ArticleRare diseases (Austin, Tex.)2014
Gene therapy for Wiskott-Aldrich Syndrome-Long-term reconstitution and clinical benefits, but increased risk for leukemogenesis.
Article in Rare diseases (Austin, Tex.), 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
10 citing papers in PubMed.
- Article
- Identification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells.Methods in molecular biology (Clifton, N.J.) · 2023Article
- Gene Therapy Developments for Pompe Disease.Biomedicines · 2022Review
- Gene Edited T Cell Therapies for Inborn Errors of Immunity.Frontiers in genome editing · 2022Review
- HIV-based lentiviral vectors: origin and sequence differences.Molecular therapy. Methods & clinical development · 2021Article
- CRISPR/Cas9 for the treatment of haematological diseases: a journey from bacteria to the bedside.British journal of haematology · 2021Review
- Isolation of a Highly Purified HSC-enriched CD34Transplantation direct · 2020Article
- Mouse models in hematopoietic stem cell gene therapy and genome editing.Biochemical pharmacology · 2020Review
- Reconstructing the immune system with lentiviral vectors.Virus genes · 2017Review
- Model Based Analysis of Clonal Developments Allows for Early Detection of Monoclonal Conversion and Leukemia.PloS one · 2016Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Wiskott-Aldrich-Syndrome (WAS) is a rare X-linked recessive disease caused by mutations of the WAS gene. It is characterized by immunodeficiency, autoimmunity, low numbers of small platelets (microthrombocytopenia) and a high risk of cancer, especially B cell lymphoma and leukemia.
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.