New-born infants may have a “unique window” during which they could receive gene therapy targeted at blood stem cells, Italian researchers have reported.
This would enable gene therapy without the need for stem cell transplantation or chemotherapy, according to researchers at the San Raffaele-Telethon Institute for Gene Therapy, Milan.
So far, the proposals have been tested in laboratory mice. The researchers say that, in the first two weeks of the animals’ life, they have found that levels of haematopoietic stem and progenitor cells (HSPCs) in circulating blood are significantly higher than at older ages. They have also studied new-born human infants and found high levels of circulating HSPCs.
Researcher Dr Michela Milani explained: “After birth, blood stem cells need to move from the liver, where they have resided throughout the last months of pregnancy to their definitive home in the bone marrow. We found that as they so travel in the circulation they can be more easily accessed by intravenous delivered vectors and thus be genetically modified without the need to harvest and process them outside of the body.”
The researchers tested gene lentiviral-based gene therapies in mouse models of Fanconi anaemia, adenosine deaminase-deficient severe combined immunodeficiency and autosomal recessive osteopetrosis. Reporting in Nature, the researchers say that gene transfer led to prolongation of life and significant therapeutic benefits in all three cases.
The researchers say that in the case of Fanconi anaemia, corrected stem cells repopulated the blood system and prevented bone marrow failure.
Fellow researcher Dr Alessio Cantore said: “This study provides proof of concept that in vivo lentiviral gene delivery to blood stem cells is feasible during a short but accessible period early in life as a gene therapy strategy for blood disorders. While the efficiency currently remains limited as compared to established ex vivo treatments, it may suffice, if replicated in human babies, to benefit some genetic diseases such as severe immunodeficiencies or Fanconi anaemia.”
Source:
Milani M, Fabiano A, Perez-Rodriguez M, Hernandez RJ, Zecchillo A, Zonari E, Ottonello S, Basso-Ricci L, Canepari C, Volpin M, Iannello V, Capo V, Quaranta P, Seffin L, Russo F, Biffi M, Ormoli L, Brombin C, Carlucci F, Forlino A, Filibian M, Montini E, Scala S, Villa A, Bueren JA, Rio P, Aiuti A, Cantore A, Naldini L. (2025) “In vivo haemopoietic stem cell gene therapy enabled by postnatal trafficking.” Nature, 28 May 2025, doi: 10.1038/s41586-025-09070-3. Link: https://www.nature.com/articles/s41586-025-09070-3
Disclaimer: The news stories shared on this site are used as a way to inform our members and followers of updates and relevant information happening in Haematology. The BSH does not endorse the content of news items from external sources, and is not in a position to verify the findings, accuracy or the source of any studies mentioned. Any medical or drugs information is provided as an information resource only, and is not to be relied on for any diagnostic or treatment purposes.
News service provided by Englemed News.