Gene Therapy That Works

  title={Gene Therapy That Works},
  author={Inder M. Verma},
  pages={853 - 855}
  • I. Verma
  • Published 23 August 2013
  • Biology, Medicine
  • Science
Gene therapy trials show a beneficial effect in children suffering from a neurodegenerative disorder or an immunodeficiency disease. [Also see Research Articles by Biffi et al. and Aiuti et al.] The concept of gene therapy is disarmingly simple: Introduce a healthy gene in a patient and its product should alleviate the defect caused by a faulty gene or slow the progression of disease (1). Why then, over the past three decades, have there been so few clinical successes in treating patients with… 
A snapshot of gene therapy in Latin America
The survey shows that the number of scientists involved in the development of gene therapy and DNA vaccines in Latin America is still very low and higher levels of investment are necessary to boost the advancement of innovation and intellectual property in this field.
Next-generation muscle-directed gene therapy by in silico vector design
Significantly higher and sustained human micro-dystrophin and follistatin expression levels are attained than when conventional promoters are used, resulting in robust phenotypic correction in dystrophic mice, without triggering apoptosis or evoking an immune response.
Gene Therapy: The View from NCATS
Support for gene therapy is considered across its different programs, including NCATS new initiatives to establish gene therapy-based platforms for rare diseases, which are a special area of focus for NCATS.
Human artificial chromosome-based gene delivery vectors for biomedicine and biotechnology
This review summarises recent work on gene transfer into mammalian cells using the HACs, which have a great potential for gene therapy, regenerative medicine, screening of anticancer drugs and biotechnology.
Lentivirus-Induced Dendritic Cells (iDC) for Immune-Regenerative Therapies in Cancer and Stem Cell Transplantation
It is demonstrated in mouse and human systems that a single overnight ex vivo lentiviral gene transfer into DC precursors for production of combination of cytokines and antigens was capable to induce autonomous self-differentiation of antigen-loaded DC in vitro and in vivo.
Platelets are efficient and protective depots for storage, distribution, and delivery of lysosomal enzyme in mice with Hurler Syndrome
Proof of concept that cells from megakaryocytic lineage and platelets are capable of generating and storing fully functional lysosomal enzymes and can also lead to efficient delivery of both the enzymes released into the circulation and those protected within platelets/microparticles.
Comparative analysis of cellular proteins in stably and transiently produced lentiviral vectors
Lentiviral vectors (LVs) are successfully used in clinical trials showing long term therapeutic benefits. Studying the role of cellular proteins during replication of lentivirus HIV-1 helped to
Human fetal liver cells for regulated ex vivo erythropoietin gene therapy
The study shows the potential of human liver endothelial cells for long-term regulated gene therapy, indicating that endothelial Cells are the most promising cell type for ex vivo liver cell gene therapy.


Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy
Lentiviral-mediated gene therapy of hematopoietic stem cells can provide clinical benefits in ALD, and progressive cerebral demyelination in the two patients stopped, a clinical outcome comparable to that achieved by allogeneic HCT.
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.
Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy
The reconstitution of ARSA activity in the cerebrospinal fluid and the arrested progression of neurodegenerative disease in the three treated patients demonstrate that the transplanted cells, or their progeny, can seed the nervous system and deliver therapeutic levels of active enzyme.
Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome
A clinical protocol based on lentiviral vector (LV) gene transfer into autologous hematopoietic stem/progenitor cells (HSCs) resulted in robust, stable, and long-term engraftment of gene-corrected HSCs in the patients’ bone marrow, and the findings support the use of LV gene therapy to treat patients with WAS.
Gene therapy - promises, problems and prospects
The prospects are good — by the year 2010, gene therapy may be as routine a practice as heart transplants are today.
Retroviral Integrations in Gene Therapy Trials
This review is aimed at critically revising the data derived from insertional profiling, with a particular focus on the evidences collected from GT clinical trials, to address broader biological questions, from basic virology to human hematopoiesis.
Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1.
These findings functionally specify a genetic network that controls growth in T cell progenitors and led to sustained remission in 3 of the 4 cases of T cell leukemia, but failed in the fourth.
Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease
It is shown that forced overexpression of EVI1 in human cells disrupts normal centrosome duplication, linking EVI 1 activation to the development of genomic instability, monosomy 7 and clonal progression toward myelodysplasia.
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.