The future of gene therapy

  title={The future of gene therapy},
  author={Marina Cavazzana‐Calvo and Adrian J. 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.

Antiangiogenic Cancer Therapy

Antiangiogenic cancer therapy / , Antiangiogenic cancer therapy / , کتابخانه دیجیتال جندی شاپور اهواز

Stem Cell Biology and Regenerative Medicine in Ophthalmology

  • S. Tsang
  • Medicine, Biology
    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.

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.

Genetic, cellular and immune approaches to disease therapy: past and future

  • G. Nabel
  • Biology, Medicine
    Nature Medicine
  • 2004
This Perspective is intended to give a sample of the progress over the past ten years in cellular, genetic and immune therapy of disease.

The future of HIV infection: gene therapy and RNA interference.

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.

Gene Therapy and Stem Cells in the Treatment of Congenital Diseases

This mini-review article focused on the platforms that have received the most attention and that are maturing in the clinical setting; in particular, the potential of in vivo gene therapy and human-induced pluripotent stem cells.

Suicidal gene therapy against tumor using reducible poly (oligo-D-arginine).




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.