Market Intelligence, Clinical Progress, and High-Purity Reagents for Retinitis Pigmentosa Type 11 (RP11) Development.
PRPF31 is a core component of the U4/U6-U5 tri-snRNP splicing complex. Its haploinsufficiency due to loss-of-function mutations (e.g., A216P, P231L) is the primary cause of autosomal dominant retinitis pigmentosa type 11 (RP11). Current therapeutic strategies focus on gene augmentation (AAV), small molecule splicing modulators, CRISPR-based editing, and antisense oligonucleotides (ASOs). TarMart provides a comprehensive toolbox for RP11 drug discovery.
TarMart Solution Ecosystem & Related Targets
| Component / Network | Product Description | Product Link |
|---|---|---|
| Antigen | PRPF31 Recombinant Protein (Wild-Type & RP11 Mutants: A216P, P231L, etc.) High purity (>95%), Endotoxin <1 EU/µg. Sequence Verified. Includes disease-associated variants for genotype-phenotype studies. |
View PRPF31 Products |
| Gene Delivery | PRPF31 Promise-ORF Lentivirus Full-length ORF for stable retinal cell line construction (ARPE-19, 661W). High titer (>10^8 TU/mL). Antibiotic selection available. |
View PRPF31 Products |
| Benchmark Antibody | Anti-PRPF31 (Research Grade Recombinant) High-specificity positive control for Western Blot, IP, IHC, and Co-IP. Verified for tri-snRNP complex detection. |
View PRPF31 Products |
| Validator | PRPF31 siRNA Set Validated for precise 40-60% knockdown (haploinsufficiency modeling). qPCR‑verified. |
View PRPF31 Products |
| Pathway Partner | PRPF8 Core U5 snRNP component; essential for spliceosomal assembly and PPI validation. |
View PRPF8 Products |
| Pathway Partner | PRPF3 (Hprp3) U4/U6 snRNP component; synergistic splicing complex analysis. |
View PRPF3 Products |
| Pathway Partner | SNRNP200 (Brr2) ATP-dependent RNA helicase in the same tri-snRNP complex. |
View SNRNP200 Products |
| Disease Model | RHO (Rhodopsin) Primary downstream photoreceptor target; mutation in PRPF31 often disrupts RHO splicing. Alternative RP target for comparative degeneration studies. |
View RHO Products |
Critical Assay Challenges & TarMart Advantages
| Critical Assay Challenge | The TarMart Advantage (Technical Spec) |
|---|---|
| Haploinsufficiency Modeling (WT vs Mutant comparison) | Validated siRNA Set achieving precise 40-60% knockdown; lentivirus for dose‑controlled rescue. |
| Tri‑snRNP Complex Reconstitution & Assembly Analysis | High‑purity WT and mutant proteins (>95%) for Co‑IP pull‑down assays; multi‑tag formats (N‑His, GST, Avi). |
| Retinal Cell Line Construction (ARPE‑19, 661W) | Lentivirus premade particles with puromycin selection; titers >10^8 TU/mL. |
| Gene Therapy Expression Quantification | Sequence‑verified, high‑purity recombinant protein serves as absolute standard for ELISA/Western. |
| Mutant Protein Stability & Off‑target Screening | Specific mutants (A216P, P231L, etc.) expressed and aggregation‑tested; homolog panel (PRPF3, PRPF8, SNRNP200) available for cross‑reactivity. |
Live PRPF31 R&D Tracker
Market data changes daily. Access the latest global pipeline status directly:
Global Clinical Landscape & Future Outlook
The landscape for PRPF31‑associated retinitis pigmentosa (RP11) is transitioning from diagnostic characterization to therapeutic intervention. As the most extensively characterized splicing factor linked to adRP, PRPF31 represents a foundational target for haploinsufficiency drug discovery. The race for PRPF31 therapeutics is intensifying, with major players shifting focus from traditional small molecules to precision genetic medicines. First‑generation AAV gene augmentation is advancing toward clinical trials; emerging modalities include CRISPR‑based transcriptional activation (CRISPRa) to upregulate the remaining wild‑type allele, small‑molecule splicing correctors to stabilize residual tri‑snRNP function, and antisense oligonucleotides targeting aberrant splicing. A key challenge is avoiding overexpression toxicity in retinal pigment epithelium (RPE) cells, which requires precise expression control.
Competitive Modality & Indication Snapshot
| Modality | Representative Players | Key Indications | Critical Assay Need (Why TarMart?) |
|---|---|---|---|
| Gene Therapy (AAV) | MeiraGTx, Novartis (academic partnerships), ophthalmic gene therapy developers | Autosomal Dominant Retinitis Pigmentosa (adRP, RP11) | Haploinsufficiency rescue assay (need precise knockdown siRNA + overexpression lentivirus) |
| Small Molecule Splicing Modulator | ProQR, specialized rare‑disease biotechs | adRP (PRPF31 haploinsufficiency) | Splicing reporter assay (need WT vs mutant protein controls) |
| CRISPR/Cas9 Editing | Editas Medicine, Intellia, ophthalmic gene‑editing innovators | Dominant negative alleles; inherited retinal dystrophies | Genotype‑phenotype correlation (need mutant‑specific proteins for validation) |
| Antisense Oligo (ASO) / Splice‑Switching | RNA therapeutics companies | adRP | Target engagement assay (need high‑purity recombinant PRPF31; lentivirus cell models) |