Gene therapy for severe combined immunodeficiencies and beyond.
Adenosine Deaminase
/ deficiency
Agammaglobulinemia
/ therapy
Animals
Gene Editing
Gene Transfer Techniques
Genetic Therapy
/ adverse effects
Genetic Vectors
/ genetics
Humans
Interleukin Receptor Common gamma Subunit
/ genetics
Leukemia
/ etiology
Mutagenesis, Insertional
/ methods
Mutation, Missense
Retroviridae
/ genetics
Severe Combined Immunodeficiency
/ therapy
T-Lymphocytes
/ immunology
Wiskott-Aldrich Syndrome
/ therapy
X-Linked Combined Immunodeficiency Diseases
/ therapy
Journal
The Journal of experimental medicine
ISSN: 1540-9538
Titre abrégé: J Exp Med
Pays: United States
ID NLM: 2985109R
Informations de publication
Date de publication:
06 01 2020
06 01 2020
Historique:
received:
30
08
2019
revised:
10
10
2019
accepted:
06
11
2019
entrez:
12
12
2019
pubmed:
12
12
2019
medline:
2
9
2020
Statut:
ppublish
Résumé
Ex vivo retrovirally mediated gene therapy has been shown within the last 20 yr to correct the T cell immunodeficiency caused by γc-deficiency (SCID X1) and adenosine deaminase (ADA) deficiency. The rationale was brought up by the observation of the revertant of SCIDX1 and ADA deficiency as a kind of natural gene therapy. Nevertheless, the first attempts of gene therapy for SCID X1 were associated with insertional mutagenesis causing leukemia, because the viral enhancer induced transactivation of oncogenes. Removal of this element and use of a promoter instead led to safer but still efficacious gene therapy. It was observed that a fully diversified T cell repertoire could be generated by a limited set (<1,000) of progenitor cells. Further advances in gene transfer technology, including the use of lentiviral vectors, has led to success in the treatment of Wiskott-Aldrich syndrome, while further applications are pending. Genome editing of the mutated gene may be envisaged as an alternative strategy to treat SCID diseases.
Identifiants
pubmed: 31826240
pii: 132743
doi: 10.1084/jem.20190607
pmc: PMC7041706
pii:
doi:
Substances chimiques
IL2RG protein, human
0
Interleukin Receptor Common gamma Subunit
0
Adenosine Deaminase
EC 3.5.4.4
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Review
Langues
eng
Sous-ensembles de citation
IM
Informations de copyright
© 2019 Fischer and Hacein-Bey-Abina.
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