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  1. 8 Νοε 2023 · In this article, we highlight success and ongoing challenges in three areas of high activity in gene therapy: inherited blood cell diseases by targeting hematopoietic stem cells, malignant...

  2. 1 Δεκ 2018 · This review focuses on the application of gene therapy for the correction of inherited diseases, the limitations and drawbacks encountered in some of the early clinical trials and the revival...

  3. 1 Ιαν 2019 · PDF | On Jan 1, 2019, Susanne Hempel published Gene Therapy in 2019: Milestones and Challenges | Find, read and cite all the research you need on ResearchGate.

  4. 8 Νοε 2023 · In this article, we highlight success and ongoing challenges in three areas of high activity in gene therapy: inherited blood cell diseases by targeting hematopoietic stem cells, malignant...

  5. 7 Μαΐ 2024 · To address this knowledge gap, this review summarizes core principles, benefits, potential risks and challenges of gene therapy, with a particular focus on hemophilia and spinal muscular atrophy, and discusses future perspectives.

  6. 27 Ιαν 2024 · Download Full Issue. Current challenges in cell and gene therapy: a joint view from the European Committee of the International Society for Cell & Gene Therapy (ISCT) and the European Society for Blood and Marrow Transplantation (EBMT)

  7. 9 Οκτ 2020 · Full size table. Disease targets and strategies for gene therapy. Gene therapy techniques are aimed at treating a variety of human diseases, including dominant and recessive genetic...

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