ReviewExpert opinion on biological therapy2008
Development of lentiviral gene therapy for Wiskott Aldrich syndrome.
Review in Expert opinion on biological therapy, 2008. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01515462 (A Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Wiskott-Aldrich Syndrome), which is not on this map. Cited by 18 papers.
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.
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.
A Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Wiskott-Aldrich Syndrome
Who cites it
18 citing papers in PubMed, 53 citations in OpenAlex.
- Ex Vivo Gene and Cell Therapy in Hematopoietic Stem Cells.International journal of molecular sciences · 2025Review
- Identification of a novel WAS mutation and the non-splicing effect of a second-site mutation in a Chinese pedigree with Wiskott-Aldrich syndrome.Orphanet journal of rare diseases · 2022Article
- Hematopoietic Stem Cell Therapy for Wiskott-Aldrich Syndrome: Improved Outcome and Quality of Life.Journal of blood medicine · 2021Review
- Gene Therapy for Primary Immunodeficiency.HemaSphere · 2021Review
- Toward Combined Cell and Gene Therapy for Genodermatoses.Cold Spring Harbor perspectives in biology · 2020Review
- Managing Inflammatory Manifestations in Patients with Chronic Granulomatous Disease.Paediatric drugs · 2016Article
- Clinical applications of gene therapy for primary immunodeficiencies.Human gene therapy · 2015Review
- Cancer gene discovery: exploiting insertional mutagenesis.Molecular cancer research : MCR · 2013Review
- Review
- Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome.Molecular therapy : the journal of the American Society of Gene Therapy · 2013Article
- Autoimmunity in wiskott-Aldrich syndrome: an unsolved enigma.Frontiers in immunology · 2012Article
- Article
- Gene therapy of chronic granulomatous disease: the engraftment dilemma.Molecular therapy : the journal of the American Society of Gene Therapy · 2011Review
- Recent advances in lentiviral vector development and applications.Molecular therapy : the journal of the American Society of Gene Therapy · 2010Review
- Article
- Clinical and molecular characteristics of 35 Chinese children with Wiskott-Aldrich syndrome.Journal of clinical immunology · 2009Article
- Hematopoietic stem cell gene therapy for adenosine deaminase deficient-SCID.Immunologic research · 2009Article
- Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 3 institutions in 4 countries.
Funding
Abstract
backgroundWiskott Aldrich syndrome (WAS) is a rare X-linked primary immunodeficiency. This complex disease is characterised by microthrombocytopenia, recurrent infections, eczema and is associated with a high incidence of autoimmunity and of lymphoid malignancies. WAS is attracting growing attention not only because it highlights the rich cellular and systems biology revolving around cytoskeletal regulation but also because it is candidate for a haematopoietic stem cell gene therapy indication.
objectivesAs several groups are developing this novel approach, this review discusses the state of the art and challenges in clinical development of gene therapy for WAS, with particular regard to biosafety.
methodsIn spite of the successes of haematopoietic gene therapy for genetic immune deficiencies, there is a need for more efficient transduction protocols and for vectors with a superior safety profile. Preclinical studies have provided reasonable expectations that haematopoietic gene therapy with a self-inactivated HIV-1-derived vector using the native gene promoter for expression of the WAS transgene will be safe and will lead to the restoration of WAS protein in the haematopoietic and immune system at levels sufficient to provide an improvement in the condition of WAS patients.
conclusionsPhase I/II clinical studies will soon be initiated in several European centres to assess the safety and efficacy of this lentiviral vector in WAS patients.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
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.