Evidence map›Paper›PMID 32425925›Full record

ReviewFrontiers in immunology2020

Translational Potential of Immune Tolerance Induction by AAV Liver-Directed Factor VIII Gene Therapy for Hemophilia A.

Benjamin J Samelson-Jones, Valder R Arruda

Open access · goldAbstract readReview
In one paragraph

Review in Frontiers in immunology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers.

0numbers the graph read from it
0cells of the map it votes in
27citing papers in PubMed
2.4field-weighted citation impact, top 9% 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

27 citing papers in PubMed, 36 citations in OpenAlex.

  1. Review
  2. Receptor-guided AAV Tropism Engineering via MATCH.bioRxiv : the preprint server for biology · 2026
    Article
  3. Review
  4. Review
  5. Review
  6. The curious case of AAV immunology.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
  7. Gene therapy for hemophilia - From basic science to first approvals of "one-and-done" therapies.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
  8. Article
  9. Review
  10. Review
  11. Review
  12. Article
  13. Article
  14. Immune tolerance induction by hepatic gene transfer: First-in-human evidence.Molecular therapy : the journal of the American Society of Gene Therapy · 2024
    Article
  15. Article
  16. From a bispecific monoclonal antibody to gene therapy: A new era in the treatment of hemophilia A.Biomedical papers of the Medical Faculty of the University Palacky, Olomouc, Czechoslovakia · 2023
    Review
  17. Review
  18. Review
  19. Review
  20. Article
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.

Benjamin J Samelson-JonesThe Children's Hospital of Philadelphia, Philadelphia, PA, United States.
Valder R ArrudaThe Children's Hospital of Philadelphia, Philadelphia, PA, United States.
Center for Molecular Medicine and Immunology · US

Funding

Skills DevelopmentU54HL142012 · NHLBI · CHILDREN'S HOSP OF PHILADELPHIA · PI CAMIRE, RODNEY M · 2018 to 2022
$7.0M
Engineering cellular immunotherapy to modulate immune responses in hemophiliaR01HL137335 · NHLBI · CHILDREN'S HOSP OF PHILADELPHIA · PI MILONE, MICHAEL C., SAMELSON-JONES, BEN J · 2017 to 2020
$1.7M
Rational Development of Bioengineered Factor IX Variants for Hemophilia B TherapyK08HL140078 · NHLBI · CHILDREN'S HOSP OF PHILADELPHIA · PI SAMELSON-JONES, BEN J · 2018 to 2021
$638k
NHLBI NIH HHS K08 HL140078NHLBI NIH HHS R01 HL137335NHLBI NIH HHS U54 HL142012
6 · The paper itself

Abstract

Hemophilia A (HA) is an X-linked bleeding disorder due to deficiencies in coagulation factor VIII (FVIII). The major complication of current protein-based therapies is the development of neutralizing anti-FVIII antibodies, termed inhibitors, that block the hemostatic effect of therapeutic FVIII. Inhibitors develop in about 20-30% of people with severe HA, but the risk is dependent on the interaction between environmental and genetic factors, including the underlying

Indexed as

Genetic TherapyImmune ToleranceAnimalsDependovirusDisease Models, AnimalDogsFactor VIIIHemophilia AHumansLiverFactor VIIIadeno-associated virusanti-drug antibodiesgene therapyhemophilia Aimmune toleranceinhibitors

Identifiers

PMID32425925
PMCPMC7212376
OpenAlexW3023187062

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.