Advancing RNA Therapies in Neuromuscular Disease
RNA-based therapeutics, including antisense oligonucleotides (ASOs), have opened new pathways for treating genetically driven neuromuscular diseases like Amyotrophic Lateral Sclerosis (ALS) and Duchenne Muscular Dystophy (DMD). By targeting underlying mutations at the transcript level, ASOs offer the promise of disease-modifying effects, whether by restoring protein expression, correcting splicing defects, or silencing toxic transcripts.
Landmark advances, such as FDA-approved ASO therapies for spinal muscular atrophy and ALS (e.g., Tofersen, targeting SOD1), have shown what's possible. The next challenge lies in delivery, tracking, and tissue-specific uptake, especially in hard-to-reach targets like motor neurons and muscle fibers.
These insights underscore the need for reliable molecular tools to:
Landmark advances, such as FDA-approved ASO therapies for spinal muscular atrophy and ALS (e.g., Tofersen, targeting SOD1), have shown what's possible. The next challenge lies in delivery, tracking, and tissue-specific uptake, especially in hard-to-reach targets like motor neurons and muscle fibers.
Tracking and Validating RNA Therapuetics In Situ
Recent research from Roche (Nature, 2025) highlights the importance of intracellular trafficking in determining the efficacy of RNA therapies. Using a CRISPR screen, the study identified key regulators of endosomal transport, providing new levers to enhance RNA drug bioavailability in disease-relevant tissues.These insights underscore the need for reliable molecular tools to:
- Confirm ASO uptake in affected tissues
- Quantify target protein modulation
- Visualize ASO localization at cellular and subcellular levels
Rockland's Tools for the RNA Therapeutics Workflow
Antibodies from Rockland and antibodies-online support critical steps in RNA theraputic development for ALS and DMD:ALS-related Targets
Product | Target | Relevance | Applications |
Anti-TDP43 | TARDBP | Hallmark of RNA-binding portein mislocalized in ALS | WB, IHC, ICC |
Anti-SOD1 | SOD1 | Mutated in familial ALS, target of approved ASO therapy (Tofersen) | WB, IHC, ICC |
Anti-C9ORF72 | C9ORF72 | Most common genetic ALS mutation; key ASO target | |
Anti-STMN2 | STMN2 | Key ASO efficacy readout downstream of TDP-43 dysfunction | WB, IHC, ICC |
Anti-Neurofilament L | NEFL (NF-L) | Biomarker of axonal damage; used as a clinical endpoint in trials | WB, IHC |
Anti-EAAT2 | SLC1A2 (EAAT2) | Excitotoxicity regulator; target of neuroprotection strategies | WB, IHC |
Anti-SQSTM1/p62 | SQSTM1 (p62) | Aggregation and autophagy marker, ALS biomarker | WB, IHC |
Anti-Nogo-A | RTN4 (Nogo-A) | Inhibitor of axonal regeneration; ALS and neurotrauma models | WB, IHC |
DMD-related Targets
Product | Target | Relevance | Application |
Anti-Dystrophin | Dystrophin | Gold-standard marker for ASO rescue of DMD expression | WB, IHC |
Anti-Utrophin | Utrophin | Functional dystophin analog; upregulation supports muscle integrity | WB, IHC, ICC |
Anti-MSTN | MSTN (Mystatin) | Inhibitor of muscle growth; suppression is a therapeutic strategy | WB, IHC |
Anti-CTGF | CTGF | Pro-fibrotic marker upregulated in DMD; monitors muscle healing response | WB, IHC |
Anti-VDAC1 | VDAC1 | Marker of mitochondiral dysfunction in dystrophic muscle | WB, IHC, ICC, IF |
HDAC1-10 | HDACs | Epigenetic regulators; HDAC inhibiton may enhance regeneration | WB, IHC |
Anti-TGFB1 | TGF-β1 | Master regulator of fibosis; often used alongside CTGF | WB, ELISA, IHC |
Anti-CD68 | CD68 | Macrophage infiltration marker; tracks inflammation and immune response | WB, IHC |
Track ASO Distribution and Localization with ModDetect™
- Compatible with IHC, ICC, and quantitative assays
- Enables co-localization with ALS/DMD biomarkers