Evidence map›Paper›PMID 18716130›Full record

ArticleBlood2008

Intraarticular factor IX protein or gene replacement protects against development of hemophilic synovitis in the absence of circulating factor IX.

Junjiang Sun, Narine Hakobyan, Leonard A Valentino, Brian L Feldman, R Jude Samulski, Paul E Monahan

Open access · greenAbstract readEvaluation Study
In one paragraph

Article in Blood, 2008. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 37 papers.

0numbers the graph read from it
0cells of the map it votes in
37citing papers in PubMed
2.1field-weighted citation impact, top 13% 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

37 citing papers in PubMed, 68 citations in OpenAlex.

  1. Article
  2. Article
  3. Article
  4. Rationally engineered novel AAV capsids for intra-articular gene delivery.Molecular therapy. Methods & clinical development · 2024
    Article
  5. Article
  6. Article
  7. Gene therapy approaches for equine osteoarthritis.Frontiers in veterinary science · 2022
    Review
  8. Article
  9. Article
  10. Hemophilia A and B mice, but not VWFScientific reports · 2019
    Article
  11. Article
  12. Article
  13. Article
  14. Review
  15. Application of polyploid adeno-associated virus vectors for transduction enhancement and neutralizing antibody evasion.Journal of controlled release : official journal of the Controlled Release Society · 2017
    Article
  16. Article
  17. Article
  18. Article
  19. Review
  20. Gene therapy in an era of emerging treatment options for hemophilia B.Journal of thrombosis and haemostasis : JTH · 2015
    Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

6 authors at 3 institutions in 1 country.

Junjiang SunGene Therapy Center, School of Medicine, University of North Carolina at Chapel Hill, NC 27599-7220, USA.
Narine Hakobyan
Leonard A Valentino
Brian L Feldman
R Jude Samulski
Paul E Monahan
University of North Carolina at Chapel Hill · USRush University Medical Center · USPediatrics and Genetics · US

Funding

Resolving rate limiting steps for AAV vectorsP01HL066973 · NHLBI · UNIVERSITY OF NORTH CAROLINA CHAPEL HILL · PI SAMULSKI, RICHARD J · 2001 to 2005
$9.3M
NHLBI NIH HHS P01 HL066973NHLBI NIH HHS P01 HL66973
6 · The paper itself

Abstract

Hemophilic bleeding into joints causes synovial and microvascular proliferation and inflammation (hemophilic synovitis) that contribute to end-stage joint degeneration (hemophilic arthropathy), the major morbidity of hemophilia. New therapies are needed for joint deterioration that progresses despite standard intravenous (IV) clotting factor replacement. To test whether factor IX within the joint space can protect joints from hemophilic synovitis, we established a hemophilia B mouse model of synovitis. Factor IX knockout (FIX(-/-)) mice received a puncture of the knee joint capsule with a needle to induce hemarthrosis; human factor IX (hFIX) was either injected through the needle into the joint space (intraarticularly) or immediately delivered IV. FIX(-/-) mice receiving intraarticular FIX protein were protected from synovitis compared with mice receiving same or greater doses of hFIX IV. Next, adeno-associated virus (AAV) gene transfer vectors expressing hFIX were injected into knee joints of FIX(-/-) mice. Joints treated with 10(10) vector genomes (vg)/joint AAV2-, AAV5-, or AAV8-hFIX or 2.5 x 10(9) vg/joint AAV5-hFIX developed significantly fewer pathologic changes 2 weeks after injury compared with the pathology of control injured contralateral hind limbs. Extravascular factor activity and joint-directed gene transfer may ameliorate hemophilic joint destruction, even in the absence of circulating FIX.

Indexed as

Genetic TherapyAnimalsBiological AvailabilityDependovirusDisease Models, AnimalFactor IXGenetic VectorsHemophilia BHemorrhageInjections, Intra-ArticularInjections, IntravenousJoint DiseasesMiceMice, Inbred C57BLMice, KnockoutSynovitisFactor IX

Identifiers

PMID18716130
PMCPMC2954682
OpenAlexW2014018178

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.