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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...
- (PDF) Gene therapy: advances, challenges and perspectives - ResearchGate
Gene therapy is proven to be a potential disease-modifying...
- (PDF) Successes and challenges in clinical gene therapy - ResearchGate
In this article, we highlight success and ongoing challenges...
- (PDF) Gene therapy: advances, challenges and perspectives - ResearchGate
1 Σεπ 2017 · Gene therapy is proven to be a potential disease-modifying treatment for AD. This article discusses about gene therapy, its merits and demerits and the various ways of gene delivery.
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...
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...
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.
molecular tools that fix errors within genes rather than replacing or inserting a whole gene. Yet despite recent progress, gene therapy faces numerous hur - dles on the path to wider...
Changes to the genome might result in the gain or loss of a gene’s function, altered gene regulation, or changes in the amount of a given gene product. These changes can afect the survival of cells or afect organ function and, thus, the health of the individual.
