A new technique to reprogramme bone marrow cells has been developed, paving the way to simplify the donation process.
Transplants of bone marrow can be used to treat blood cell cancers, as well as cure other blood cell disorders, but experts say there are challenges to finding appropriate donors.
However, Laura Breda and her colleagues at the Children’s Hospital of Philadelphia in the US, has revealed a new strategy to deliver mRNA straight into stem cells, using a technique similar to that developed with the COVID-19 vaccines.
The mRNA then edits the genetic defects and helps regrow the bone marrow with healthy cells.
They demonstrated the technology in the bone marrow of living mice, as well as haematopoietic stem cells in humans taken from four patients with sickle cell disorder.
The findings have been published in the Science journal, which say in the human sample, the team corrected the genetic defect, suggesting there could be a route for gene-editing of bone marrow without the need for the usual transplantation process.
That includes hunting for a suitable bone marrow match and re-engineering the patient’s own cells outside the body.
The authors write: “These findings may potentially transform gene therapy in two ways.
“First, the cure of monogenic disorders, including non-malignant haematopoietic disorders (haemoglobinopathies, congenital anaemias or thrombocytopenias, and immunodeficiencies) and non-haematopoietic diseases (cystic fibrosis, metabolic disorders, and myopathies) with a simple intravenous infusion of targeted genetic medicines.
“Second, effecting cell type-specific state changes in vivo with minimal risk could allow previously impossible manipulations of physiology.
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“Such delivery systems may help translate the promise of decades of concerted genetic and biomedical research to treat a wide array of human diseases.”
However, in response, gene therapists Samuele Ferrari and Luigi Naldini warn it could be “a long, winding mountain road” to using the technique in humans, and that more studies will be needed to assess safety and effectiveness.
