Search results
4 dni temu · In gene therapy, a normal gene is inserted into the genome to replace an abnormal gene responsible for causing a certain disease. Of the various challenges involved in the process, one of the most significant is the difficulty in releasing the gene into the stem cell.
- Open in a separate window
Gene therapy protocols. Disease Objective Stem cells Release...
- Figure 1
Combination of stem cells and gene therapy. Source: Adapted...
- PubMed
Clustered regularly interspaced short palindromic repeats...
- Open in a separate window
8 lis 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 lis 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 ...
7 maj 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.
Tematy dnia
9 paź 2020 · Gene therapy techniques have been aimed at mitigating disease features of recessive and dominant disorders, as well as several cancers and other diseases.
2 paź 2019 · Although the first gene therapies have been approved and offer significant clinical benefit, they have run into challenges that require rethinking the drug development and delivery system across key stakeholders.
16 lis 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....
