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• W3108166834 abstract "Objectives: To analyze technological and ethical aspects of studies involving modern gene editing therapies registered in public trial registries: genetic diseases targeted for gene therapy; technologies used (CRISPR, ZFN, TALEN, viral vectors); status; if modern methods for off target and on target mutations were used as part of safety assessment: transparency of the trials, based on availability of publications, results posted to registry, IPD sharing and completeness of data. Methods: Registries were searched for “gene editing” and “genome editing” and eligible trials were analyzed. Results: 42 trials for single gene diseases were analyzed, and their characteristics were presented in tables. Zinc Finger Nuclease (ZFN) with adeno associated virus vector (AAV) was used for gene editing in vivo. CRISPR and ZFN techniques were used for ex vivo editing (autologous and allogeneic), mostly in Hematopoietic Stem Cells and CAR T cells. Most of the trials were open label, Phase 1 or 1/2, with 17% completed, one of them getting FDA approval. 21% withdrawn, terminated or of unknown status. Two of the studies, done in China, did editing on germline cells with major ethics violations, and one of them resulted in birth of genetically edited babies. Almost all trials did not post results to registry, did not share Individual Patient Data, and most lacked publications that reflected content of the trials. Conclusion: Advancements in gene editing techniques such as ZFN, CRISPR/Cas9, and use of safer viral vectors (AAV) as delivery system decreased the risk of insertional mutagenesis and cytotoxicity, and made possible genetic therapy of single gene disorders in humans, with various cancers, HIV, sickle cell disease, and thalassemia being the most often investigated. First gene therapy drug, approved by FDA for spinal muscular atrophy, Zolgensma, sets the standard for gene therapies, including high price and FDA warning for liver toxicity. Lack of results posted to registries, lack of publications that reflect content of the trial, unwillingness to share IPD, and incomplete data show the low level of transparency in gene editing trials. Measures to increase transparency, for example, proposed by World Health Organization creation of special registry dedicated to gene therapy and gene editing trials are needed." @default.
• W3108166834 created "2020-12-07" @default.
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• W3108166834 date "2020-09-30" @default.
• W3108166834 modified "2023-09-27" @default.
• W3108166834 title "CHARACTERISTICS OF GENE EDITING CLINICAL TRIALS FROM PUBLIC TRIAL REGISTRIES" @default.
• W3108166834 hasPublicationYear "2020" @default.
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