Gene Therapy in Blood Diseases: Milestones and Challenges

Have you ever wondered how gene therapy is changing the way scientists treat blood disorders? Well, the journey started with the discovery of DNA's structure and gene regulation mechanisms in the early 20th century. This foundation laid the groundwork for gene therapy (GT), which uses platforms like viruses and non-viral methods to introduce healthy genes into target cells. ClinicalTrial. gov hosts many clinical trials for GT, including those focusing on blood diseases. A recent review found 41 such trials, mostly in the USA (56. 1%). Interestingly, 41. 46% were privately funded, highlighting the financial aspects involved. Only half of these trials targeted specific blood diseases with genetic origins, like hemophilia A, hemophilia B, and Fanconi anemia. The number of registered trials in a country correlated positively with its economic development, scientific advancements, health investment per capita, and quality of life. However, despite significant progress, numerous ethical, social, political, and economic issues remain unresolved.