Journal
FRONTIERS IN PEDIATRICS
Volume 7, Issue -, Pages -Publisher
FRONTIERS MEDIA SA
DOI: 10.3389/fped.2019.00443
Keywords
gene therapy; SCID; thalassemia; sickle cell disease; gene editing; lysosomal storage disorder; clinical trial; curative treatment
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Funding
- EU H2020 grant SCIDNET
- EU H2020 grant RECOMB
- Fondazione Telethon
- E-rare EUROCID
- European Union's Horizon 2020 research and innovation program
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Gene therapy using patient's own stem cells is rapidly becoming an alternative to allogeneic stem cell transplantation, especially when suitably compatible donors cannot be found. The advent of efficient virus-based methods for delivering therapeutic genes has enabled the development of genetic medicines for inherited disorders of the immune system, hemoglobinopathies, and a number of devastating metabolic diseases. Here, we briefly review the state of the art in the field, including gene editing approaches. A growing number of pediatric diseases can be successfully cured by hematopoietic stem-cell-based gene therapy.
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