What are lentiviral vectors and how are they used in gene therapy?

Lentiviral vectors are repurposed from HIV, stripped of destructive genes, and used to deliver therapeutic genes directly into a patient's DNA, enabling treatment for complex diseases.

How does in vivo gene therapy differ from ex vivo gene therapy like Kymriah?

Ex vivo therapy involves treating cells outside the body and reintroducing them, while in vivo therapy delivers genetic instructions directly inside the patient's body.

What is the future outlook for gene therapy with companies like Umoja Biopharma?

Gene therapy is expected to become more accessible and routine, potentially treating a wide range of diseases and transforming medicine from boutique care to mainstream treatment.

Home / Science / Gene Therapy Revolution: From Rare Diseases to Mainstream Care

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.

Gene Therapy Revolution: From Rare Diseases to Mainstream Care

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.

This progression signals a future where gene therapy could become a routine medical intervention, akin to receiving a vaccine, with potential applications for autoimmune diseases and solid tumors. As the market for cell and gene therapies is projected to exceed $100 billion by 2034, overcoming manufacturing bottlenecks and ensuring ethical oversight will be crucial for realizing this transformative potential.

Disclaimer: This story has been auto-aggregated and auto-summarised by a computer program. This story has not been edited or created by the Feedzop team.

Home / Science / Gene Therapy Revolution: From Rare Diseases to Mainstream Care

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.

Disclaimer: This story has been auto-aggregated and auto-summarised by a computer program. This story has not been edited or created by the Feedzop team.