4LBA A phase 1 study of first-in-class microRNA-34 mimic, MRX34, in patients with hepatocellular carcinoma or advanced cancer with liver metastasis

Beg, Mahjabeen Mirza; Brenner, A. J.; Sachdev, Jasgit C.; Borad, Mitesh J.; Cortés, Javier; Tibes, Raoul; Kang, Y.K.; Bader, Andreas G.; Stoudemire, Jay; Smith, S.; Kim, S.; Hong, D.S.

doi:10.1016/s0959-8049(14)70725-7

Cited by 6 publications

(3 citation statements)

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“…Nevertheless, the feature of miRNA non-specificity elicits a great advantage that enables a possibility of treating diseases with multiple pathologic mechanisms such as diabetic cardiomyopathy. To date, miRNA therapies that only target miR-122 and miR-34 are undergoing clinical trials for the treatment of hepatitis C and cancer respectively [41, 114, 115]. miR-208/499 and miR-15/195 antagomiRs have entered preclinical development stage for treating chronic heart failure and infarction remodeling respectively [116].…”

Section: Clinical Applicationmentioning

confidence: 99%

Role of microRNA in diabetic cardiomyopathy: From mechanism to intervention

Guo

Nair

2017

Biochimica et Biophysica Acta (BBA) - Molecular Basis of Diseas

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Diabetic cardiomyopathy is a chronic and irreversible heart complication in diabetic patients, and is characterized by complex pathophysiologic events including early diastolic dysfunction, cardiac hypertrophy, ventricular dilation and systolic dysfunction, eventually resulting in heart failure. Despite these characteristics, the underlying mechanisms leading to diabetic cardiomyopathy are still elusive. Recent studies have implicated microRNA, a small and highly conserved non-coding RNA molecule, in the etiology of diabetes and its complications, suggesting a potentially novel approach for the diagnosis and treatment of diabetic cardiomyopathy. This brief review aims at capturing recent studies related to the role of microRNA in diabetic cardiomyopathy.

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Section: Clinical Applicationmentioning

confidence: 99%

Role of microRNA in diabetic cardiomyopathy: From mechanism to intervention

Guo

Nair

2017

Biochimica et Biophysica Acta (BBA) - Molecular Basis of Diseas

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“…Therefore, correcting these miRNA deficiencies by either antagonizing or restoring miRNA function may provide a therapeutic benefit. Several miRNA-based therapeutics are in clinical trials, such as MRX34 (a miR-34a mimic for cancer treatment)12, 13 and RG-101 (an miR-122 inhibitor for the treatment of hepatitis C) (M.H. van der Ree et al., 2015, Hepatology, abstract).…”

Section: Introductionmentioning

confidence: 99%

Preclinical and Clinical Advances of GalNAc-Decorated Nucleic Acid Therapeutics

Huang

2017

Molecular Therapy - Nucleic Acids

232

203

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A main challenge in realizing the full potential of nucleic acid therapeutics is efficient delivery of them into targeted tissues and cells. N-acetylgalactosamine (GalNAc) is a well-defined liver-targeted moiety benefiting from its high affinity with asialoglycoprotein receptor (ASGPR). By conjugating it directly to the oligonucleotides or decorating it to a certain delivery system as a targeting moiety, GalNAc has achieved compelling successes in the development of nucleic acid therapeutics in recent years. Several oligonucleotide modalities are undergoing pivotal clinical studies, followed by a blooming pipeline in the preclinical stage. This review covers the progress of GalNAc-decorated oligonucleotide drugs, including siRNAs, anti-miRs, and ASOs, which provides a panorama for this field.

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“…Although the miRNA field is relatively young, a miRNA-based therapeutic has already entered phase 2 clinical trials [ 11 , 12 ]. In cancer, therapeutic strategies targeting miRNAs can be divided into two opposing approaches: (1) MiRNA mimics, which restore the function of tumor suppressive miRNAs; and (2) MiRNA inhibitors, which antagonize the function of oncogenic miRNAs.…”

Section: Introductionmentioning

confidence: 99%

Competing endogenous RNA networks: tying the essential knots for cancer biology and therapeutics

2015

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A recently discovered dimension of post-transcriptional gene regulation involves co-regulatory crosstalk between RNA transcripts, which compete for common pools of microRNA (miRNA) molecules. These competing endogenous RNAs (ceRNAs), or natural miRNA sponges, have an active role in regulating miRNA availability within the cell and form intertwined regulatory networks. Recent reports have implicated diverse RNA species including protein-coding messenger RNAs and non-coding RNAs as ceRNAs in human development and diseases including human cancer. In this review, we discuss the most recent discoveries that implicate natural miRNA decoys in human cancer biology, as well as exciting advances in the study of ceRNA networks and dynamics. The structure and topology of intricate genome-scale ceRNA networks can be predicted computationally, and their dynamic response to fluctuations in ceRNA and miRNA levels can be studied via mathematical modeling. Additionally, the development of new methods to quantitatively determine absolute expression levels of miRNA and ceRNA molecules have expanded the capacity to accurately study the efficiency of ceRNA crosstalk in diverse biological models. These major milestones are of critical importance to identify key components of ceRNA regulatory networks that could aid the development of new approaches to cancer diagnostics and oligonucleotide-based therapeutics.

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4LBA A phase 1 study of first-in-class microRNA-34 mimic, MRX34, in patients with hepatocellular carcinoma or advanced cancer with liver metastasis

Cited by 6 publications

References 0 publications

Role of microRNA in diabetic cardiomyopathy: From mechanism to intervention

Role of microRNA in diabetic cardiomyopathy: From mechanism to intervention

Preclinical and Clinical Advances of GalNAc-Decorated Nucleic Acid Therapeutics

Competing endogenous RNA networks: tying the essential knots for cancer biology and therapeutics

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