{"doi":"10.1101/2021.07.22.453413","title":"Binocular Benefit Following Monocular Subretinal AAV Injection in a Mouse Model of Autosomal Dominant Retinitis Pigmentosa (adRP)","abstract":"Abstract Autosomal dominant retinitis pigmentosa (adRP) is frequently caused by mutations in RHO , the gene for rhodopsin. In previous experiments in dogs with the T4R mutation in RHO , an AAV2/5 vector expressing both an shRNA directed to human and dog RHO mRNA and an shRNA-resistant human RHO cDNA (AAV- RHO820-shRNA820 ) prevented retinal degeneration for more than 8 months following injection. To confirm that this same vector could protect the retinas of a different species and bearing a different RHO mutation, we injected mice transgenic for human P23H RHO at postnatal day 30 in one eye. For nine months, we monitored their retinal structure using spectral- domain optical coherence tomography (SD-OCT) and retinal function using electroretinography (ERG). We compared these to P23H RHO transgenic mice injected with AAV-GFP. Though retinas continued to thin over time, compared to control injected eyes, AAV-RHO820-shRNA820 slowed the loss of photoreceptor cells and decreased ERG amplitudes in AAV- RHO820-shRNA820 eyes during the nine-month study period. Unexpectedly, we also observed preservation of retinal structure and function in the untreated contralateral eyes of AAV- RHO820-shRNA820 treated mice. PCR analysis and western blots provided evidence that a low amount of vector from injected eyes was present in uninjected eyes.","journal":"bioRxiv (Cold Spring Harbor Laboratory)","year":2021,"id":225822,"datarank":0.0,"base_score":0.0,"endowment":0.0,"self_citation_contribution":0.0,"citation_network_contribution":0.0,"self_endowment_contribution":0.0,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":0,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9529,"is_data_producer":false,"deposit_databanks":null,"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2021-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":374405,"name":"Michael T. Massengill","orcid":"0000-0003-0408-6596","position":1,"is_corresponding":false},{"id":392473,"name":"Cristhian J. Ildefonso","orcid":"0000-0001-6179-720X","position":2,"is_corresponding":false},{"id":535537,"name":"Ping Zhu","orcid":"0000-0003-2189-5563","position":3,"is_corresponding":false},{"id":681908,"name":"Hong Li","orcid":"0000-0001-9371-1212","position":4,"is_corresponding":false},{"id":828571,"name":"Anil Patel","orcid":"0000-0003-1634-7650","position":5,"is_corresponding":false},{"id":325707,"name":"Alfred S. Lewin","orcid":"0000-0002-4192-9727","position":6,"is_corresponding":false},{"id":327462,"name":"Chulbul M. Ahmed","orcid":null,"position":0,"is_corresponding":true}],"reference_count":35,"raw_metadata":null,"created_at":"2026-07-18T23:54:30.292454Z","pmid":null,"pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}