Animal studies suggest that Gaucher disease could be treated pre-natally using foetal gene therapy, British researchers reported last night.
Researchers reported that the treatment was successful in a mouse model.
Writing in Nature Medicine, they report on further trials involving macaques, suggesting the treatment is feasible in primates and can lead to distribution of a transgene to a developing brain.
The study involved University College London, UK, and the KK Women’s and Children’s Hospital and National University Health System in Singapore.
The researchers injected an adeno-associated virus vector into foetal mice carrying neuropathic Gaucher disease.
They found that these animals lived for at least 18 weeks after birth – while untreated animals lived on average for just 15 days.
Dr Simon Waddington, from UCL, said: “We found that the mice who received an injection of adeno-associated virus vector were more able to break down fatty chemicals and re-express the gene encoding an enzyme that is deficient in Gaucher disease.”
Associate Professor Jerry Chan, from Singapore, said: “This new approach will bring hope, not only for Gaucher disease, but also for other inborn errors of metabolism that can potentially be treated using foetal gene therapy.”
Fetal gene therapy for neurodegenerative disease of infants. Nature Medicine 16 July 2018; doi: 10.1038/s41591-018-0106-7
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