The notorious R.N.A. in the spotlight - drug or target for the treatment of disease

Reautschnig, Philipp; Vogel, P.; Stafforst, Thorsten

doi:10.1080/15476286.2016.1208323

Cited by 26 publications

(19 citation statements)

References 169 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Numerous antisense oligonucleotides have progressed to human clinical trial for diseases such as muscular dystrophy [ 130 ]. While antisense oligonucleotides are still struggling their ways to the clinic, major challenges include off-target effects, efficacy, and immune system activation [ 131 ]. Interestingly, many marketed drugs also regulate the alternative splicing machinery, the contribution of those marketed drugs for the treatment of diseases in targeting alternative RNA splicing remains to be explored.…”

Section: Discussionmentioning

confidence: 99%

Alternative mRNA Splicing in the Pathogenesis of Obesity

Wong

Yau

2018

IJMS

View full text Add to dashboard Cite

Alternative mRNA splicing is an important mechanism in expansion of proteome diversity by production of multiple protein isoforms. However, emerging evidence indicates that only a limited number of annotated protein isoforms by alternative splicing are detected, and the coding sequence of alternative splice variants usually is only slightly different from that of the canonical sequence. Nevertheless, mis-splicing is associated with a large array of human diseases. Previous reviews mainly focused on hereditary and somatic mutations in cis-acting RNA sequence elements and trans-acting splicing factors. The importance of environmental perturbations contributed to mis-splicing is not assessed. As significant changes in exon skipping and splicing factors expression levels are observed with diet-induced obesity, this review focuses on several well-known alternatively spliced metabolic factors and discusses recent advances in the regulation of the expressions of splice variants under the pathophysiological conditions of obesity. The potential of targeting the alternative mRNA mis-splicing for obesity-associated diseases therapies will also be discussed.

show abstract

Section: Discussionmentioning

confidence: 99%

Alternative mRNA Splicing in the Pathogenesis of Obesity

Wong

Yau

2018

IJMS

View full text Add to dashboard Cite

show abstract

“…In a complementary fashion, miRNAs have commonly been silenced using antagomirs, synthetic phosphorothioate‐ and 2′OCH 3 ‐modified oligonucleotides,, which have been placed under optical control through a nucleobase‐caging approach (Figure d) . This enabled the spatiotemporal investigation of miR‐22 and miR‐124 function in migrating neurons .…”

Section: Optical Control Of Oligonucleotidesmentioning

confidence: 99%

Optochemical Control of Biological Processes in Cells and Animals

et al. 2018

View full text Add to dashboard Cite

Biological processes are naturally regulated with high spatial and temporal control, as is perhaps most evident in metazoan embryogenesis. Chemical tools have been extensively utilized in cell and developmental biology to investigate cellular processes, and conditional control methods have expanded applications of these technologies toward resolving complex biological questions. Light represents an excellent external trigger since it can be controlled with very high spatial and temporal precision. To this end, several optically regulated tools have been developed and applied to living systems. In this review we discuss recent developments of optochemical tools, including small molecules, peptides, proteins, and nucleic acids that can be irreversibly or reversibly controlled through light irradiation, with a focus on applications in cells and animals.

show abstract

“…It is likely that there is still space for further optimization of the guideRNA′s design to obtain better editing results with all three enzymes. In summary, both findings are important for the advancement of site-directed RNA editing as a tool in basic biology or as a platform for therapeutic editing [ 7 , 28 ].…”

Section: Discussionmentioning

confidence: 99%

Applying Human ADAR1p110 and ADAR1p150 for Site-Directed RNA Editing—G/C Substitution Stabilizes GuideRNAs against Editing

Heep

Mach

Reautschnig

et al. 2017

Genes

Self Cite

View full text Add to dashboard Cite

Site-directed RNA editing is an approach to reprogram genetic information at the RNA level. We recently introduced a novel guideRNA that allows for the recruitment of human ADAR2 to manipulate genetic information. Here, we show that the current guideRNA design is already able to recruit another human deaminase, ADAR1, in both isoforms, p110 and p150. However, further optimization seems necessary as the current design is less efficient for ADAR1 isoforms. Furthermore, we describe hotspots at which the guideRNA itself is edited and show a way to circumvent this auto-editing without losing editing efficiency at the target. Both findings are important for the advancement of site-directed RNA editing as a tool in basic biology or as a platform for therapeutic editing.

show abstract

The notorious R.N.A. in the spotlight - drug or target for the treatment of disease

Cited by 26 publications

References 169 publications

Alternative mRNA Splicing in the Pathogenesis of Obesity

Alternative mRNA Splicing in the Pathogenesis of Obesity

Optochemical Control of Biological Processes in Cells and Animals

Applying Human ADAR1p110 and ADAR1p150 for Site-Directed RNA Editing—G/C Substitution Stabilizes GuideRNAs against Editing

Contact Info

Product

Resources

About