2026-06-30T03:03:26Zhttps://repisalud.isciii.es/rest/oai/requestoai:repisalud.isciii.es:20.500.12105/261782025-12-18T12:55:54Zcom_20.500.12105_2327com_20.500.12105_2290col_20.500.12105_16991
Repisalud
author
Schambach, Axel
author
Buchholz, Christian J
author
Torres-Ruiz, Raul
author
Cichutek, Klaus
author
Morgan, Michael
author
Trapani, Ivana
author
Büning, Hildegard
author
Torres-Ruiz, Raul
funder
German Research Foundation (DFG)
funder
Federal Ministry of Education & Research (BMBF)
funder
United States Department of Health & Human Services National Institutes of Health (NIH) - USA
funder
Wellcome Trust
funder
Jose Carreras Leukemia Foundation
funder
European Research Council (ERC)
funder
European Innovation Council
2025-01-28T16:07:44Z
2025-01-28T16:07:44Z
2024-02-10
Lancet . 2024 Feb 10;403(10426):568-582.
Lancet
38006899
https://hdl.handle.net/20.500.12105/26178
Gene therapy has become a clinical reality as market-approved advanced therapy medicinal products for the treatment of distinct monogenetic diseases and B-cell malignancies. This Therapeutic Review aims to explain how progress in genome editing technologies offers the possibility to expand both therapeutic options and the types of diseases that will become treatable. To frame these impressive advances in the context of modern medicine, we incorporate examples from human clinical trials into our discussion on how genome editing will complement currently available strategies in gene therapy, which still mainly rely on gene addition strategies. Furthermore, safety considerations and ethical implications, including the issue of accessibility, are addressed as these crucial parameters will define the impact that gene therapy in general and genome editing in particular will have on how we treat patients in the near future.
eng
IN-VIVO
T-CELLS
GENOMIC-DNA
BASE
BACTERIAL
EFFICACY
VECTORS
PHASE-1
SYSTEM
A new age of precision gene therapy.
research article
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URL
https://repisalud.isciii.es/bitstreams/b887f080-7749-4a5e-8f78-5487eb650d60/download
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Anewageofprecision-2024.pdf
URL
https://repisalud.isciii.es/bitstreams/a22dafbb-da47-448d-b35f-78037d081815/download
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Carátula repisalud.docx