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miR-193a inhibitor
- Development stage
- Preclinical
- Lead developer
- Children's Hospital Los Angeles
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
miR-193a inhibitor refers to a class of therapeutic agents, typically antisense oligonucleotides (antagomirs), designed to inhibit the activity of microRNA-193a (miR-193a). In the kidney, upregulation of miR-193a is associated with podocyte injury, cell cycle reentry, and dedifferentiation, which are key drivers of glomerular diseases such as Alport Syndrome (AS) and focal segmental glomerulosclerosis (FSGS). Researchers at Children's Hospital Los Angeles (CHLA) and the University of Southern California (USC) are developing a podocyte-targeted peptide amphiphile micelle nanoparticle delivery system carrying a miR-193a inhibitor. This therapeutic approach aims to restore the quiescent G0 state of podocytes, upregulate Wilms tumor protein 1 (WT1), and preserve the glomerular filtration barrier, thereby slowing or reversing the progression of chronic kidney disease.
- Other names
- anti-miR-193aanti-miR193aanti-miR 193amiR-193a antagomirmiR193a antagomirmiR 193a antagomirmiR-193a-3p inhibitormiR193a-3p inhibitormiR 193a-3p inhibitormiR-193a-5p inhibitormiR193a-5p inhibitormiR 193a-5p inhibitor
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