ArticleThe Journal of clinical investigation2004
Lenti in red: progress in gene therapy for human hemoglobinopathies.
Article in The Journal of clinical investigation, 2004. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 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
7 citing papers in PubMed, 15 citations in OpenAlex.
- Supplying clotting factors from hematopoietic stem cell-derived erythroid and megakaryocytic lineage cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2009Review
- Reprogramming erythroid cells for lysosomal enzyme production leads to visceral and CNS cross-correction in mice with Hurler syndrome.Proceedings of the National Academy of Sciences of the United States of America · 2009Article
- Genotoxic potential of lineage-specific lentivirus vectors carrying the beta-globin locus control region.Molecular therapy : the journal of the American Society of Gene Therapy · 2009Article
- Article
- The sea urchin sns5 insulator protects retroviral vectors from chromosomal position effects by maintaining active chromatin structure.Molecular therapy : the journal of the American Society of Gene Therapy · 2009Article
- Harnessing endogenous miR-181a to segregate transgenic antigen receptor expression in developing versus post-thymic T cells in murine hematopoietic chimeras.The Journal of clinical investigation · 2009Article
- Stem cell-derived erythroid cells mediate long-term systemic protein delivery.Nature biotechnology · 2006Article
Corrections and comments
- Comment on
Authors and funding
3 authors at 2 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Hemoglobinopathies are caused by abnormal structure or synthesis of hemoglobin chains and represent serious monogenic disorders. A new study demonstrates that lentiviral vectors can express clinically relevant levels of human transgenic beta-globin in red cells of xenografted mice. While some safety concerns must be addressed, this study is an important step toward potential clinical trials of gene therapy for hemoglobinopathies.
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.