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Publication Detail
Genome editing for primary immunodeficiencies: A therapeutic perspective on Wiskott-Aldrich syndrome
  • Publication Type:
    Journal article
  • Authors:
    Naseem A, Steinberg Z, Cavazza A
  • Publisher:
    Frontiers Media SA
  • Publication date:
    2022
  • Journal:
    Frontiers in Immunology
  • Volume:
    13
  • Article number:
    966084
  • Medium:
    Electronic-eCollection
  • Status:
    Published
  • Country:
    Switzerland
  • Language:
    English
  • Keywords:
    CRISPR/Cas9, Wiskott-Aldrich syndrome, gene therapy, genome editing, primary immunodeficiency, rare diseases
  • Notes:
    © 2022 Naseem, Steinberg and Cavazza. This is an open-access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0/).
Abstract
Primary immunodeficiency diseases (PIDs) are a group of rare inherited disorders affecting the immune system that can be conventionally treated with allogeneic hematopoietic stem cell transplantation and with experimental autologous gene therapy. With both approaches still facing important challenges, gene editing has recently emerged as a potential valuable alternative for the treatment of genetic disorders and within a relatively short period from its initial development, has already entered some landmark clinical trials aimed at tackling several life-threatening diseases. In this review, we discuss the progress made towards the development of gene editing-based therapeutic strategies for PIDs with a special focus on Wiskott - Aldrich syndrome and outline their main challenges as well as future directions with respect to already established treatments.
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