future, the research focus is on the binding mechanism or impact of the splicing function of the oligonucleotides or compounds to miRNA. In the long run, the understanding of the regulatory mechanism of lncRNA and other ncRNAs on target genes/proteins may increase.
Since the active mechanism of ncRNAs is non-traditional and is not been fully uncovered yet, there are some controversies. Many possible mechanisms for manipulating expression using lncRNA are just beginning to be understood. These unknown or uncertain pharmacological mechanisms make the identification and development of lead compounds more challenging. Although the therapeutic potential of oligonucleotides has been recognized for more than 40 years, few drugs have been approved so far. Part of the reason is that the specificity of ncRNA is difficult to control because of its few thousand Daltons mass of oligonucleotides which contains many hydrogen bond donors, receptors, and multiple negative charges. The RNA-targeting oligonucleotides must enter the cell to be active. One miRNA can usually regulate multi genes, thus, blocking a single miRNA may manipulate multiple signal pathways to produce different effects. lncRNA sequences are less conserved between human and animal models. It makes it difficult to study the physiological/pathological activities of lncRNA in humans. This research topic will focus on all research results of ncRNA in pharmacological mechanisms, hoping to make breakthroughs in the above problems.
To promote the research on genetic modification in pharmacology, we create this research topic. It is encouraged to publish any research reports on gene modification related to disease foundations and clinics. It also provides an ideal platform for the academic exchange of gene editing in genetic study. This research topic will include but not limit to the following research fields:
1. Genes and target proteins regulation by miRNA, intronic RNA, repetitive RNA, and lncRNA.
2. Emerging classes of RNA drug targets, including miRNAs, intron/exon junctions, repetitive RNAs, and lncRNAs.
3. The pharmacological mechanism of miRNA inhibitors and mimetics.
4. Role of antisense oligonucleotides (ASO) and double-stranded RNA in ncRNA regulation.
5. Off-target effects and mechanism of RNAi.
6. Pharmacological regulation mechanism of genasense or phosphorothioate DNA (PS-DNA) on apoptosis and other cell pathology.
7. Novel gene modification techniques.
