Rare Diseases Symptoms Automatic Extraction

Progress in gene therapy for primary immunodeficiencies using lentiviral vectors.

[wiskott-aldrich syndrome]

This review gives an overview over the most recent progress in the field of lentiviral gene therapy for primary immunodeficiencies (PIDs). The history and state-of-the-art of lentiviral vector development are summarized and the recent advancements for a number of selected diseases are reviewed in detail. Past retroviral vector trials for these diseases, the most recent improvements of lentiviral vector platforms and their application in preclinical development as well as ongoing clinical trials are discussed.Main focus is on the preclinical studies and clinical trials for the treatment of Wiskott-Aldrich syndrome, chronic granulomatous disease, adenosine deaminase deficient severe combined immunodeficiency (ADA-SCID) and X-linked severe combined immunodeficiency with lentiviral gene therapy.Gene therapy for PIDs is an effective treatment, providing potential long-term clinical benefit for affected patients. Substantial progress has been made to make lentiviral gene therapy platforms available for a number of rare genetic diseases. Although many ongoing gene therapy trials are based on ex-vivo approaches with autologous hematopoietic stem cells, other approaches such as in-vivo gene therapy or gene-repair platforms might provide further advancement for certain PIDs.

Diseases presenting "severe combined immunodeficiency" symptom

  • achondroplasia
  • alpha-thalassemia
  • child syndrome
  • cholangiocarcinoma
  • junctional epidermolysis bullosa
  • krabbe disease
  • omenn syndrome
  • severe combined immunodeficiency
  • wiskott-aldrich syndrome

This symptom has already been validated