New breakthrough: Italian scientists pioneer a new paradigm for in - vivo gene therapy of hematopoietic stem cells.
Release time:
2025-07-21
In recent years, significant progress has been made in gene therapy for genetic diseases. In particular, the ex - vivo gene therapy strategy based on lentiviral vectors has been successfully applied to diseases such as severe combined immunodeficiency caused by adenosine deaminase deficiency (ADA - SCID). However, existing therapies still face challenges such as high toxic side effects of chemotherapy pre - treatment, complex operations, and high costs, and have limited efficacy for diseases involving multiple tissues.
Recently, the teams led by Luigi Naldini and Michela Milani from the San Raffaele Scientific Institute in Italy published a groundbreaking study in Nature. They proposed an innovative in - vivo gene therapy strategy. By leveraging the biological characteristic of neonatal hematopoietic stem cells migrating from the liver to the bone marrow and combining it with an anti - phagocytic lentiviral vector (CD47high - LV), they successfully achieved efficient and low - toxicity gene modification, opening up a new path for the treatment of genetic blood diseases.
The research team first revealed the dynamic distribution pattern of neonatal hematopoietic stem cells: the number of hematopoietic stem cells in the peripheral blood of newborn mice is more than 10 times that of adult mice, and this numerical advantage persists within 2 weeks after birth. In addition, they precisely mapped the spatio - temporal dynamics of the migration of these cells from fetal hematopoietic organs to the bone marrow. Based on this, the team developed the CD47high lentiviral vector system. The high - expression CD47 molecule (a "don't eat me" signal) on its surface enables it to effectively evade the clearance of the immune system, and they designed multiple tissue - specific promoters to meet the needs of different diseases.
In three genetic disease models, this strategy showed significant efficacy:
In the ADA - SCID model: The survival rate of the treatment group reached 71%, significantly better than that of the control group, and the liver detoxification function synergistically enhanced the treatment effect.
In the Fanconi anemia model: Gene - corrected cells showed a selective advantage under the pressure of bone marrow failure, and the vector copy number increased from 0.1 to 1.0, almost completely reconstructing the hematopoietic system.
In the osteopetrosis model: Although the efficacy was limited, it confirmed the feasibility of the technology itself, and the rapid progression of the disease was the main limiting factor.
Notably, the number of hematopoietic stem cells in the peripheral blood of human neonates is also significantly higher than that of adults, providing direct evidence for clinical translation.
This study not only breaks through the bottleneck of ex - vivo gene therapy but also avoids the risks of chemotherapy pre - treatment and significantly reduces the treatment cost. This innovative paradigm provides a new solution for early - onset genetic diseases and marks a leap from "ex - vivo manipulation" to "in - vivo precise repair" in gene therapy. In the future, with the advancement of clinical translation, it is expected to bring hope of cure to more patients with genetic diseases.
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