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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. Gene therapy is a promising but challenging field that faces technical, financial, and ethical hurdles. Learn about the main challenges, such as gene delivery and activation, immune response, and gene integration, and how researchers overcome them.

  3. 9 Οκτ 2020 · Gene therapy techniques have been aimed at mitigating disease features of recessive and dominant disorders, as well as several cancers and other diseases.

  4. 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.

  5. 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 disorders using immune effector cells genetically modified with chimeric antigen receptors, and ophthalmologic, neurologic, and coagulation disorders using ...

  6. 8 Σεπ 2024 · Gene therapy clinical trials are predominantly in their initial phases (Figure 2B). Phase I trials account for the majority at 56.5%, closely followed by Phase I/II trials at 23.3%. ... An essential advantage of gene therapy is the elimination of the challenges posed by donor searches, making this approach universally applicable. 31. EMA's ...

  7. This webcast will explore how best to address challenges at each stage of in vivo AAV gene therapy discovery and development, from plasmid development and manufacture to clinical production...

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