The future of gene therapy

  title={The future of gene therapy},
  author={Marina Cavazzana‐Calvo and Adrian James Thrasher and Fulvio Mavilio},
Balancing the risks and benefits of clinical trial. 
Development of CXCR4-Inhibiting Nanoparticles for the Treatment of Metastatic Cancer
Development of CXCR4-Inhibiting Nanoparticles for the Treatment of Metastatic Cancer is progressing well and data is expected to be published in the Journal of Clinical Oncology later this year. Expand
European attitudes to gene therapy and pharmacogenetics.
Views on pharmacogenetics and gene therapy systematically differ across European countries. But despite a complex regulatory regime there is a balance of support, albeit laced with considerableExpand
Antiangiogenic Cancer Therapy
Antiangiogenic cancer therapy / , Antiangiogenic cancer therapy / , کتابخانه دیجیتال جندی شاپور اهواز
A review of gene therapy for haematological disorders
The current status of gene therapy is reviewed and the challenges faced by this emerging technology that holds so much promise for many suffering from catastrophic disorders are outlined. Expand
Stem Cell Biology and Regenerative Medicine in Ophthalmology
  • S. Tsang
  • Medicine
  • Stem Cell Biology and Regenerative Medicine
  • 2013
This research presents a novel and scalable approaches that combine stem cell research and regenerative medicine in ophthalmology with real-time information about the response of the immune system to single-cell transplants. Expand
Genetic, cellular and immune approaches to disease therapy: past and future
This Perspective is intended to give a sample of the progress over the past ten years in cellular, genetic and immune therapy of disease. Expand
The future of HIV infection: gene therapy and RNA interference.
A previously unrecognized pathway in which small interfering, 21 to 23 mer, double-stranded RNA (siRNA) mediates sequence-specific degradation of mRNA is becoming one the most useful techniques in cell biology and genetics research. Expand
Gene therapy in the treatment of heart failure.
Advances in the understanding of the molecular basis of conditions such as heart failure, together with the evolution of increasingly efficient gene transfer technology, has placed congestive heart failure within reach of gene-based therapy. Expand
Suicidal gene therapy against tumor using reducible poly (oligo-D-arginine).
Summary Reducible poly(oligo-D-arginine) (rPOA) is a promising non-viral gene delivery carrier, providing lower cytotoxicity and higher gene expression efficiency compare to PEI. rPOA showed a higherExpand
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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. Expand
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. Expand
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. Expand
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. Expand
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. Expand
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. Expand
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. Expand
Primary immunodeficiency diseases: an experimental model for molecular medicine
About three quarters of 100 primary immunodeficiency diseases can now be reliably diagnosed with molecular probes, and gene identification has enabled significant insight into the physiopathology of the related conditions. Expand
Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968–99
The improvement in survival over time indicates more effective prevention and treatment of disease-related and procedure-related complications and graft versus host disease in the HLA-non-identical setting by use of more efficient methods of T-cell depletion. Expand