Evidence map›Paper›PMID 30691064›Full record

ReviewViruses2019

Adeno-associated Virus (AAV) versus Immune Response.

Joseph Rabinowitz, Ying Kai Chan, Richard Jude Samulski

Open access · goldAbstract readReview
In one paragraph

Review in Viruses, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 82 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
82citing papers in PubMed, 1 pooled it
13.6field-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

82 citing papers in PubMed, 1 synthesis or guideline pooled it, 123 citations in OpenAlex.

  1. Pooled it
  2. Trial
  3. Review
  4. In Vivo T-Cell Engineering: Revolution in Delivery Strategies and Clinical Translation.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2026
    Review
  5. Review
  6. Review
  7. Optimizing a Subunit Vaccine ofIranian journal of public health · 2025
    Article
  8. AAV-Based Gene Therapy: Opportunities, Risks, and Scale-Up Strategies.International journal of molecular sciences · 2025
    Review
  9. Article
  10. A comprehensive atlas of AAV tropism in the mouse.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Article
  11. Review
  12. Article
  13. Review
  14. Neurosurgical gene therapy for central nervous system diseases.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2024
    Review
  15. Review
  16. Proof-of-concept for multiple AON delivery by a single U7snRNA vector to restore splicing defects in ABCA4.Molecular therapy : the journal of the American Society of Gene Therapy · 2024
    Article
  17. Review
  18. Review
  19. Review
  20. Article

22 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

3 authors at 3 institutions in 1 country.

Joseph RabinowitzSenior Director of Capsid Development, Rare Disease Research Unit, Pfizer Inc., 7030 Kit Creek Road, Morrisville, NC 27560, USA. Joseph.Rabinowitz@pfizer.com.
Ying Kai ChanDepartment of Genetics, Harvard Medical School, Boston, 02115, USA. YingKai_Chan@hms.harvard.edu.
Richard Jude SamulskiDepartment of Pharmacology, School of Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA. rjs@med.unc.edu.
Harvard University · USPfizer (United States) · USUniversity 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
NHLBI NIH HHS P01 HL112761
6 · The paper itself

Abstract

Decades ago, Friedmann and Roblin postulated several barriers to gene therapy, including tissue targeting, delivery across the blood⁻brain barrier (BBB), and host immune responses. These issues remain pertinent till today. Since then, several advances have been made in elucidating structures of adeno-associated virus (AAV) serotypes, antibody epitopes, and ways to modify antibody-binding sites. AAVs capsid has also been engineered to re-direct tissue tropism, reduce ubiquitination, and promote passage across the BBB. Furthermore, the use of high(er) dose recombinant AAV (rAAV) has been accompanied by a better understanding of immune responses in both experimental animals and early clinical trials, and novel work is being performed to modulate the immune response. While the immune responses to rAAV remains a major challenge in translating experimental drugs to approved medicine, and will likely require more than a single solution, we now better understand the hurdles to formulate and test experimental solutions to surmount them.

Indexed as

Genetic TherapyImmunity, InnateAdaptive ImmunityAnimalsCapsid ProteinsClinical Trials as TopicDependovirusGenetic VectorsHost Microbial InteractionsHumansMiceParvoviridae InfectionsCapsid Proteinsimmune responserAAVrare diseases

Identifiers

PMID30691064
PMCPMC6409805
OpenAlexW2911711918

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.