Gene Therapy Insertional Mutagenesis Insights
Explore this paper's citation graph
Summary
In a milestone study describing the first “cure” of a genetic disease by retroviral gene therapy, 9 out of 10 infants born with X-linked severe combined immunodeficiency were successfully treated with autologous bone marrow stem cells infected with Tournaisian stem cells.
- Type
- article
- Published
- 2004-01-16
- Cited by
- 272
- References
- 7
- OpenAlex
- https://openalex.org/W1967098175
- Semantic Scholar
- https://api.semanticscholar.org/CorpusID:35124200
Keywords
Insertional mutagenesis, Genetic enhancement, Mutagenesis, Severe combined immunodeficiency, Milestone
References
- Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease.
- TTG-2, a new gene encoding a cysteine-rich protein with the LIM motif, is overexpressed in acute T-cell leukaemia with the t(11;14)(p13;q11).
- The rhombotin family of cysteine-rich LIM-domain oncogenes: distinct members are involved in T-cell translocations to human chromosomes 11p15 and 11p13.
- Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy.
- LMO2-Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1
- A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency.
Cited by
- Tendon healing in vitro: genetic modification of tenocytes with exogenous PDGF gene and promotion of collagen gene expression.
- Gene therapy for severe combined immunodeficiency.
- Structure-based prediction of insertion-site preferences of transposons into chromosomes
- The development of gene therapy: from monogenic recessive disorders to complex diseases such as cancer.
- Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1.
- Detection of replication competent retrovirus and lentivirus.
- Recombinant Adeno-Associated Virus-Mediated Gene Transfer for the Potential Therapy of Adenosine Deaminase-Deficient Severe Combined Immune Deficiency
- Programming stem cells for therapeutic angiogenesis using biodegradable polymeric nanoparticles.
- AAV vectors for gene correction.
- Los elementos genéticos móviles en la célula tumoral: el despertar de un gigante
- Gene therapy to induce cellular resistance to HIV-1 infection: lessons from clinical trials.
- Murine models of human acute myeloid leukemia.
- Reducing the genotoxic potential of retroviral vectors.
- Adenovirus and its Vector for Developing Vaccines Against Biological Warfare Agents
- Dévelappement de vecteurs lentiviraux non-intergratifs en vue du transfert de gènes dans le système nerveux central.
- Current Status and Future Prospects of Gene Therapy
- Mécanisme de leucémogénèse par les oncogènes SCL et LMO1
- deficiency using ex-vivo hematopoietic stem cell gene therapy Correction of the disease phenotype in canine leukocyte adhesion
- Screening for Candidate Brain Tumor Genes : Identifying Genes that Cooperate with Platelet-Derived Growth Factor in Glioma Development and Progression
- Investigation of the effects of virus integration on host gene expression in mouse tumour samples
Related papers
- Insertional versus targeted mutagenesis in mice.
- [Development of retrovirus-mediated insertional mutagenesis in zebrafish and its application in saturation mutagenesis and gene screening].
- Mutagenesis in the post genomics era: tools for generating insertional mutations in the lactic acid bacteria.
- Insertional mutagenesis in transgenic mice
- Using retroviruses as a mutagenesis tool to explore the zebrafish genome.
- Insertion-duplication mutagenesis of Salmonella enterica and related species using a novel thermosensitive vector.
- New insights and unresolved issues regarding insertional mutagenesis in X-linked SCID gene therapy.