Evidence map›Paper›PMID 34254844›Full record

ArticleHuman gene therapy2021

Chemical Modifications of the Capsid for Redirecting and Improving the Efficacy of Adeno-Associated Virus Vectors.

Anh K Lam, Dylan Frabutt, Lei Li, Weidong Xiao

Abstract read
In one paragraph

Article in Human gene therapy, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.

0numbers the graph read from it
0cells of the map it votes in
17citing 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

17 citing papers in PubMed.

  1. Article
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  7. Identification of a robust promoter in mouse and human hepatocytes by in vivo biopanning of a barcoded AAV library.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Article
  8. The deLIVERed promises of gene therapy: Past, present, and future of liver-directed gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
  9. Review
  10. Article
  11. Article
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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

4 authors.

Anh K LamDepartment of Pediatrics, Herman B Wells Center for Pediatrics Research, Indiana University School of Medicine, Indianapolis, Indiana, USA.ORCID 0000-0002-8830-9349
Dylan FrabuttDepartment of Pediatrics, Herman B Wells Center for Pediatrics Research, Indiana University School of Medicine, Indianapolis, Indiana, USA.
Lei LiDepartment of Chemistry, Georgia State University, Atlanta, Georgia, USA.
Weidong XiaoDepartment of Pediatrics, Herman B Wells Center for Pediatrics Research, Indiana University School of Medicine, Indianapolis, Indiana, USA.

Funding

BASIC SCIENCE STUDIES ON GENE THERAPY OF BLOOD DISEASEST32HL007910 · NHLBI · INDIANA UNIV-PURDUE UNIV AT INDIANAPOLIS · PI Roland W. Herzog, Reuben Kapur · 1999 to 2026
$7.6M
Temple Project 1: Genetic characterization of factor VIII Inhibitors and glydosylation patternsU54HL142019 · NHLBI · TEMPLE UNIV OF THE COMMONWEALTH · PI LI, LEI, MIAO, CAROL H · 2018 to 2022
$6.9M
Development of highly efficient factor VIII mini-gene therapyR01HL130871 · NHLBI · TEMPLE UNIV OF THE COMMONWEALTH · PI XIAO, WEIDONG · 2016 to 2019
$1.9M
NHLBI NIH HHS R01 HL130871NHLBI NIH HHS T32 HL007910NHLBI NIH HHS U54 HL142019
6 · The paper itself

Abstract

Adeno-associated virus (AAV) vector-directed gene therapy is one of the most exciting modalities of biotechnology as more applications enter clinical stage. Although AAV vectors generally feature low toxicity, high stability, and long-lasting transgene expression, potential challenging issues of AAV include high vector dose, limited tissue tropism, and the host immune response and inflammation, which are all related to the capsid protein. To overcome these challenges, various strategies have been developed to engineer AAV capsids. Apart from widely employed genetic engineering of capsid protein, powerful and versatile chemical modification strategies are underexploited. This minireview summarizes recent advances and our perspectives for future direction in AAV capsid chemical modification to enhance its therapeutic use for gene therapy.

Indexed as

CapsidDependovirusCapsid ProteinsGenetic TherapyGenetic VectorsCapsid ProteinsAAVadeno-associated viruscapsid engineeringcapsid modificationchemical modification

Identifiers

PMID34254844
PMCPMC8905246

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.