{
  "abstract": "Significant progress has been made in adeno-associated virus (AAV)-based gene therapy. However, host immune responses remain a major challenge to its widespread clinical application, making immunosuppressive regimens an important component of clinical management. We performed a systematic review and meta-analysis to characterize immunosuppressive regimens and immune-related outcomes in clinical AAV gene therapy. The primary outcomes were the proportions of humoral immune responses, gene therapy-associated uveitis (GTAU), elevated alanine aminotransferase (ALT), and elevated creatine kinase (CK). A total of 73 studies involving 1773 participants were included and categorized as ocular (n = 31), liver (n = 22), muscle (n = 12), or central nervous system (n = 8) according to the primary target organ. Primary subgroup analyses were restricted to studies in which immunosuppression was used prophylactically. The pooled proportions of humoral immune responses were 27.7%, 75.8%, 35.2%, and 42.6% for ocular-, liver-, muscle-, and central nervous system-directed AAV gene therapy, respectively. The pooled proportions of GTAU in ocular-directed studies, elevated ALT in liver-directed studies, elevated CK in muscle-directed studies, and elevated ALT in central nervous system-directed studies were 24.2%, 49.5%, 5.1%, and 16.5%, respectively. Statistically significant regimen-based subgroup differences were observed for GTAU in ocular-directed studies and for humoral immune responses in muscle-directed studies. However, the current evidence does not establish the superiority of any specific immunosuppressive regimen. These findings highlight variation in immune-related outcomes across target organs and the need for more clinical studies with standardized outcome reporting to better inform immunosuppressive strategies in AAV gene therapy.",
  "abstract_shown": true,
  "affiliations": [],
  "article_type": null,
  "arxiv": null,
  "author_total": 5,
  "authors": [
    {
      "name": "Ziyang Wang"
    },
    {
      "name": "Ni Wu"
    },
    {
      "name": "Diyi Fu"
    },
    {
      "name": "Qian Zhao",
      "orcid": "0000-0001-9438-1059"
    },
    {
      "name": "Hongyun Wang",
      "orcid": "0000-0002-8852-6490"
    }
  ],
  "bibtex": "https://research.draconitic.com/paper/8cbznjb0ctfgt.bib",
  "cited_by": 0,
  "doi": "10.1038/s41598-026-71872-w",
  "fulltext_url": null,
  "id": "8cbznjb0ctfgt",
  "issns": [
    "2045-2322"
  ],
  "landing_url": "https://www.nature.com/articles/s41598-026-71872-w",
  "language": null,
  "licence": "CC-BY-4.0",
  "licence_label": "CC BY 4.0",
  "licence_source": "article",
  "licence_url": "https://creativecommons.org/licenses/by/4.0",
  "metadata_licence": null,
  "native_subjects": [
    [
      "openalex",
      "1311|Virus-based gene therapy research"
    ]
  ],
  "openalex": "W7215106218",
  "pdf_url": "https://www.nature.com/articles/s41598-026-71872-w_reference.pdf",
  "pmcid": null,
  "pmid": null,
  "published": "2026-10-01",
  "publisher": "Springer Science and Business Media LLC",
  "ris": "https://research.draconitic.com/paper/8cbznjb0ctfgt.ris",
  "sources": [
    {
      "first_seen": "2026-10-30T12:17:19Z",
      "native_id": "10.1038/s41598-026-71872-w",
      "source": "crossref",
      "url": "https://www.nature.com/articles/s41598-026-71872-w"
    }
  ],
  "subjects": [
    {
      "field": "biology",
      "origin": "openalex:1311|Virus-based gene therapy research",
      "subfield": "genetics-genomics"
    }
  ],
  "title": "A systematic review and meta-analysis of immunosuppressive regimens and immune-related outcomes in adeno-associated virus-based gene therapy",
  "title_text": "A systematic review and meta-analysis of immunosuppressive regimens and immune-related outcomes in adeno-associated virus-based gene therapy",
  "type": "peer-reviewed",
  "updated_at": "2026-10-30T12:17:19Z",
  "url": "https://research.draconitic.com/paper/8cbznjb0ctfgt",
  "venue": "Scientific Reports",
  "versions": []
}