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Gene Therapy Revolution: From Rare Diseases to Mainstream Care
2 Jan
Summary
- Lentiviral vectors are expanding gene therapy beyond rare disorders.
- In vivo gene therapy promises lower costs and wider accessibility.
- Gene therapy is poised to become as routine as vaccines.
A new generation of gene therapies is emerging, leveraging lentiviral vectors to treat complex diseases far beyond rare inherited disorders. Initially confined to conditions affecting non-dividing cells, gene therapy's reach has dramatically expanded. The development of lentiviral vectors, repurposed from HIV, allows for gene integration into rapidly dividing cells, proving revolutionary in oncology and other complex illnesses.
These advancements are moving from complex "ex vivo" treatments, which require months of specialized cell manufacturing and high costs, to more accessible "in vivo" approaches. Companies are now developing therapies that deliver genetic instructions directly into the body, eliminating lengthy procedures and reducing production expenses. This innovation aims to make treatments like those for leukemia and lymphoma available in more hospitals.
