Dashboard

Platform Overview

What would you like to do?

Explore a target, review mechanism-specific candidates, and keep the supporting evidence in view. Start with a gene below, or use an existing sequence. Support and evidence vary by mechanism; computational outputs require experimental validation.

41

Therapeutic Mechanisms

9

Therapeutic Goals

11

Computational Modules

Add

Increase or restore useful RNA or protein.

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Remove

Reduce harmful RNA or its activity.

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Modify

Change or correct RNA and molecular function.

Explore options

1. Choose your target

Organism *
Disease Name
Gene symbol or ID *

Search by Disease

Don't know the gene yet? Search by disease name — associations are human-based (Open Targets) and run directly on your selected organism.

Upload Sequence

Action

Import custom ASO, siRNA, or RNA sequences for analysis and design.

Direct Designers

Power user

Skip the guided flow — go straight to any mechanism designer (A21–A41).

Differential Splicing

Discovery

Discover disease-associated splicing events from RNA-seq and design splice-switching oligonucleotides.

Analysis Modules

  • 01Biological Information Retrieval
  • 02Therapeutic Mechanism Configuration
  • 03Molecular Defect Identification
  • 04Therapeutic Goal Prediction
  • 05Rulebook Execution
  • 06Mechanism-specific Target Discovery
  • 07Candidate Design
  • 08Candidate Optimization
  • 09Candidate Ranking
  • 10Computational Evidence Review
  • 11Final Report Generation

Therapeutic Mechanisms

Gene Silencing (8)

  • A1: RNase H-mediated Gapmer Knockdown
  • A2: Steric-Blocking Translation Inhibition
  • A12: microRNA Inhibition (Anti-miR / AntagomiR)
  • A15: Promoter-Targeted RNA Silencing (Experimental; design unavailable)
  • A21: RNA Interference (siRNA)
  • A22: External Guide Sequence (EGS) — RNase P Recruitment
  • A40: Target-Directed miRNA Degradation (TDMD) Trigger
  • A41: U1 Adaptor — snRNP-Tethered Nuclear mRNA Degradation

Gene Upregulation (6)

  • A3: Poison Exon Blocking (TANGO)
  • A4: AntagoNAT (NAT Knockdown)
  • A5: uORF Blocking
  • A6: miRNA Binding Site Blocking
  • A23: saRNA-mediated Transcriptional Activation
  • A28: Translation Repressor Site Blocking (RBP Masking)

RNA Editing / Correction (6)

  • A13: ADAR-Mediated Site-Directed RNA Editing (SDRE)
  • A16: C-to-U Editing (APOBEC / RESCUE)
  • A17: LEAPER — Endogenous ADAR Recruitment
  • A18: CIRTS — Programmable RNA Effector Recruitment
  • A19: REPAIR — Cas13b–ADAR2 A-to-I Editing
  • A20: SMaRT Trans-Splicing

RNA Processing Modulation (5)

  • A7: Exon Skipping
  • A8: Exon Inclusion (Splice Correction)
  • A9: Pseudoexon Suppression
  • A10: Cryptic Splice-site Blocking
  • A11: Alternative Polyadenylation (APA) Modulation

RNA Neutralization (1)

  • A14: Toxic RNA Neutralization (RNA Foci Disruption)

Translational Regulation (4)

  • A27: Riboswitch / RNA Structure Targeting
  • A29: IRES-Targeted Translation Modulation
  • A30: Kozak Sequence Modulation
  • A31: Poly(A) Tail / PABP Competition

Isoform Engineering (2)

  • A32: Alternative Promoter Switch
  • A33: Targeted Intron Retention

Protein Replacement (5)

  • A24: Messenger RNA (mRNA) Replacement Therapy
  • A26: Circular RNA (circRNA)-Mediated Protein Replacement
  • A34: Trans-splicing ASO-Mediated Exon Replacement
  • A35: Readthrough-Inducing ASO
  • A36: Engineered Suppressor tRNA

Protein Function Modulation (4)

  • A25: RNA Aptamer Therapeutics
  • A37: Allosteric RNA Aptamer
  • A38: Aptamer-Drug Conjugate (ApDC)
  • A39: Bifunctional RNA Aptamer

Platform Architecture

Biological Information
Knowledge Retrieval
Mechanism Rulebooks
Computational Engine
Validation & Output