STRC Cross-Hypothesis Parameter Audit 2026-04-23 — Batch 2 (h04, h06, h07, h08, h27)

Extension of the morning sweep (h01, h02, h05, h09, h26). Five additional hypotheses audited by a single Sonnet 4.6 agent using cat/grep/WebSearch only. Read-only; no scripts modified.


Scoreboard

#HypothesisTierPhantom citesWrong valuesStructural issuesDefensibility
h04STRC mRNA-LNP Strategy B Full-LengthB00 (all params inherited)STRC t½ + Hill constants traceable only to internal JSON, not primary literatureCLEAN — internally self-consistent; primary-lit gaps inherited from h06
h06STRC mRNA Therapy (Strategy A)A1 confirmed phantom (Gao 2020 PMID 32493791 ≠ cochlear LNP paper); 1 unverified (Leclere 2024 exists but PMID not confirmed)STRC t½ 14 d + RBM24 t½ 2 d unsourced from primary literature; ENDO_MRNA_SS 50 copies/OHC biological estimate onlyABR calibration points “OTOF DB-OTO cohort” real but individual datapoint attribution imprecise⚠ Core pharmacology robust; Gao 2020 PMID phantom; half-life priors lack primary citation
h07Prime Editing for STRC E1659AC1 confirmed phantom (Gao 2020 PMID 32493791 = COVID diagnostics paper); 0 others (Chen 2024 ✅, Villiger 2021 ✅, Chemla 2025 ✅, Anzalone 2019 ✅, Fang 2021 ✅, Zhang 2025 ✅, Kim 2023 ✅)None — note makes no unsourced numerical claims beyond “15–40%” efficiency range which is explicitly stated as extrapolationPhase 3 script (pe_phase3_5_strcp1_aware.py) relies on genome FASTA; zero external bio-constantsCLEAN on scripts; 1 phantom in atomic note prose (Gao 2020 citation)
h08STRC ASO Exon SkippingC0 phantoms — no external papers cited in scriptsENCODE RBM24 RBNS accession ENCSR742AEU cited (not verified by web search but ENCODE format is valid); nearest-neighbor ΔH/ΔS values from SantaLucia standard tables (plausible, not individually cited)ASO design is fully in-silico with Ensembl REST API pulls; no external bio-constants with suspicious magnitudesCLEAN — scripts are self-contained calculations
h27STRC STRCP1 Activation RescueC0GTEx ENSG IDs correct (verified: ENSG00000242866.9 STRC, ENSG00000166763.7 STRCP1); no numerical constants beyond TPM threshold 0.1 (standard field convention)Hypothesis explicitly pre-Gate 1; no compute work yet; script is a live GTEx API query, not a hardcoded modelCLEAN — no constants to audit; zero external bio-constants embedded

Per-Hypothesis Parameter-Provenance Tables


h04 — STRC mRNA-LNP Strategy B Full-Length (strc_mrna_strategy_b_pkpd.py, strategy_b_audiogram_rescue.py)

ConstValueClaimed sourceStatusVerdict
S_WT (endogenous STRC protein SS)3021.1 unitsrbm24_mrna_dose_response_results.json (inherited from h06 single-dose model)⚠ Internal chaining — traces to h06 unsourced Hill fitINHERITED RISK — see h06
STRC_HL_D (STRC protein t½)14 drbm24_mrna_dose_response_results.json (labelled “dominant eigenvalue — inherited”)⚠ No primary literature citation in any script or noteUNSOURCED in primary lit — plausible for structural extracellular protein but no paper pinned
ENDO_MRNA_SS (endogenous STRC mRNA SS per OHC)50 copies/OHC”biological estimate” per script comment❌ No source at allUNSOURCED — acknowledged as estimate; single-cell cochlear transcriptomics (Liu 2020 PNAS; Ellwanger 2018 Sci Data) do not report per-cell absolute counts; magnitude is plausible but unverified
LNP_ENDO_ESCAPE2%“literature” per note⚠ No specific citation; literature review confirms <10% is a known range; 2% is within published bounds (all LNPs <10%)UNSOURCED but defensible — consistent with published endosomal escape literature (Sahay & Bhosale, Bhatt reviews)
LNP_UNTARGETED0.8% = 96/12,000 OHC”standard LNP, literature” per note⚠ No specific citationUNSOURCED but plausible — consistent with Gao 2018 Beethoven model (lipid delivery to cochlea neonatal); no LNP-to-OHC specific published efficiency figure confirmed
LNP_COCHLEAR_TROPIC5%“hypothetical next-gen LNP”✅ Explicitly labelled hypotheticalCORRECTLY FLAGGED
LNP_OHC_TARGETED20%“hypothetical ligand LNP”✅ Explicitly labelled hypotheticalCORRECTLY FLAGGED
mRNA t½ unmod OHC4 hmrna_stability_cochlear_results.json⚠ JSON exists; generating script not found; citation trace unclearINTERNAL — generating script missing
mRNA t½ m1ψ OHC12 hmrna_stability_cochlear_results.json⚠ Same as aboveINTERNAL
K_TRANSLATE_STRCDerived: S_WT × k_s / ENDO_MRNA_SSDerived constant✅ Math is internally consistentDERIVATION CLEAN (but rests on ENDO_MRNA_SS = unsourced)
Misha s_endo_frac0.15”maternal E1659A × 0.5 hemizygous × ~0.3 residual activity”⚠ Estimate acknowledged in Limitations sectionACKNOWLEDGED ESTIMATE — appropriately disclosed
ABR calibration pointsSee h06 (shares abr_transfer_model.py)See h06See h06See h06

h04 summary: Zero phantom citations. All suspicious values either inherited from h06 (and carry h06’s risk) or explicitly labelled hypothetical/estimate. The model itself is arithmetically correct. The primary-literature sourcing gap for STRC 14 d t½ and ENDO_MRNA_SS is the main outstanding issue, but it is shared across h04 and h06 and originates in h06.


h06 — STRC mRNA Therapy Hypothesis (mrna_lnp_pkpd_integration.py, mrna_lnp_pkpd_integration_v2_ohc_tropism.py, mrna_lnp_audiogram_rescue.py)

ConstValueClaimed sourceStatusVerdict
STRC_HL_D (STRC protein t½)14 drbm24_mrna_dose_response_results.json → labelled “dominant eigenvalue”⚠ No primary citation in any file; generating script not found in models dir; traced back to an early single-dose ODE fit whose priors are undocumentedUNSOURCED from primary lit — 14 d for a structural extracellular protein is biologically plausible (comparable ECM proteins) but no paper confirms stereocilin t½ specifically
RBM24_HL_D (RBM24 protein t½)2 drbm24_mrna_dose_response_results.json⚠ Same provenance gapUNSOURCED — 2 d for an RBP is a reasonable heuristic but no STRC-OHC-specific RBM24 t½ measurement exists; CycloheximideCHX experiments could confirm but are not cited
Hill K_M (RBM24 → STRC boost)200 (relative units)“dose-response fit”⚠ Fit to self-generated ODE output, not to external binding/splicing dataCIRCULAR FIT — unitless relative to the same model; cannot fail; not a measurement
Hill n2”dose-response fit”⚠ Same as above⚠ Same
max_boost3.0דdose-response fit”⚠ Same; no experimental splicing measurement confirms 3× as an upper bound for RBM24-driven STRC boostUNSOURCED — biologically plausible (STRC is one of RBM24’s targets) but the specific 3× ceiling has no experimental basis
LNP_ENDO_ESCAPE2%“literature”⚠ Same as h04 — no specific citation⚠ Defensible range, missing citation
ER_CEILING_FOLD (v2 only)5.0× (tanh soft cap)“Sahin 2014, Hadas 2019; chronic-mRNA UPR in muscle and liver”Sahin 2014 is a review paper (Nat Rev Drug Discov 2014, 13:759) — discusses mRNA therapeutics broadly, NOT a primary UPR threshold measurement. Hadas 2019 (PMID not confirmed) appears to be a cardiac modRNA study about Pkm2/ceramidase, not an ER stress threshold study. Neither supports the specific “5× overexpression = UPR threshold” claim as a measured number. Note explicitly says “cochlea-specific data is missing.”WRONG-PAPER-MATCH for Sahin 2014 (review, not UPR threshold data); Hadas 2019 citation not verified as UPR-related; “5× ceiling” is a reasonable engineering judgment but does not have primary-lit backing from either cited paper
Wang 2018 A666 LNP ~1% OHC targeting (v2 prose)~1% OHC targeting”Wang 2018 A666-DEX-NP in vivo”PAPER EXISTS — “A666-conjugated nanoparticles target prestin of outer hair cells preventing cisplatin-induced hearing loss,” IJN 2018, PMID 30532536. A666-DEX-NP (dexamethasone, not LNP-mRNA) demonstrated OHC targeting in vivo.VALUE-CITATION MISMATCH — Wang 2018 used dexamethasone-loaded NPs, not LNP-mRNA; the ~1% figure for lipid-mRNA is an engineering extrapolation, not a direct reading from this paper
Gao 2020 LNP-CBE cochlear delivery, 0.3–0.6% editing efficiency0.3–0.6% editing, Organ of CortiCited in atomic note as “Gao 2020 (Sci Transl Med, PMID 32493791)”🚨 PMID 32493791 = “COVID-19 diagnostics in context” (Science Translational Medicine, 2020) — completely unrelated to cochlear gene therapy. NO cochlear LNP-CBE paper by Gao with this PMID exists.🚨 PHANTOM CITATION
Leclere 2024 (non-viral vectors)cited for LNP cochlear delivery efficiency data”Leclere 2024” atomic note reference⚠ A 2024 Hearing Research review paper by a French author on non-viral cochlear gene therapy vectors was found by web search (PMID 39427589). The paper exists but is a review, not a primary efficiency measurement.CITATION PLAUSIBLY REAL but unconfirmed PMID; review paper rather than primary data
Geng 2017, Landegger 2017 (tonotopic gradient for LNP)Basal 2×, mid 1×, apical 0.5× gradientCited in audiogram script docstring⚠ Landegger 2017 (Landegger et al., Nat Biotechnol) is a real paper on Anc80L65 cochlear delivery. Geng 2017 not individually verified but the basal > apical gradient for round-window injection is well-established across multiple groups.Landegger 2017 likely real; Geng 2017 not verified; gradient shape is standard convention
ABR calibration: “OTOF DB-OTO cohort 1 & 2 estimates”30 dB @ 70% transduction; 47 dB @ 40%“OTOF DB-OTO cohort”⚠ DB-OTO trial (Regeneron) is real and published (NEJM, Lancet). But specific calibration point assignments to cohort 1 vs cohort 2 are author-derived interpolations, not tabulated values from DB-OTO papers.OVER-PRECISE ATTRIBUTION — values plausible but the specific cohort assignments are interpretive
ABR calibration: “Bredberg 1968, Schuknecht & Gacek 1993”Nonlinear OHC-count/threshold relationshipClassic histopathology references✅ Both papers are real and foundationalREAL CITATIONS
ABR calibration: “Lesperance et al. 2026, Ear and Hearing”OTOF trial as endpoint modelListed as motivation in abr_transfer_model.py⚠ No Lesperance 2026 Ear and Hearing paper found in web search; DB-OTO 2026 results are in ScienceDaily/clinical reports but attributed to Regeneron/Fudan groups, not a Lesperance Ear and Hearing paperUNVERIFIED — possible pre-publication or mismatch

h06 summary: 1 confirmed phantom (Gao 2020 PMID 32493791 = COVID paper). 1 wrong-paper-match (Sahin 2014 cited for ER stress 5× threshold but is a drug discovery review). Wang 2018 exists but has a delivery-modality mismatch (DEX-NP ≠ LNP-mRNA). Core Hill constants and half-lives are internally circular (fit to own model) with no primary experimental anchor. The computational structure of the model is sound; the quantitative output rests on unvalidated priors.


h07 — Prime Editing for STRC E1659A (pe_phase3_5_strcp1_aware.py)

ConstValueClaimed sourceStatusVerdict
STRC genomic coordinatechr15:43600551 (GRCh38)Ensembl REST API (fetched live in prior phases)✅ Verifiable from Ensembl
Best PAM: TGG at chr15:43600540, 14 nt from variantConfirmed April 2026Ensembl REST-derived + PAM search✅ Genomic coordinates derivable
Chen 2024 dual-AAV PE, 42% cortical neurons42% efficiency in post-mitotic cortex”Nature Biotechnology, PMID 37142705”PAPER EXISTS AND IS CORRECT — Chen PJ et al. Nat Biotechnol 2024:42:253. PE-AAV up to 42% cortex, 46% liver, 11% heart. PMID 37142705 confirmed.CONFIRMED
Villiger 2021 dual-AAV split PE, retinaSplit-intein PE delivery to adult retina”Molecular Therapy, PMID 34298129”PAPER EXISTS — “Dual-AAV delivering split prime editor system for in vivo genome editing,” Mol Therapy 2021, PMID 34298129 confirmed.CONFIRMED
Chemla 2025 PE4 in iPSC-CMs, 34.8%34.8% T→C editing post-mitotic CMs”Mol Therapy Nucleic Acids, PMID 41210585”PAPER EXISTS AND NUMBERS CORRECT — “Prime editing corrects the dilated cardiomyopathy causing RBM20-P633L-mutation in human cardiomyocytes,” PMID 41210585. PE4 with epegRNA-12: 34.8% ± 4.6% confirmed.CONFIRMED — exact value matches
Anzalone 2019 PE in post-mitotic neurons”low frequency, no number given""Nature, PMID 31634902”PAPER EXISTS — Nature 576:149, 2019, PMID 31634902. Work confirmed PE in primary cortical neurons via synapsin-PE lentivirus. “Low frequency” characterisation is accurate.CONFIRMED
Fang 2021 dual-AAV9-PHP.B STRC, 50% DPOAE recovery50% mice show DPOAE recovery”Sci Adv, PMID 34910522”PAPER EXISTS AND IS THE DFNB16 paper — “Dual-vector gene therapy restores cochlear amplification and auditory sensitivity in a mouse model of DFNB16 hearing loss,” Sci Adv 2021, PMID 34910522 confirmed.CONFIRMED
Gao 2020 LNP-CBE cochlear, 0.3–0.6% editing”proof of delivery, very low efficiency""Gao 2020 (Sci Transl Med, PMID 32493791)”🚨 PMID 32493791 = COVID-19 diagnostics paper. NOT a cochlear LNP paper. The actual Gao 2018/2020 inner ear work (Tmc1 CRISPR) used PMID 30181527 / lipid injection in Beethoven mice — different paper, different system, different year.🚨 PHANTOM CITATION — PMID is wrong and paper does not exist as described
Zhang 2025 ABE SchABE8e Anc80L65 POU4F3 cochlea”near-complete hearing recovery, 4+ months""Nature Communications, PMID 40968144”⚠ Web search confirms a Zhang 2025 Nat Commun paper on Pou4f3WT/Q113* base editing with Anc80L65 SchABE8e, near-complete hearing recovery, but PMID 40968144 could not be confirmed via direct PubMed lookup; the actual PMID for this paper may differ slightlyPAPER LIKELY REAL but PMID not independently confirmed; value (near-complete recovery) matches search results
Kim 2023 ACBE, 35–45% A→C35% average, up to 45% cell culture”Nature Biotechnology, PMID 37322276”PAPER EXISTS — “Adenine transversion editors enable precise, efficient A•T-to-C•G base editing,” Nat Biotechnol 2024 (epub 2023), PMID 37322276 confirmed. The paper reports up to 73% with evolved variants. The 35% “average” is more conservative than the actual published data.CONFIRMED (note: value is under-stated vs published 73%; conservative is fine)
STRC exon coordinate GRCh38/GRCh37chr15:43600551 / chr15:43892749Standard coordinate liftover✅ Plausible given typical chr15 liftover offset (~292 kb)
Realistic OHC PE efficiency range15–40%“extrapolating from cortical neurons (42%) and cardiomyocytes (35%)”✅ Explicitly labeled as extrapolation with stated basisAPPROPRIATELY DISCLOSED
SEED_MISMATCH_GATE≥2 seed mismatches requiredStandard Cas9 seed-region biology✅ Consistent with published Cas9 specificity literature

h07 summary: 7 out of 8 key literature citations confirmed real. 1 confirmed phantom (Gao 2020 PMID 32493791 = COVID paper, replicated from h06). 1 citation likely real but PMID not independently verified (Zhang 2025). The script itself (pe_phase3_5_strcp1_aware.py) has zero external bio-constants — it reads genomic sequence from FASTA and computes mismatch counts. Model risk is in the atomic note prose only.


h08 — STRC ASO Exon Skipping (aso_phase1_design.py, aso_phase2_strcp1_specificity.py)

ConstValueClaimed sourceStatusVerdict
TRANSCRIPT_ID ENST00000450892canonical STRC transcriptUniProt Q7RTU9-1, Ensembl✅ Verified Ensembl ID
PROTEIN_ID ENSP00000401513STRC canonical proteintranslation of ENST00000450892
E1659A in exon 27missense in exon 27 of 29stated in atomic note⚠ Not verified independently in this audit but consistent with standard exon mappingPLAUSIBLE, not independently checked here
RBM24 motifs TGTGTG/GTGTGT, GCTCTTCENCODE RBNS ENCSR742AEU, enrichment ≥4.5ENCODE accession⚠ ENCODE RBNS accession format is valid; web search confirmed RBM24 RBNS data exists on ENCODE portal; specific accession ENCSR742AEU not directly verified (ENCODE portal not queried)ENCODE FORMAT VALID; SPECIFIC ACCESSION UNVERIFIED
Nearest-neighbor ΔH/ΔS tablesSantaLucia 1998 DNA:DNA parameters used as proxy for DNA:RNAStandard SantaLucia NN tables✅ The NN values embedded in the script match published SantaLucia 1998 unified parameters exactly (e.g., AA: dH=-7.9, dS=-22.2; CG: dH=-10.6, dS=-27.2)CONFIRMED — standard SantaLucia 1998 values, correctly applied
FLANK_NT = 80 nt (exon flank context)80 nt intronic flankStandard ASO design convention✅ 50–100 nt is standard for splice-blocking ASO design
ASO_LENS = (18, 20, 22)Industry standard PMO lengthsASO chemistry convention✅ 18–22mer is the standard PMO range for splice-switching
DMD precedent: 4 FDA-approved PMOs (Exondys 51, Vyondys 53, Viltepso, Amondys 45)All 4 named and correctly described”FDA-approved”✅ All 4 drugs exist, all use PMO chemistry for exon skipping in DMD. Eteplirsen=exon 51 ✅, Golodirsen=exon 53 ✅, Viltolarsen=exon 53 ✅, Casimersen=exon 45 ✅CONFIRMED
STRC has 29 exons”STRC 29 exons”Standard annotation✅ Correct per Ensembl ENSG00000242866
STRC_START/END, STRCP1_START/END chr15 coordinatesSTRC: 43.58–43.64 Mb; STRCP1: 43.68–43.72 Mb”Verified via PE Phase 4 on-target locus and STRCP1 paralog cluster”✅ Consistent with GRCh38 chr15 annotation for both loci
E1 event: dPSI −0.542, FDR 2.1e-5From Sun 2026 rMATS analysis”SD03 (Sun 2026)”⚠ Sun 2026 is an internal reference to computed splicing analysis; the rbm24_sd03_splicing_analysis.json artifact is real but “Sun 2026” is not a published paper found in web searchINTERNAL DATA — the rMATS analysis was done on the project’s own data; “Sun 2026” is mislabelled as a citation if it refers to an internal analysis

h08 summary: Zero phantom citations in scripts. The ASO Phase 1 script is the most self-contained calculation of the five audited hypotheses — it fetches sequences live from Ensembl REST, uses standard NN thermodynamics with correctly-sourced SantaLucia parameters, and makes no external biophysical claims. Phase 2 script is a pure Hamming-distance scan over chr15 FASTA. Main flag: ENCODE accession ENCSR742AEU not independently verified, and “Sun 2026” is referenced as if a published paper when it appears to be an internal computation.


h27 — STRC STRCP1 Activation Rescue (ohc_strcp1_expression_check.py)

ConstValueClaimed sourceStatusVerdict
GENCODE_STRCENSG00000242866.9GENCODE v26 as used by GTEx v8✅ Correct GENCODE ID for STRC
GENCODE_STRCP1ENSG00000166763.7GENCODE v26✅ Correct GENCODE ID for STRCP1 pseudogene
GTEx v8 API endpointgtexportal.org/api/v2/expression/medianGeneExpressionGTEx public API✅ Live API call; real data pulled at runtime
TPM threshold for “expressed”> 0.1 TPM”standard field convention”✅ 0.1 TPM is the widely-used expression floor in GTEx studies
STRCP1 transcribed in 54 GTEx tissues, TPM 0.01–2.6From script output in atomic noteohc_strcp1_expression_check.json (live GTEx query)✅ These values are GTEx-derived and were queried at runtime; reproducible
STRCP1 GENCODE biotype “transcribed_unprocessed_pseudogene”GENCODE biotype labelStandard annotation✅ Correct per Ensembl/GENCODE annotation
CRISPRa dCas9-VP64/VPR 10–20× upregulation achievableAssumed in hypothesis framingGeneral CRISPRa literature⚠ Correct order of magnitude (CRISPRa achieves 2–100× depending on locus/construct) but no specific citation for STRCP1 promoter contextPLAUSIBLE — no false number
STRCP1 promoter at chr15:~43,700,xxxInferred from STRCP1 locus position (~100 kb downstream of STRC)Genomic logic⚠ Position is plausible but not directly fetched; promoter boundaries need Ensembl/GENCODE regulatory annotation lookupESTIMATE — not computed

h27 summary: Zero phantom citations. Zero wrong numerical constants. The script is a live API query with no embedded parameters that could be fabricated. This is the cleanest possible audit target: a hypothesis-framing note paired with a live-data-pull script. The only outstanding item is the absence of primary-source backing for the CRISPRa upregulation claim, which is a reasonable background claim rather than a model constant.


Aggregated Root-Cause Analysis

Constants that appear across multiple hypotheses

ConstantAppears inStatusRoot cause
STRC protein t½ = 14 dh04, h06 (both scripts and pkpd note)⚠ Not primary-lit sourcedOriginated in rbm24_mrna_dose_response_results.json as an assumed “dominant eigenvalue”; no stereocilin-specific t½ measurement published
RBM24 protein t½ = 2 dh06 (all three scripts)⚠ Not primary-lit sourcedSame JSON source; RBM24 t½ data in OHCs does not exist in literature; 2 d is a plausible heuristic
Hill K_M = 200, n = 2, max_boost = 3×h06 (all scripts)⚠ Circular fit to own ODENever measured experimentally; values are set so that the model achieves “therapeutic” at a reasonable dose — i.e., fit to a judgment call, not data
LNP endosomal escape = 2%h04, h06⚠ Unsourced generic “literature”Correct range (published literature consistently <10%) but no specific paper cited; Patel et al. 2019 PNAS (endosomal bottleneck) would anchor this
ENDO_MRNA_SS = 50 copies/OHCh04 only❌ Acknowledged biological estimateNo OHC single-cell transcript counting study reports absolute STRC mRNA copies per cell
Gao 2020 PMID 32493791h06 (atomic note), h07 (atomic note)🚨 PHANTOM (= COVID paper)Both h06 and h07 cite the same wrong PMID for what appears to be an attempt to reference a cochlear LNP-CBE efficiency paper (likely the Gao 2018 Tmc1 CRISPR/lipid paper, PMID 30181527, or a different 2020 study entirely)

The phantom pattern in batch 2 vs batch 1

Batch 1 (h01, h02, h05, h09, h26): 4/5 hypotheses had phantoms, mostly in external binding affinity constants (Kd, d31, CREB rates).

Batch 2 (h04, h06, h07, h08, h27): 2/5 hypotheses have the Gao 2020 phantom — but notably it is the same phantom in two places, not two independent inventions. This is citation laundering: the wrong PMID (32493791) was generated once and copy-pasted. The underlying claim (LNP cochlear delivery at very low OHC efficiency) is biologically real — the Gao 2018 Beethoven cochlear lipid-delivery paper (PMID 30181527) or the Leclere 2024 review would be appropriate sources — but the PMID is wrong.

The h08 and h27 scripts are structurally different: they make no external bio-constant claims and instead derive values from public APIs or standard tables. These represent the healthiest pattern in the STRC computational portfolio.


Ranking Deltas

#Axis changeVerdict
h04 (B-tier)No change to tier. All numbers internally consistent; primary-lit gaps are inherited from h06.B held
h06 (A-tier)1 confirmed phantom (Gao 2020), 1 wrong-paper-match (Sahin 2014 for ER ceiling), Hill constants circular. Core pharmacology structure sound.A held but requires: (1) replace Gao 2020 PMID 32493791 with correct cochlear delivery reference; (2) source or acknowledge STRC 14d t½ and RBM24 2d t½ as estimates
h07 (C-tier)Same Gao 2020 phantom in note prose; all other 7 citations confirmed real; scripts have zero constants.C held (tier is not about citation quality; tier is about delivery feasibility and competition with h03/h04/h08)
h08 (C-tier)No phantoms, no wrong values, cleanest scripts in entire portfolio.C held (tier reflects scientific risk profile of exon skipping for missense, not citation quality)
h27 (C-tier)No phantoms, no wrong values, hypothesis explicitly pre-Gate-1.C held pending Gate 1 wet-lab (Holt lab ribosome profiling)

h04 — Strategy B Full-Length

  1. Measure or source STRC t½. CHX-pulse-chase in any OHC-like cell line (HEI-OC1 or neonatal mouse organ of Corti) would anchor the 14 d assumption. Alternatively, search for structural extracellular matrix proteins with known t½ in published proteomics turnover datasets (SILAC-based proteome turnover studies). Flag as ESTIMATED_14D until sourced.
  2. Source or replace ENDO_MRNA_SS = 50 copies/OHC. Liu 2020 PNAS single-cell atlas or Ellwanger 2018 Sci Data mouse OHC transcriptome should contain relative STRC expression; converting to absolute copies requires UMI calibration, which most scRNA-seq papers don’t provide. Acceptable to leave as estimate with explicit disclosure.
  3. No script changes needed.

h06 — Strategy A PKPD

  1. Replace Gao 2020 PMID 32493791. The correct reference for cochlear LNP delivery efficiency is likely: (a) Gao et al. 2018 PMID 30181527 (Tmc1 CRISPR/lipid neonatal cochlear delivery), or (b) Leclere 2024 PMID 39427589 (review), or (c) pending identification of the actual cochlear CBE + LNP paper.
  2. Correct Sahin 2014 citation for ER stress 5× ceiling. Sahin 2014 is a drug-discovery review and does not provide a 5× secretory protein UPR threshold. Replace with a primary-literature UPR induction threshold paper or explicitly label ER_CEILING_FOLD = 5.0 as engineering judgment with no primary citation.
  3. Source STRC t½ and RBM24 t½ (same action as h04).
  4. Verify Lesperance 2026 Ear and Hearing citation or replace with the confirmed DB-OTO NEJM paper.

h07 — Prime Editing

  1. Replace Gao 2020 PMID 32493791 in atomic note with correct cochlear delivery reference (same fix as h06).
  2. Verify Zhang 2025 POU4F3 PMID 40968144 directly on PubMed — the paper appears to exist but the PMID couldn’t be confirmed from web search. Correct if wrong.
  3. Scripts are clean — no action needed.

h08 — ASO Exon Skipping

  1. Verify ENCODE accession ENCSR742AEU for RBM24 RBNS data. Direct query to encodeproject.org/experiments/ENCSR742AEU/ would confirm existence. Low priority given it’s a secondary tool-use citation, not a model constant.
  2. Clarify “Sun 2026” attribution — if this refers to the project’s own rMATS analysis of an external dataset, rename to something like “internal splicing analysis (rMATS on Ensembl cochlear RNA-seq, 2026-04-20)” rather than an author-year citation format that implies an external publication.
  3. Scripts are clean — no action needed.

h27 — STRCP1 Activation Rescue

  1. No immediate citation actions needed.
  2. Gate 1 is the only blocking action — email Holt lab re: ribosome profiling on STRC OHC lysates.
  3. If Gate 1 passes, next audit turn should check CRISPRa upregulation literature for STRCP1 locus-specific data.

Summary: What Is Actually Defensible Today

Defensible as-is (requires no citation fix to run computations):

  1. h08 ASO Phase 1/2 scripts — purest calculation in the portfolio; Ensembl REST + SantaLucia NN; zero external bio-constants requiring citation
  2. h27 GTEx expression check — live API query, no embedded constants
  3. h04 Strategy B model structure — arithmetic is correct; it inherits h06 risk but adds no new phantoms

Requires fix before citing in publications:

  1. h06 Gao 2020 PMID 32493791 — confirmed phantom; easy fix (find the real cochlear LNP paper)
  2. h07 Gao 2020 same phantom — copy of same error; fix simultaneously
  3. h06 Hill constants (K_M, n, max_boost) — not phantoms but circular fits; need either experimental calibration or explicit disclosure as “ODE-fitted parameters with no experimental validation”

Connections