The future of gene therapy

@article{CavazzanaCalvo2004TheFO,
  title={The future of gene therapy},
  author={Marina Cavazzana‐Calvo and Adrian J. Thrasher and Fulvio Mavilio},
  journal={Nature},
  year={2004},
  volume={427},
  pages={779-781}
}
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References

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Gene Therapy Insertional Mutagenesis Insights

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.

Gene Therapy--New Challenges Ahead

The discovery that 2 of the 10 patients with X-linked severe combined immunodeficiency developed leukemia within 3 years of gene therapy reinforces the need to develop even more specific gene therapy interventions.

Correction of ADA-SCID by Stem Cell Gene Therapy Combined with Nonmyeloablative Conditioning

Sustained engraftment of engineered HSCs with differentiation into multiple lineages resulted in increased lymphocyte counts, improved immune functions, and lower toxic metabolites, indicating the safety and efficacy of HSC gene therapy combined with nonmyeloablative conditioning for the treatment of SCID.

Side effects of retroviral gene transfer into hematopoietic stem cells.

It is the intention to emphasize the need for a critical and hypothesis-driven analysis of "transgene toxicology," in order to improve safety, efficiency, and prognosis for the yet small but expanding group of patients that could benefit from gene therapy.

Safety of retroviral gene marking with a truncated NGF receptor

Exposure to ∆LNGFR did not alter the function or survival of T lymphocytes derived from peripheral blood mononuclear cells transduced with a variety of vectors and studied in different animal models, and the risk of oncogenic transformation after transduction with a ∆ LNGFR-encoding retroviral vector was estimated to be <1 in 10 integration events.

Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease.

A gene therapy trial for SCID-X1 was initiated, based on the use of complementary DNA containing a defective gammac Moloney retrovirus-derived vector and ex vivo infection of CD34+ cells, which provided full correction of disease phenotype and clinical benefit.

LMO2-Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1

Retrovirus vector insertion can trigger deregulated premalignant cell proliferation with unexpected frequency, most likely driven by retrovirus enhancer activity on the LMO2 gene promoter.