Encouraging and Unsettling Findings in Long-Term Follow-up of AAV Gene Transfer
Open accessAbstract
Gene therapy for genetic disease requires long-term therapeutic gene expression. Adeno-associated viral (AAV) vectors are considered ideal for this task when administered to non-dividing target cells. The value of these therapies becomes obvious during long-term follow-up. A study published last year showed that substantial improvements in the vision of patients that received ocular AAV gene transfer for correction of Leber’s congenital amaurosis, a rare form of inherited blindness, were stable for at least 4 years.
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?
AAV viral vectors provide valuable opportunities for clinical applications in the treatment of neurodegenerative diseases.
“Thus, AAV viral vectors provide valuable opportunities for clinical applications in the treatment of neurodegenerative diseases [5, 6].”
From Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside (2024) · cites this paper as [5]
The main text (page unavailable) describes AAV vectors as suitable for long-term gene therapy and reports clinical benefits for inherited blindness and hemophilia. It does not address treatment of neurodegenerative diseases, so it supports the general clinical potential of AAV vectors but not the disease-specific claim. (LLM, conf=0.86)
Experimental — accuracy not yet measured
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
Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A
Pasi, K. J., Rangarajan, S., Mitchell, N., Lester, W., Symington, E., Madan, B., Laffan, M., Russell, C. B., Li, M., Pierce, G. F., et al. (2020) · New England Journal of Medicine · doi:10.1056/nejmoa1908490 · Cited by 448 · see citing works
Maguire, A. M., Russell, S., Wellman, J. A., Chung, D. C., Yu, Z.-F., Tillman, A., Wittes, J., Pappas, J., Elci, O., Marshall, K. A., et al. (2019) · Ophthalmology · doi:10.1016/j.ophtha.2019.06.017 · Cited by 409 · cited 1× · see citing works
“A study published last year showed that substantial improvements in the vision of patients that received ocular AAV gene transfer for correction of Leber’s congenital amaurosis, a rare form of inherited blindness, were stable for at least 4 years.(1) However, very recent results raise new questions and concerns about…”
Background5 Year expression and neutrophil defect repair after gene therapy in alpha-1 antitrypsin deficiency
Mueller, C., Gernoux, G., Gruntman, A. M., Borel, F., Reeves, E. P., Calcedo, R., Rouhani, F. N., Yachnis, A., Humphries, M., Campbell-Thompson, M., et al. (2017) · Molecular Therapy · doi:10.1016/j.ymthe.2017.03.029 · Cited by 112 · see citing works
Kumar, S. R. P., Hoffman, B. E., Terhorst, C., de Jong, Y. P., & Herzog, R. W. (2017) · Molecular Therapy · doi:10.1016/j.ymthe.2017.02.014 · Cited by 83 · see citing works
Self-citationGernoux, G., Gruntman, A. M., Blackwood, M., Zieger, M. V., Flotte, T. R., & Mueller, C. (2020) · Molecular Therapy · doi:10.1016/j.ymthe.2020.01.004 · Cited by 44 · see citing works
Liver Gene Therapy: Reliable and Durable
Herzog, R. W., & Pierce, G. F. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2019.10.005 · Cited by 23 · see citing works
Self-citation
Flagged first, then by citations
Cited by
Sorted by citations
rAAV immunogenicity, toxicity, and durability in 255 clinical trials: A meta-analysis
Shen, W., Liu, S., & Ou, L. (2022) · Frontiers in Immunology · doi:10.3389/fimmu.2022.1001263 · Cited by 173 · see citing works
Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency
Tai, C., Lee, N., Chien, Y., Byrne, B. J., Muramatsu, S., Tseng, S., & Hwu, W. (2021) · Molecular Therapy · doi:10.1016/j.ymthe.2021.11.005 · Cited by 166 · see citing works
Immunogenicity of CRISPR therapeutics—Critical considerations for clinical translation
Ewaisha, R., & Anderson, K. S. (2023) · Frontiers in Bioengineering and Biotechnology · doi:10.3389/fbioe.2023.1138596 · Cited by 145 · see citing works
Xu, L., Yao, S., Ding, Y. E., Xie, M., Feng, D., Sha, P., Tan, L., Bei, F., & Yao, Y. (2024) · Journal of Translational Medicine · doi:10.1186/s12967-024-05661-2 · Cited by 48 · see citing works
Gernoux, G., Gruntman, A. M., Blackwood, M., Zieger, M. V., Flotte, T. R., & Mueller, C. (2020) · Molecular Therapy · doi:10.1016/j.ymthe.2020.01.004 · Cited by 44 · see citing works
Yang, Y.-S., Kim, J., Xie, J., Chaugule, S. R., Lin, C., Ma, H., Hsiao, E. C., Hong, J., Chun, H., Shore, E. M., et al. (2022) · Nature Communications · doi:10.1038/s41467-022-33956-9 · Cited by 43 · see citing works
Engineering monoclonal antibody-based contraception and multipurpose prevention technologies†
Anderson, D. J., Politch, J. A., Cone, R. A., Zeitlin, L., Lai, S. K., Santangelo, P. J., Moench, T. R., & Whaley, K. J. (2020) · Biology of Reproduction · doi:10.1093/biolre/ioaa096 · Cited by 33 · see citing works
Biswas, M., Marsic, D., Li, N., Zou, C., González‐Aseguinolaza, G., Zolotukhin, I., Kumar, S. R. P., Rana, J., Butterfield, J. S. S., Kondratov, O., et al. (2020) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2020.09.019 · Cited by 28 · see citing works
Self-citationEx vivo lung perfusion: recent advancements and future directions
Nakata, K., Alderete, I. S., Hughes, B. A., & Hartwig, M. G. (2025) · Frontiers in Immunology · doi:10.3389/fimmu.2025.1513546 · Cited by 28 · see citing works
Hemophilia Gene Therapy Value Assessment: Methodological Challenges and Recommendations
Garrison, L. P., Pezalla, E., Towse, A., Yang, H., Faust, E., Wu, E. Q., Li, N., Sawyer, E. K., & Recht, M. (2021) · Value in Health · doi:10.1016/j.jval.2021.05.008 · Cited by 25 · see citing works
Su, W., Sun, S., Tian, B., Tai, P. W. L., Luo, Y., Ko, J., Zhan, W., Xiao, K., Zheng, Q., Li, X., et al. (2021) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2021.06.007 · Cited by 23 · see citing works
Yang, Y.-S., Lin, C., Mā, H., Xie, J., Kaplan, F. S., Gao, G., & Shim, J. (2023) · Biomolecules · doi:10.3390/biom13091364 · Cited by 21 · see citing works
Liver-directed gene-based therapies for inborn errors of metabolism
Piccolo, P., Rossi, A., & Brunetti‐Pierri, N. (2020) · Expert Opinion on Biological Therapy · doi:10.1080/14712598.2020.1817375 · Cited by 16 · see citing works
Nature-inspired IL-1 targeted therapy to treat chronic inflammatory diseases
Yang, Y.-S., Kim, M., Chaugule, S. R., Mayer, E., DeSouza, N. H., Ma, H., Xie, J., Lee, K., Li, S., Gravallese, E. M., et al. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.09.008 · Cited by 7 · see citing works
Almaghrabi, S. A., Azzouz, M., & Tazi‐Ahnini, R. (2020) · Clinical & Translational Immunology · doi:10.1002/cti2.1166 · Cited by 7 · see citing works
Precision and efficacy of RNA-guided DNA integration in high-expressing muscle loci
Padmaswari, M. H., Bulliard, G. N., Agrawal, S., Jia, M. S., Khadgi, S., Murach, K. A., & Nelson, C. E. (2024) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2024.102320 · Cited by 5 · see citing works
BMP9 attenuates microgravity-related disuse osteoporosis by modulating TGFβ and BMP signaling
Zhao, H., Yang, M., Liu, Y., Tu, X., & Shao, G. (2025) · npj Microgravity · doi:10.1038/s41526-025-00510-y · Cited by 3 · see citing works
Moreno-Gutiérrez, D. S., Zepeda–Cervantes, J., Vaca, L. A., & Hernández-García, A. (2021) · Materials Science and Engineering C · doi:10.1016/j.msec.2021.112348 · Cited by 2 · see citing works
- Authors
- Herzog, R. W.
- Affiliations
- University of Florida Health (US)
- Corresponding author
- Herzog, Roland W.
- ORCID
- Herzog, Roland W.
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2020-02
- Volume
- 28
- Issue
- 2
- Pages
- 341-342
- ISSN
- 1525-0016 (ISSN-L)
- PMC
- PMC7001052
- OpenAlex
- W2999077142
- Cited by
- 18
- References
- 6
- Citations by year
- 2025: 3 · 2024: 2 · 2023: 2 · 2022: 2 · 2021: 4 · 2020: 5
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- Molecular Therapy (publishedVersion, cc-by-nc-nd)
- Europe PMC (PubMed Central) (publishedVersion)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- IUScholarWorks (Indiana University) (submittedVersion)
- Purdue University Indianapolis (Indiana University) (submittedVersion)
- 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
- adeno-associated virus vectors, gene therapy, long-term follow-up, therapeutic gene expression, Leber congenital amaurosis, vision improvement, inherited blindness, non-dividing cells
- MeSH terms
- Dependovirus, Animals, Follow-Up Studies, Genetic Vectors, Humans, Organ Specificity, Genetic Therapy, Gene Expression, Gene Transfer Techniques, Transgenes
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
