Evidence map›Paper›PMID 22737594›Full record

ArticleJournal of genetic syndromes & gene therapy2011

Advances in Overcoming Immune Responses following Hemophilia Gene Therapy.

Carol H Miao

Open access · greenAbstract read
In one paragraph

Article in Journal of genetic syndromes & gene therapy, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.

0numbers the graph read from it
0cells of the map it votes in
10citing papers in PubMed
1.7field-weighted citation impact, top 15% 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

10 citing papers in PubMed, 15 citations in OpenAlex.

  1. Nanomaterials Drive In Vivo CAR Immune Cells Engineering.Advanced materials (Deerfield Beach, Fla.) · 2026
    Review
  2. Article
  3. CD4Cellular immunology · 2020
    Article
  4. Article
  5. How to discuss gene therapy for haemophilia? A patient and physician perspective.Haemophilia : the official journal of the World Federation of Hemophilia · 2019
    Review
  6. Article
  7. Gene therapy for hemophilia.Frontiers in bioscience (Landmark edition) · 2015
    Review
  8. Inhibitors - cellular aspects and novel approaches for tolerance.Haemophilia : the official journal of the World Federation of Hemophilia · 2014
    Review
  9. Review
  10. 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

1 author at 1 institution in 1 country.

Carol H MiaoSeattle Children's Research Institute & University of Washington, Seattle WA, USA.
Seattle Children's Hospital · US

Funding

Non-viral Gene Medicine for the Treatment of HemophilaR01HL069049 · NHLBI · UNIVERSITY OF WASHINGTON · PI MIAO, CAROL H · 2003 to 2010
$3.9M
Immune Response for Hemophilia After Replacement TherapyR01HL082600 · NHLBI · SEATTLE CHILDREN'S HOSPITAL · PI MIAO, CAROL H · 2005 to 2008
$1.8M
NHLBI NIH HHS R01 HL069049NHLBI NIH HHS R01 HL082600
6 · The paper itself

Abstract

Both Clinical trials and pre-clinical experiments for hemophilia gene therapy showed that it is important to overcome potential immune responses against gene transfer vectors and/or transgene products to ensure the success of gene therapy. Recently various approaches have been investigated to prevent or modulate such responses. Gene transfer vectors have been specifically engineered and immunosuppressive regimens have been administered to avoid or manipulate the immune responses against the vectors. In order to prevent cytotoxic lymphocyte or antibody formation induced by transgene expression, novel approaches have been developed, including methods to manipulate antigen presentation, development of variant genes encoding less immunogenic proteins or gene transfer protocols to evade immune responses, as well as immunosuppressive strategies to target either T and/or B cell responses. Most of these successful protocols involve the induction of activated regulatory T cells to create a regulatory immune environment during tolerance induction. Recent development of these strategies to evade vector-specific immune responses and induce long-term immune tolerance specific to the transgene product will be discussed.

Identifiers

PMID22737594
PMCPMC3379895
OpenAlexW2064880501

What OpenQuestion holds

Textmetadata
LicenceTDM
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.