Gene therapy clinical trials worldwide to 2012 – an update

  title={Gene therapy clinical trials worldwide to 2012 – an update},
  author={Samantha L. Ginn and Ian E. Alexander and Michael L. Edelstein and Mohammad R. Abedi and Joanne L. Wixon},
  journal={The Journal of Gene Medicine},
To date, over 1800 gene therapy clinical trials have been completed, are ongoing or have been approved worldwide. Our database brings together global information on gene therapy clinical trials from official agency sources, published literature, conference presentations and posters kindly provided to us by individual investigators or trial sponsors. 
Gene therapy clinical trials worldwide to 2017: An update
This review presents the analysis of clinical trials that, to the best of the knowledge, have been or are being performed worldwide, and discusses key trends since the previous review, namely the use of chimeric antigen receptor T cells for the treatment of cancer and advancements in genome editing technologies.
Clinical potential of gene therapy: towards meeting the demand
The current status of developments within the broad discipline of clinical gene therapy is provided, which offers the possibility of accurately and specifically targeting particular genetic abnormalities through gene correction, addition or replacement.
Gene therapies development: slow progress and promising prospect
The first gene therapy was approved in the European Union in 2012, after two decades of dashed expectations, boosted the investment in developing gene therapies and anticipated market launch in the upcoming years.
Gene therapy for heart disease: molecular targets, vectors and modes of delivery to myocardium
The authors discuss the current state of the art in terms of clinical and preclinical development, and examines vector technology and available vector-delivery strategies.
Gene therapy review.
  • J. A. Moss
  • Medicine, Biology
    Radiologic technology
  • 2014
An overview of roadblocks associated with each therapeutic methodology is presented, along with some of the scientific, social, and ethical issues associated with gene therapy.
Gene therapy for skin diseases.
The skin possesses qualities that make it desirable for gene therapy, and studies have focused on gene therapy for multiple cutaneous diseases. Gene therapy uses a vector to introduce genetic
Clinical translation of gene medicine
A comprehensive overview of the clinical translation of gene medicine is provided, focusing on the key events and latest progress made regarding clinical gene therapy products.


Gene therapy clinical trials worldwide to 2007—an update
This database set up in 1997 to bring together global information on gene therapy clinical trials as comprehensively as possible is compiled and regularly updated.
Current status of gendicine in China: recombinant human Ad-p53 agent for treatment of cancers.
Intratumoral injection of rAd-p53 in combination with cisplatin was well tolerated and there was evidence of clinical efficacy in patients treated, including patients with locally advanced transitional cell carcinoma of the bladder.
T Lymphocyte-Directed Gene Therapy for ADA− SCID: Initial Trial Results After 4 Years
It is concluded here that gene therapy can be a safe and effective addition to treatment for some patients with this severe immunodeficiency disease.
Report of a second serious adverse event in a clinical trial of gene therapy for X‐linked severe combined immune deficiency (X‐SCID)
The ESGT fully supported the investigators’ and French regulatory authority’s decision to put the trial on hold pending further investigations and published in this journal the second, serious adverse event to be reported in this trial.
Recent advances in gene therapy for severe congenital immunodeficiency diseases
Gene therapy for the most common forms of severe combined immune deficiency can lead to immune reconstitution in most patients, although a minority of patients has derived minimal clinical benefit and some have suffered severe adverse events including death.
Gene Therapy Death Prompts Review of Adenovirus Vector
The patient was the first in a gene therapy trial to die of the therapy itself; his death is the latest blow to a field that has been struggling to live up to the promise and hype surrounding the first gene therapy trials a decade ago.
One of three successfully treated CGD patients in a Swiss‐German gene therapy trial died due to his underlying disease
  • Medicine, Biology
    The journal of gene medicine
  • 2006
The principal investigators announced on May 23, 2006, that the patient’s death was most likely due to the underlying disease and not to a side effect of the gene therapy medicinal product administered, which may give important clues for further work standardizing gene therapy for severe cases of CGD.
A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency.
The sustained correction of X-linked severe combined immunodeficiency disease by ex vivo, retrovirally mediated transfer of the γc gene into CD34+ cells in four of five patients with the disease has been reported.
Gene therapy for SCID-X1: round 2.
  • R. Herzog
  • Biology, Medicine
    Molecular therapy : the journal of the American Society of Gene Therapy
  • 2010
Retroviral gene transfer of the common cytokine receptor γ-chain to bone marrow–derived hematopoietic stem and progenitor cells reconstituted development of functional T cells and thereby also partially restored the ability of the affected boys to mount proper B-cell responses.
Final Results From a Pivotal, Multicenter, International, Open-Label, Phase 2 Study of Romidepsin In Progressive or Relapsed Peripheral T-Cell Lymphoma (PTCL) Following Prior Systemic Therapy
These data support the potential for romidepsin in relapsed PTCL and suggest that it is possible to extend treatment for stable disease (SD) or response in future patients with cutaneous T-cell lymphoma.