Evidence map›Paper›PMID 32637455›Full record

ArticleMolecular therapy. Methods & clinical development2020

Development of AAV Variants with Human Hepatocyte Tropism and Neutralizing Antibody Escape Capacity.

Xiaolei Pei, Wenwei Shao, Allene Xing, Charles Askew, Xiaojing Chen, Caibin Cui, Yasmina L Abajas, David A Gerber, Elizabeth P Merricks, Timothy C Nichols and 3 more

Open access · goldAbstract read
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers.

0numbers the graph read from it
0cells of the map it votes in
21citing papers in PubMed
3.6field-weighted citation impact, top 7% 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

21 citing papers in PubMed, 29 citations in OpenAlex.

  1. Article
  2. Review
  3. Progress and Challenges in the Treatment of Fabry Disease.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2025
    Review
  4. Review
  5. Article
  6. Article
  7. Rationally engineered novel AAV capsids for intra-articular gene delivery.Molecular therapy. Methods & clinical development · 2024
    Article
  8. Review
  9. Gene Therapy Approaches for the Treatment of Hemophilia B.International journal of molecular sciences · 2023
    Review
  10. Article
  11. Review
  12. AAV vectors: The Rubik's cube of human gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2022
    Review
  13. Evaluating the state of the science for adeno-associated virus integration: An integrated perspective.Molecular therapy : the journal of the American Society of Gene Therapy · 2022
    Review
  14. Human Hepatocyte Transduction with Adeno-Associated Virus Vector.Methods in molecular biology (Clifton, N.J.) · 2022
    Article
  15. Article
  16. Frontiers in microbiology · 2022
    Review
  17. Review
  18. The intersection of vector biology, gene therapy, and hemophilia.Research and practice in thrombosis and haemostasis · 2021
    Review
  19. Article
  20. Review
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

13 authors at 2 institutions in 2 countries.

Xiaolei PeiInstitute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, China.
Wenwei ShaoGene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
Allene XingGene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
Charles AskewGene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
Xiaojing ChenGene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
Caibin CuiDepartment of Surgery, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
Yasmina L AbajasDepartment of Pediatrics, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
David A GerberDepartment of Surgery, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
Elizabeth P MerricksDepartment of Pathology and Laboratory Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
Timothy C NicholsDepartment of Pathology and Laboratory Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
Wuping LiInstitute of Pathogen Biology, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China.
R Jude SamulskiGene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
Chengwen LiGene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
University of North Carolina at Chapel Hill · USChinese Academy of Medical Sciences & Peking Union Medical College · CN

Funding

Virology Research Program (Program 4)P30CA016086 · NCI · UNIV OF NORTH CAROLINA CHAPEL HILL · PI David Darr · 1985 to 2026
$201.5M
VECTOR AND MOLECULAR BIOLOGY COREP01HL112761 · NHLBI · UNIV OF NORTH CAROLINA CHAPEL HILL · PI MONAHAN, PAUL E · 2013 to 2017
$11.3M
Optimization of AAV vector to deliver FVa for hemophilia with inhibitorsR01HL144661 · NHLBI · UNIV OF NORTH CAROLINA CHAPEL HILL · PI LI, CHENGWEN, NICHOLS, TIMOTHY CHARLES · 2019 to 2022
$2.2M
Enhance AAV Liver Transduction with Capsid Immune EvasionR01AI117408 · NIAID · UNIV OF NORTH CAROLINA CHAPEL HILL · PI LI, CHENGWEN, SAMULSKI, RICHARD J · 2016 to 2020
$2.0M
Directed evolution of AAV vectors for hemophilia to evade neutralizationR01HL125749 · NHLBI · UNIV OF NORTH CAROLINA CHAPEL HILL · PI LI, CHENGWEN, NICHOLS, TIMOTHY CHARLES · 2015 to 2018
$1.5M
NCI NIH HHS P30 CA016086NHLBI NIH HHS P01 HL112761NHLBI NIH HHS R01 HL125749NHLBI NIH HHS R01 HL144661NIAID NIH HHS R01 AI117408
6 · The paper itself

Abstract

Adeno-associated virus (AAV) vectors have been successfully used in patients with bleeding disorders and blindness. For human liver targeting, two major factors restrict effective AAV transduction after systemic administration of AAV vectors: human hepatocyte tropism and neutralizing antibodies (Nabs). In this study, we attempted to isolate AAV variants with the ability to transduce human hepatocytes and escape Nabs using a directed evolution approach

Indexed as

AAVchimeric micehuman hepatocyteNabstropism

Identifiers

PMID32637455
PMCPMC7329936
OpenAlexW3033229872

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.