Evidence map›Paper›PMID 18634839›Full record

ArticleJournal of controlled release : official journal of the Controlled Release Society2008

Gene transfer to hemophilia A mice via oral delivery of FVIII-chitosan nanoparticles.

Katherine Bowman, Rita Sarkar, Sanj Raut, Kam W Leong

Open access · greenAbstract read
In one paragraph

Article in Journal of controlled release : official journal of the Controlled Release Society, 2008. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 papers.

0numbers the graph read from it
0cells of the map it votes in
28citing papers in PubMed
2.9field-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

28 citing papers in PubMed, 91 citations in OpenAlex.

  1. Review
  2. Article
  3. Review
  4. Review
  5. Review
  6. Formulation of Chitosan-Zein Nano-in-Microparticles for Oral DNA Delivery.Methods in molecular biology (Clifton, N.J.) · 2024
    Article
  7. Formulation of Chitosan-Zein Nano-in-Microparticles for Oral DNA Delivery.Methods in molecular biology (Clifton, N.J.) · 2024
    Article
  8. Review
  9. Article
  10. Oral Nonviral Gene Delivery for Chronic Protein Replacement Therapy.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2018
    Article
  11. Chitosan-zein nano-in-microparticles capable of mediating in vivo transgene expression following oral delivery.Journal of controlled release : official journal of the Controlled Release Society · 2017
    Article
  12. Immune Tolerance for Autoimmune Disease and Cell Transplantation.Annual review of biomedical engineering · 2016
    Review
  13. Gene therapy for hemophilia.Frontiers in bioscience (Landmark edition) · 2015
    Review
  14. Article
  15. Review
  16. Review
  17. Chitosans for delivery of nucleic acids.Advanced drug delivery reviews · 2013
    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

4 authors at 4 institutions in 2 countries.

Katherine BowmanDepartment of Biomedical Engineering, Johns Hopkins University School of Medicine, Baltimore, MD 21205, USA.
Rita Sarkar
Sanj Raut
Kam W Leong
Duke University · USJohns Hopkins University · USNational Institute for Biological Standards and Control · GBUniversity of Pennsylvania · US

Funding

Vector CoreP30DK047757 · NIDDK · UNIVERSITY OF PENNSYLVANIA · PI WILSON, JAMES M · 1993 to 2017
$18.0M
Mechanistic Studies on Polymeric Controlled Oral GeneR01EB002849 · NIBIB · JOHNS HOPKINS UNIVERSITY · PI LEONG, KAM W · 2003 to 2007
$1.3M
NIBIB NIH HHS EB002849NIBIB NIH HHS R01 EB002849NIDDK NIH HHS P30 DK047757NIDDK NIH HHS P30 DK047757-11
6 · The paper itself

Abstract

Effective oral delivery of a non-viral gene carrier would represent a novel and attractive strategy for therapeutic gene transfer. To evaluate the potential of this approach, we studied the oral gene delivery efficacy of DNA polyplexes composed of chitosan and Factor VIII DNA. Transgene DNA was detected in both local and systemic tissues following oral administration of the chitosan nanoparticles to hemophilia A mice. Functional factor VIII protein was detected in plasma by chromogenic and thrombin generation assays, reaching a peak level of 2-4% FVIII at day 22 after delivery. In addition, a bleeding challenge one month after DNA administration resulted in phenotypic correction in 13/20 mice given 250-600 microg of FVIII DNA in chitosan nanoparticles, compared to 1/13 mice given naked FVIII DNA and 0/6 untreated mice. While further optimization would be required to render this type of delivery system practical for hemophilia A gene therapy, the findings suggest the feasibility of oral, non-viral delivery for gene medicine applications.

Indexed as

NanoparticlesTransfectionAdministration, OralAnimalsChitosanDisease Models, AnimalDNADogsFactor VIIIFeasibility StudiesGenetic TherapyHemophilia AHemostasisHumansMiceMice, KnockoutChitosanDNAFactor VIIIThrombin

Identifiers

PMID18634839
PMCPMC2754405
OpenAlexW2035618175

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.