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26 Οκτ 2021 · Thanks to that new knowledge and steadfast investigations, gene therapy researchers can now point to a growing list of successful gene therapies. Here are four of the most promising.
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...
16 Νοε 2020 · Gene therapy is at an inflection point. Recent successes in genetic medicine have paved the path for a broader second wave of therapies and laid the foundation for next-generation technologies....
1 Νοε 2021 · Thanks to that new knowledge and steadfast investigations, gene therapy researchers can now point to a growing list of successful gene therapies. Here are four of the most promising.
23 Νοε 2023 · 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.
27 Ιαν 2019 · Gene therapies are gaining momentum as promising early successes in clinical studies accumulate and examples of regulatory approval for licensing increase.
8 Σεπ 2024 · The success of gene therapy in addressing RPE65-associated retinal dystrophy is exemplified by Luxturna® (Voretigene neparvovec). This groundbreaking gene therapy utilizes an AAV2 vector and is designed for individuals experiencing vision loss due to inherited retinal dystrophy stemming from confirmed biallelic RPE65 mutations.
