Evidence map›Paper›PMID 25807962›Full record

ReviewHuman gene therapy2015

Adeno-associated virus at 50: a golden anniversary of discovery, research, and gene therapy success--a personal perspective.

Eric Hastie, R Jude Samulski

Open access · hybridAbstract readHistorical ArticleReview
In one paragraph

Review in Human gene therapy, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 144 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
144citing papers in PubMed, 1 pooled it
17.0field-weighted citation impact, top 1% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

144 citing papers in PubMed, 1 synthesis or guideline pooled it, 269 citations in OpenAlex.

  1. Pooled it
  2. Article
  3. Review
  4. Review
  5. Article
  6. Article
  7. Article
  8. Genes & diseases · 2025
    Review
  9. Nanoparticle Carriers: A New Era of Precise CRISPR/Cas9 Gene Editing.MicroRNA (Shariqah, United Arab Emirates) · 2025
    Review
  10. Review
  11. Article
  12. Article
  13. Review
  14. A novel platform for engineered AAV-based vaccines.Molecular therapy. Methods & clinical development · 2025
    Article
  15. Article
  16. Review
  17. Review
  18. Review
  19. Article
  20. Article

84 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

2 authors at 1 institution in 1 country.

Eric Hastie1Gene Therapy Center, University of North Carolina, Chapel Hill, NC 27599-7352.
R Jude Samulski1Gene Therapy Center, University of North Carolina, Chapel Hill, NC 27599-7352.
University of North Carolina at Chapel Hill · US

Funding

VECTOR AND MOLECULAR BIOLOGY COREP01HL112761 · NHLBI · UNIV OF NORTH CAROLINA CHAPEL HILL · PI MONAHAN, PAUL E · 2013 to 2017
$11.3M
Rational and Combinatorial Engineering of AAV VectorsR01AI072176 · NIAID · UNIV OF NORTH CAROLINA CHAPEL HILL · PI HIRSCH, MATTHEW LOUIS, SAMULSKI, RICHARD J · 2008 to 2017
$3.7M
AAV Gene Therapy for AAT DeficiencyR01DK084033 · NIDDK · UNIV OF NORTH CAROLINA CHAPEL HILL · PI LI, CHENGWEN · 2009 to 2016
$2.2M
CTL response to AAV VectorR01AI080726 · NIAID · UNIV OF NORTH CAROLINA CHAPEL HILL · PI LI, CHENGWEN, SAMULSKI, RICHARD J · 2010 to 2014
$1.8M
Overcoming our clinical complications: AAV vector design for the treatment of DMDR01AR064369 · NIAMS · UNIV OF NORTH CAROLINA CHAPEL HILL · PI HIRSCH, MATTHEW LOUIS, SAMULSKI, RICHARD J · 2013 to 2017
$1.6M
NHLBI NIH HHS P01 HL112761NIAID NIH HHS R01 AI072176NIAID NIH HHS R01 AI080726NIAMS NIH HHS R01 AR064369NIDDK NIH HHS R01 DK084033
6 · The paper itself

Abstract

Fifty years after the discovery of adeno-associated virus (AAV) and more than 30 years after the first gene transfer experiment was conducted, dozens of gene therapy clinical trials are in progress, one vector is approved for use in Europe, and breakthroughs in virus modification and disease modeling are paving the way for a revolution in the treatment of rare diseases, cancer, as well as HIV. This review will provide a historical perspective on the progression of AAV for gene therapy from discovery to the clinic, focusing on contributions from the Samulski lab regarding basic science and cloning of AAV, optimized large-scale production of vectors, preclinical large animal studies and safety data, vector modifications for improved efficacy, and successful clinical applications.

Indexed as

Genetic TherapyGene Transfer TechniquesResearchAnimalsClinical Trials as TopicDependovirusDrug Evaluation, PreclinicalGenetic VectorsHistory, 20th CenturyHistory, 21st CenturyHumansTranslational Research, Biomedical

Identifiers

PMID25807962
PMCPMC4442590
OpenAlexW2025343661

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.