Evidence map›Paper›PMID 30815511›Full record

ReviewMolecular therapy. Methods & clinical development2019

Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors.

Hildegard Büning, Arun Srivastava

Abstract readReview
In one paragraph

Review in Molecular therapy. Methods & clinical development, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 166 papers.

0numbers the graph read from it
0cells of the map it votes in
166citing papers in PubMed
–field-weighted citation impact
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

166 citing papers in PubMed.

  1. Overview of the DREADDs chemogenetics and the patent landscape of their actuators.Medicinal chemistry research : an international journal for rapid communications on design and mechanisms of action of biologically active agents · 2026
    Review
  2. Article
  3. Article
  4. Distinct YY dinucleotide periodicity in adeno-associated virus DNA.Molecular therapy : the journal of the American Society of Gene Therapy · 2026
    Article
  5. Review
  6. Article
  7. Review
  8. Article
  9. Immunogenicity of Gene and Cell Therapies.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2026
    Review
  10. Article
  11. Review
  12. AAV capsid sites breakdown: large protein insertions impact on vector dynamics.Frontiers in bioengineering and biotechnology · 2026
    Article
  13. Article
  14. Review
  15. Article
  16. The amazing AAV capsids: Into the structure-verse.Molecular therapy. Methods & clinical development · 2025
    Review
  17. Article
  18. Review
  19. Review
  20. Review

106 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.

Hildegard BüningInstitute of Experimental Hematology, Hannover Medical School, Hannover, Germany.
Arun SrivastavaDivision of Cellular and Molecular Therapy, Departments of Pediatrics and Molecular Genetics & Microbiology, Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL, USA.

Funding

Next Generation of Recombinant AAV Serotype Vectors for Gene TherapyR01HL097088 · NHLBI · UNIVERSITY OF FLORIDA · PI GAO, GUANGPING, HERZOG, ROLAND W. · 2010 to 2018
$5.4M
Mechanism of high-efficiency transduction of hepatocytes by optimized AAV vectorsR01GM119186 · NIGMS · UNIVERSITY OF FLORIDA · PI ASLANIDI, GEORGE V, SRIVASTAVA, ARUN · 2017 to 2021
$1.5M
Safety and efficacy of optimized AAV3 vectors in murine and NHP liversR21EB015684 · NIBIB · UNIVERSITY OF FLORIDA · PI GAO, GUANGPING, SRIVASTAVA, ARUN · 2012 to 2013
$432k
AAV2 and hepatocellular carcinomaR21AI131328 · NIAID · UNIVERSITY OF FLORIDA · PI SRIVASTAVA, ARUN · 2017 to 2018
$408k
Development of Optimized AAV Serotype Vectors for Human Gene TherapyR41AI122735 · NIAID · MEDOSOME BIOTEC, LLC · PI SRIVASTAVA, ARUN · 2016 to 2016
$294k
NHLBI NIH HHS R01 HL097088NIAID NIH HHS R21 AI131328NIAID NIH HHS R41 AI122735NIBIB NIH HHS R21 EB015684NIGMS NIH HHS R01 GM119186
6 · The paper itself

Abstract

In the past decade, recombinant vectors based on a non-pathogenic parvovirus, the adeno-associated virus (AAV), have taken center stage as a gene delivery vehicle for the potential gene therapy for a number of human diseases. To date, the safety of AAV vectors in 176 phase I, II, and III clinical trials and their efficacy in at least eight human diseases are now firmly documented. Despite these remarkable achievements, it has also become abundantly clear that the full potential of first generation AAV vectors composed of naturally occurring capsids is not likely to be realized, since the wild-type AAV did not evolve for the purpose of therapeutic gene delivery. In this article, we provide a brief historical account of the progress that has been made in the development of capsid-modified

Identifiers

PMID30815511
PMCPMC6378346

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.