New Gene Therapy Reverses Three Diseases With Shots to the Bloodstream
1 min read
Summary
Researchers from the IRCCS San Raffaele Scientific Institute in Italy have developed a way to perform gene therapy on mice to treat three types of blood-related genetic disorders.
The mice were between one and five days old, as after birth there are large numbers of blood stem cells in the bloodstream which settle in the bone marrow a few days later, making them hard to reach.
The team used a single gene therapy injection to reprogram the blood stem cells, with the edits lasting for 20 weeks.
When these cells were then transplanted into mice who had not been given the therapy, they maintained their functionality and propagated.
This suggests the new approach could be applied to humans to treat severe immunodeficiencies, as well as Fanconi anaemia.
However, it remains a less efficient option than therapies which extract blood stem cells, modify them, and then reintroduce them into the body.
