The DOI matches its Crossref record; 80 of the 80 references it cites were found in a registry or an index.
Progress in the Use of Gene Transfer Methods to Treat Genetic Blood Diseases
Abstract
A report by French physician-scientists suggests a successful application of gene transfer methods in the treatment of two children with severe combined immunodeficiency (SCID) due to defective interleukin 2 receptor common gamma chain. The protocol used in this clinical trial was derived from a number of preclinical and basic studies leading to improved transduction of hematopoietic stem and primitive progenitor cells using retrovirus vectors. These improvements have also been shown to impact transduction of a long-lived progenitor cell in a chemotherapy protocol in cancer patients. The improved results of these human trials come during a period of increased scrutiny and criticism of human gene therapy trials, due, in part, to significant toxicities in some trials using adenovirus-based vectors. The potential efficacy versus toxicity of phase I trials of human gene therapy is also under question. After many years of research, however, there appears to be real evidence that genetic diseases may be successfully treated by gene transfer techniques. Future clinical studies should be based on continued progress in the understanding of the toxicology of gene delivery systems, vector technology, and target cell manipulation.
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In the citing paper
“Neutrophils derived from normal human bone marrow progenitor cells transduced by the cloned D57N Rac2 cDNA mimic the patient's neutrophil phenotypes of decreased cell migration and superoxide production (28).”
From Biochemical and Biological Characterization of a Human Rac2 GTPase Mutant Associated with Phagocytic Immunodeficiency (2001) · cites this paper as [28]
In the citing paper
“All of these data suggest that the D57N protein had a dominant negative effect on WT Rac2 protein.”
From Biochemical and Biological Characterization of a Human Rac2 GTPase Mutant Associated with Phagocytic Immunodeficiency (2001) · cites this paper as [28]
Citations
Cites
In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector
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Gene Therapy of Human Severe Combined Immunodeficiency (SCID)-X1 Disease
Cavazzana-Calvo, M., Hacein-Bey, S., Basile, G. D. S., Gross, F., Yvon, E., Nusbaum, P., Selz, F., Hue, C., Certain, S., Casanova, J.-L., et al. (2000) · Science · doi:10.1126/science.288.5466.669 · Cited by 2,167
T Lymphocyte-Directed Gene Therapy for ADA − SCID: Initial Trial Results After 4 Years
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Site-specific integration by adeno-associated virus.
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Gene Therapy in Peripheral Blood Lymphocytes and Bone Marrow for ADA − Immunodeficient Patients
Bordignon, C., Notarangelo, L. D., Nobili, N., Ferrari, G., Casorati, G., Panina, P., Mazzolari, E., Maggioni, D., Rossi, C., Servida, P., et al. (1995) · Science · doi:10.1126/science.270.5235.470 · Cited by 832
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Research works printed but not linked: 3
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Cited by
Gu, Y., Jia, B., Yang, F.-C., D'Souza, M., Harris, C. E., Derrow, C. W., Zheng, Y., & Williams, D. A. (2001) · Journal of Biological Chemistry · doi:10.1074/jbc.m010445200 · Cited by 100
Self-citation
Metadata
- Authors
- Williams, D. A., & Smith, F. O.
- Venue
- Human Gene Therapy
- Publisher
- SAGE Publications
- Published
- 2000-10
- Volume
- 11
- Issue
- 15
- Pages
- 2059-2066
- ISSN
- 1043-0342
- 1557-7422
- Cited by
- 32
- References
- 80
- Language
- en
- Licence
- www.liebertpub.com/nv/resources-tools/text-and-data-mining-policy/121/ (tdm)
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- The paper
- Williams, D. A., & Smith, F. O. (2000). Progress in the Use of Gene Transfer Methods to Treat Genetic Blood Diseases. Human Gene Therapy. https://doi.org/10.1089/104303400750001372
Data sources
Bibliographic data: Semantic Scholar, OpenAlex, Crossref.
Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.
