Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus
Open accessAbstract
Usher 1 patients are born profoundly deaf and then develop retinal degeneration. Thus they are readily identified before the onset of retinal degeneration, making gene therapy a viable strategy to prevent their blindness. Here, we have investigated the use of adeno-associated viruses (AAVs) for the delivery of the Usher 1B gene, MYO7A, to retinal cells in cell culture and in Myo7a-null mice. MYO7A cDNA, under control of a smCBA promoter, was packaged in single AAV2 and AAV5 vectors and as two overlapping halves in dual AAV2 vectors. The 7.9-kb smCBA-MYO7A exceeds the capacity of an AAV vector; packaging of such oversized constructs into single AAV vectors may involve fragmentation of the gene. Nevertheless, the AAV2 and AAV5 single vector preparations successfully transduced photoreceptor and retinal pigment epithelium cells, resulting in functional, full-length MYO7A protein and correction of mutant phenotypes, suggesting successful homologous recombination of gene fragments. With discrete, conventional-sized dual AAV2 vectors, full-length MYO7A was detected, but the level of protein expression was variable, and only a minority of cells showed phenotype correction. Our results show that MYO7A therapy with AAV2 or AAV5 single vectors is efficacious; however, the dual AAV2 approach proved to be less effective.
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Integrity signals
Registry
Cites
Statistics
Claims the paper makes
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
A study found dual overlapping vectors less efficacious than a single vector with a genome of approximately 7.9 kb for delivering MYO7A to mouse retinas.
“a study investigating the efficacy of AAV2 and AAV5 vectors to deliver MYO7A, mutations in which cause Usher syndrome 1b, to mouse retinas found the dual overlapping vectors to be less efficacious than a single vector with a genome of * 7.9 kb in size. 48”
From Expressing Transgenes That Exceed the Packaging Capacity of Adeno-Associated Virus Capsids (2016) · cites this paper as [48]
The Introduction and Discussion report that overlapping dual AAV2 vectors were substantially less effective than single AAV2 or AAV5 vectors at delivering functional MYO7A to retinas of Myo7a-mutant mice. The Results describe the single vector’s 6.7-kb MYO7A cDNA and say the construct exceeds nominal AAV carrying capacity by about 2 kb, but our copy does not establish the claimed approximately 7.9-kb genome size. (LLM, conf=0.85)
Experimental — accuracy not yet measuredSubretinal injection of single oversized AAV2/2 and AAV2/5 vectors has shown retinal transduction and phenotype correction.
“For instance, subretinal injection of single oversized AAV2/2 and AAV2/5 vectors has shown retinal transduction and phenotype correction, indicating that fragmented genomes could reconstitute the full-length cDNA post-infection [97,98].”
From Circumventing the packaging limit of AAV-mediated gene replacement therapy for neurological disorders (2022) · cites this paper as [98]
Fragmented genomes could reconstitute the full-length cDNA post-infection.
“indicating that fragmented genomes could reconstitute the full-length cDNA post-infection [97,98]”
From Circumventing the packaging limit of AAV-mediated gene replacement therapy for neurological disorders (2022) · cites this paper as [98]
Self-check
Percentages, sums and reported differences re-computed from the paper’s own numbers.
The self-check needs the paper’s full text.
Cites
Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis
Maguire, A. M., Simonelli, F., Pierce, E. A., Pugh, E. N., Mingozzi, F., Bennicelli, J., Banfi, S., Marshall, K. A., Testa, F., Surace, E. M., et al. (2008) · New England Journal of Medicine · doi:10.1056/nejmoa0802315 · Cited by 2,145 · see citing works
Effect of gene therapy on visual function in Leber’s congenital amaurosis
Bainbridge, J. W. B., Smith, A. J., Barker, S. S., Robbie, S., Henderson, R., Balaggan, K., Viswanathan, A., Holder, G. E., Stockman, A., Tyler, N., et al. (2008) · New England Journal of Medicine · doi:10.1056/nejmoa0802268 · Cited by 1,924 · see citing works
Defective myosin VIIA gene responsible for Usher syndrome type IB
Well, D., Blanchard, S., Kaplan, J., Guilford, P., Gibson, F., Walsh, J., Mburu, P., Varela, A., Levilliers, J., Weston, M. D., et al. (1995) · Nature · doi:10.1038/374060a0 · Cited by 1,068 · see citing works
Effect of genome size on AAV vector packaging
Wu, Z., Yang, H., & Colosi, P. (2010) · Molecular Therapy · doi:10.1038/mt.2009.255 · Cited by 1,025 · see citing works
Hauswirth, W. W., Aleman, T. S., Kaushal, S., Cideciyan, A. V., Schwartz, S. B., Wang, L., Conlon, T. J., Boye, S. L., Flotte, T. R., Byrne, B. J., et al. (2008) · Human Gene Therapy · doi:10.1089/hum.2008.107 · Cited by 954 · see citing works
Self-citationA type VII myosin encoded by the mouse deafness gene shaker-1
Gibson, F., Walsh, J. C., Mburu, P., Varela, A., Brown, K. A., Antonio, M., Beisel, K. W., Steel, K. P., & Brown, S. D. M. (1995) · Nature · doi:10.1038/374062a0 · Cited by 682 · see citing works
Cideciyan, A. V., Alemán, T. S., Boye, S. L., Schwartz, S., Kaushal, S., Román, A. J., Pang, J., Sumaroka, A., Windsor, E. A. M., Wilson, J. M., et al. (2008) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0807027105 · Cited by 674 · see citing works
Self-citationZhong, L., Li, B., Mah, C. S., Govindasamy, L., Agbandje-McKenna, M., Cooper, M., Herzog, R. W., Zolotukhin, I., Warrington, K. H., Weigel-Van Aken, K. A., et al. (2008) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0802866105 · Cited by 558 · see citing works
Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors
Zolotukhin, S., Potter, M., Zolotukhin, I., Sakai, Y., Loiler, S., Fraites, T. J., Chiodo, V. A., Philipsberg, T., Muzyczka, N., Hauswirth, W. W., et al. (2002) · Methods · doi:10.1016/s1046-2023(02)00220-7 · Cited by 556 · see citing works
Self-citationAcland, G. M., Aguirre, G. K., Bennett, J., Alemán, T. S., Cideciyan, A. V., Bennicelli, J. L., Dejneka, N. S., Pearce-Kelling, S. E., Maguire, A. M., Palczewski, K., et al. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2005.08.008 · Cited by 478 · see citing works
Self-citationHasson, T., Heintzelman, M. B., Santos-Sacchi, J., Corey, D. P., & Mooseker, M. S. (1995) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.92.21.9815 · Cited by 427 · see citing works
Grieger, J. C., & Samulski, R. J. (2005) · Journal of Virology · doi:10.1128/jvi.79.15.9933-9944.2005 · Cited by 419 · see citing works
Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector
Bowles, D. E., McPhee, S. W., Li, C., Gray, S. J., Samulski, J. J., Camp, A. S., Li, J., Wang, B., Monahan, P. E., Rabinowitz, J. E., et al. (2012) · Molecular Therapy · doi:10.1038/mt.2011.237 · Cited by 400 · see citing works
Auricchio, A. (2001) · Human Molecular Genetics · doi:10.1093/hmg/10.26.3075 · Cited by 394 · see citing works
Myosin VIIa Participates in Opsin Transport through The Photoreceptor Cilium
Liu, X., Udovichenko, I. P., Brown, S. D. M., Steel, K. P., & Williams, D. S. (1999) · Journal of Neuroscience · doi:10.1523/jneurosci.19-15-06267.1999 · Cited by 294 · see citing works
Self-citationAllocca, M., Doria, M., Petrillo, M., Colella, P., Garcia-Hoyos, M., Gibbs, D., Kim, S. R., Maguire, A., Rex, T. S., Di Vicino, U., et al. (2008) · Journal of Clinical Investigation · doi:10.1172/jci34316 · Cited by 292 · see citing works
Self-citationDuan, D., Yue, Y., & Engelhardt, J. F. (2001) · Molecular Therapy · doi:10.1006/mthe.2001.0456 · Cited by 262 · see citing works
Gibbs, D., Kitamoto, J., & Williams, D. S. (2003) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1130432100 · Cited by 250 · see citing works
Self-citationCharacterization of Genome Integrity for Oversized Recombinant AAV Vector
Dong, B., Nakai, H., & Xiao, W. (2010) · Molecular Therapy · doi:10.1038/mt.2009.258 · Cited by 230 · see citing works
Jacobson, S. G., Acland, G. M., Aguirre, G. D., Aleman, T. S., Schwartz, S. B., Cideciyan, A. V., Zeiss, C. J., Komaromy, A. M., Kaushal, S., Roman, A. J., et al. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.03.005 · Cited by 224 · see citing works
Self-citationYang, G. S., Schmidt, M., Yan, Z., Lindbloom, J. D., Harding, T. C., Donahue, B. A., Engelhardt, J. F., Kotin, R. M., & Davidson, B. L. (2002) · Journal of Virology · doi:10.1128/jvi.76.15.7651-7660.2002 · Cited by 200 · see citing works
Usher syndrome: Animal models, retinal function of Usher proteins, and prospects for gene therapy
Williams, D. S. (2008) · Vision Research · doi:10.1016/j.visres.2007.08.015 · Cited by 188 · see citing works
Self-citationWeil, D., Levy, G. G., Sahly, I., Lévi-Acobas, F., Blanchard, S., El-Amraoui, A., Crozet, F., Philippe, H., Abitbol, M. M., & Petit, C. (1996) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.93.8.3232 · Cited by 176 · see citing works
Mutant myosin VIIa causes defective melanosome distribution in the RPE of shaker-1 mice
Liu, X., Ondek, B., & Williams, D. S. (1998) · Nature Genetics · doi:10.1038/470 · Cited by 168 · see citing works
Self-citationEvidence for the Failure of Adeno-associated Virus Serotype 5 to Package a Viral Genome ≥8.2 kb
Lai, Y., Yue, Y., & Duan, D. (2010) · Molecular Therapy · doi:10.1038/mt.2009.256 · Cited by 166 · see citing works
Ghosh, A., Yue, Y., Lai, Y., & Duan, D. (2008) · Molecular Therapy · doi:10.1038/sj.mt.6300322 · Cited by 161 · see citing works
Role of myosin VIIa and Rab27a in the motility and localization of RPE melanosomes
Gibbs, D., Azarian, S. M., Lillo, C., Kitamoto, J., Klomp, A. E., Steel, K. P., Libby, R. T., & Williams, D. S. (2004) · Journal of Cell Science · doi:10.1242/jcs.01580 · Cited by 152 · see citing works
Self-citationPang, J., Dai, X., Boye, S. L., Barone, I., Boye, S. L., Mao, S., Everhart, D., Dinculescu, A., Liu, L., Umino, Y., et al. (2010) · Molecular Therapy · doi:10.1038/mt.2010.273 · Cited by 148 · see citing works
Self-citationChen, Z., Hasson, T., Kelley, P. M., Schwender, B. J., Schwartz, M. F., Ramakrishnan, M., Kimberling, W. J., Mooseker, M. S., & Corey, D. P. (1996) · Genomics · doi:10.1006/geno.1996.0489 · Cited by 136 · see citing works
Effects of shaker-1 mutations on myosin-VIIa protein and mRNA expression
Hasson, T., Walsh, J. C., Cable, J., Mooseker, M. S., Brown, S. D. M., & Steel, K. P. (1997) · Cell Motility and the Cytoskeleton · doi:10.1002/(sici)1097-0169(1997)37:2 · Cited by 122 · see citing works
Halbert, C. L., Allen, J. M., & Miller, A. D. (2002) · Nature Biotechnology · doi:10.1038/nbt0702-697 · Cited by 121 · see citing works
Lentiviral gene replacement therapy of retinas in a mouse model for Usher syndrome type 1B
Hashimoto, T., Gibbs, D., Lillo, C., Azarian, S. M., Legacki, E. L., Zhang, X., Yang, X.-J., & Williams, D. S. (2007) · Gene Therapy · doi:10.1038/sj.gt.3302897 · Cited by 118 · see citing works
Self-citationA FERM domain autoregulates Drosophila myosin 7a activity
Yang, Y., Baboolal, T. G., Siththanandan, V. B., Chen, M. X., Walker, M., Knight, P. J., Peckham, M., & Sellers, J. R. (2009) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0808682106 · Cited by 105 · see citing works
Structure of MyTH4-FERM Domains in Myosin VIIa Tail Bound to Cargo
Wu, L., Pan, L., Wei, Z., & Zhang, M. (2011) · Science · doi:10.1126/science.1198848 · Cited by 104 · see citing works
Pang, J.-J., Lauramore, A., Deng, W.-T., Li, Q., Doyle, T. J., Chiodo, V., Li, J., & Hauswirth, W. W. (2008) · Vision Research · doi:10.1016/j.visres.2007.08.009 · Cited by 97 · see citing works
Self-citationGene Therapy of mdx Mice With Large Truncated Dystrophins Generated by Recombination Using rAAV6
Odom, G. L., Gregorevic, P., Allen, J. M., & Chamberlain, J. S. (2011) · Molecular Therapy · doi:10.1038/mt.2010.205 · Cited by 95 · see citing works
Zhang, Y., & Duan, D. (2012) · Human Gene Therapy · doi:10.1089/hum.2011.131 · Cited by 95 · see citing works
Lopes, V. S., Ramalho, J. S., Owen, D. M., Karl, M. O., Strauss, O., Futter, C. E., & Seabra, M. C. (2007) · Traffic · doi:10.1111/j.1600-0854.2007.00548.x · Cited by 95 · see citing works
Self-citationAAV-microdystrophin Therapy Improves Cardiac Performance in Aged Female mdx Mice
Bostick, B., Shin, J.-H., Yue, Y., & Duan, D. (2011) · Molecular Therapy · doi:10.1038/mt.2011.154 · Cited by 85 · see citing works
Retinal Disease Course in Usher Syndrome 1B Due to MYO7A Mutations
Jacobson, S. G., Cideciyan, A. V., Gibbs, D., Sumaroka, A., Román, A. J., Alemán, T. S., Schwartz, S., Olivares, M. B., Russell, R. C., Steinberg, J. D., et al. (2011) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.11-8313 · Cited by 83 · see citing works
Self-citationThe many different cellular functions of MYO7A in the retina
Williams, D. S., & Lopes, V. S. (2011) · Biochemical Society Transactions · doi:10.1042/bst0391207 · Cited by 73 · see citing works
Self-citationGhosh, A., Yue, Y., & Duan, D. (2006) · The Journal of Gene Medicine · doi:10.1002/jgm.835 · Cited by 69 · see citing works
Haire, S. E., Pang, J., Boye, S. L., Sokal, I., Craft, C. M., Palczewski, K., Hauswirth, W. W., & Semple‐Rowland, S. L. (2006) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.06-0086 · Cited by 66 · see citing works
Self-citationLopes, V. S., Gibbs, D., Libby, R. T., Alemán, T. S., Welch, D. L., Lillo, C., Jacobson, S. G., Radu, R. A., Steel, K. P., & Williams, D. S. (2011) · Human Molecular Genetics · doi:10.1093/hmg/ddr155 · Cited by 64 · see citing works
Self-citationBoye, S. L., Conlon, T. J., Erger, K. E., Ryals, R. C., Neeley, A. W., Cossette, T. L., Pang, J., Dyka, F. M., Hauswirth, W. W., & Boye, S. L. (2011) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.11-7867 · Cited by 63 · see citing works
Self-citationNative Molecular State of Adeno-Associated Viral Vectors Revealed by Single-Molecule Sequencing
Kapranov, P., Chen, L., Dederich, D. A., Dong, B., He, J., Steinmann, K. E., Moore, A. R., Thompson, J. F., Milos, P. M., & Xiao, W. (2011) · Human Gene Therapy · doi:10.1089/hum.2011.160 · Cited by 58 · see citing works
The unconventional myosin-VIIa associates with lysosomes
Soni, L. E., Warren, C. M., Bucci, C., Orten, D. J., & Hasson, T. (2005) · Cell Motility and the Cytoskeleton · doi:10.1002/cm.20080 · Cited by 49 · see citing works
Function of MYO7A in the Human RPE and the Validity of Shaker1 Mice as a Model for Usher Syndrome 1B
Gibbs, D., Diemer, T., Khanobdee, K., Hu, J. G., Bok, D., & Williams, D. S. (2010) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.09-4032 · Cited by 48 · see citing works
Self-citationAllocca, M., Manfredi, A., Iodice, C., Vicino, U. D., & Auricchio, A. (2011) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.10-6269 · Cited by 43 · see citing works
Isolation and Culture of Primary Mouse Retinal Pigmented Epithelial Cells
Gibbs, D., & Williams, D. S. (2003) · Advances in experimental medicine and biology · doi:10.1007/978-1-4615-0067-4_44 · Cited by 41 · see citing works
Self-citationSchwander, M., Lopes, V. S., Sczaniecka, A., Gibbs, D., Lillo, C., Delano, D. L., Tarantino, L. M., Wiltshire, T., Williams, D. S., & Müller, U. (2009) · Journal of Neuroscience · doi:10.1523/jneurosci.4876-09.2009 · Cited by 40 · see citing works
Self-citationKLOMP, A. E. M., Teofilo, K., Legacki, E. L., & Williams, D. S. (2007) · Cell Motility and the Cytoskeleton · doi:10.1002/cm.20198 · Cited by 36 · see citing works
Self-citationCapping of the barbed ends of actin filaments by a high-affinity profilin-actin complex
DiNubile, M. J., & Huang, S. C. (1997) · Cell Motility and the Cytoskeleton · doi:10.1002/(sici)1097-0169(1997)37:3 · Cited by 14 · see citing works
Flagged first, then by citations
Cited by
Sorted by citations
Effective delivery of large genes to the retina by dual AAV vectors
Trapani, I., Colella, P., Sommella, A., Iodice, C., Cesi, G., de Simone, S., Marrocco, E., Rossi, S., Giunti, M., Palfi, A., et al. (2014) · EMBO Molecular Medicine · doi:10.1002/emmm.201302948 · Cited by 269 · see citing works
Tornabene, P., Trapani, I., Minopoli, R., Centrulo, M., Lupo, M., de Simone, S., Tiberi, P., Dell’Aquila, F., Marrocco, E., Iodice, C., et al. (2019) · Science Translational Medicine · doi:10.1126/scitranslmed.aav4523 · Cited by 192 · see citing works
Expressing Transgenes That Exceed the Packaging Capacity of Adeno-Associated Virus Capsids
Chamberlain, K., Riyad, J. M., & Weber, T. (2016) · Human Gene Therapy Methods · doi:10.1089/hgtb.2015.140 · Cited by 141 · see citing works
Dyka, F. M., Boye, S. L., Chiodo, V. A., Hauswirth, W. W., & Boye, S. E. (2014) · Human Gene Therapy Methods · doi:10.1089/hgtb.2013.212 · Cited by 128 · see citing works
Self-citationGene therapy and genome surgery in the retina
DiCarlo, J. E., Mahajan, V. B., & Tsang, S. H. (2018) · Journal of Clinical Investigation · doi:10.1172/jci120429 · Cited by 127 · see citing works
Seeing the light after 25 Years of retinal gene therapy
Trapani, I., & Auricchio, A. (2018) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2018.06.006 · Cited by 124 · see citing works
Ocular gene therapies in clinical practice: viral vectors and nonviral alternatives
Bordet, T., & Behar-Cohen, F. (2019) · Drug Discovery Today · doi:10.1016/j.drudis.2019.05.038 · Cited by 120 · see citing works
Immunology of AAV-Mediated Gene Transfer in the Eye
Willett, K., & Bennett, J. (2013) · Frontiers in Immunology · doi:10.3389/fimmu.2013.00261 · Cited by 105 · see citing works
Efficient gene delivery to the cone-enriched pig retina by dual AAV vectors
Colella, P., Trapani, I., Cesi, G., Sommella, A., Manfredi, A., Puppo, A., Iodice, C., Rossi, S., Simonelli, F., Giunti, M., et al. (2014) · Gene Therapy · doi:10.1038/gt.2014.8 · Cited by 103 · see citing works
Trapani, I., Toriello, E., de Simone, S., Colella, P., Iodice, C., Polishchuk, E. V., Sommella, A., Colecchi, L., Rossi, S., Simonelli, F., et al. (2015) · Human Molecular Genetics · doi:10.1093/hmg/ddv386 · Cited by 94 · see citing works
Gene therapy for retinal disease
McClements, M. E., & MacLaren, R. E. (2013) · Translational Research · doi:10.1016/j.trsl.2012.12.007 · Cited by 80 · see citing works
Adeno-Associated Viral Vectors as a Tool for Large Gene Delivery to the Retina
Trapani, I. (2019) · Genes · doi:10.3390/genes10040287 · Cited by 73 · see citing works
Marrone, L., Marchi, P. M., & Azzouz, M. (2022) · Expert Opinion on Biological Therapy · doi:10.1080/14712598.2022.2012148 · Cited by 56 · see citing works
“tute the full-length cDNA post-infection [97,98].”
- Authors
- Lopes, V. S., Boye, S. E., Louie, C. M., Boye, S., Dyka, F., Chiodo, V, Fofo, H., Hauswirth, W. W., & Williams, D. S.
- Affiliations
- University of California, Los Angeles (US)
- University of Florida (US)
- Corresponding author
- Williams, D S
- Venue
- Gene Therapy
- Published
- 2013-01-24
- Volume
- 20
- Issue
- 8
- Pages
- 824-833
- ISSN
- 0969-7128 (ISSN-L)
- 1476-5462
- PMC
- PMC3640772
- OpenAlex
- W2075794747
- Cited by
- 112
- References
- 53
- Citations by year
- 2026: 3 · 2025: 9 · 2024: 2 · 2023: 7 · 2022: 10 · 2021: 10 · 2020: 6 · 2019: 9 · 2018: 7 · 2017: 7 · 2016: 7 · 2015: 10 · 2014: 18 · 2013: 7
- FWCI
- 9.29
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Gene Therapy (publishedVersion)
- PubMed (publishedVersion)
- Europe PMC (PubMed Central) (acceptedVersion)
- PubMed Central (acceptedVersion)
- eScholarship (California Digital Library) (submittedVersion)
- Funders
- Foundation Fighting Blindness
- Macula Vision Research Foundation
- National Institutes of Health
- Fundação para a Ciência e a Tecnologia
- National Eye Institute (P30EY021721, EY-021721, EY-00331, R01EY007042, EY07042, P30EY000331)
- National Institute of General Medical Sciences (GM008243, T32GM008243)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Retinal Development and Disorders
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Keywords
- Usher syndrome type 1, MYO7A, retinal gene therapy, adeno-associated virus vectors, AAV2, AAV5, homologous recombination, photoreceptors, retinal pigment epithelium, deafness, retinal degeneration
- MeSH terms
- Myosin VIIa, Dependovirus, Animals, Humans, Myosins, Retina, Retinal Degeneration, Genetic Therapy, Gene Expression, Gene Transfer Techniques, DNA, Complementary, Green Fluorescent Proteins, Mice, Usher Syndromes
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
