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Groundbreaking Nature Study Unveils Innovative Approach to In Vivo Gene Therapy for Blood Stem Cells
- Researchers at SR-Tiget in Milan have discovered a critical post-birth window for gene therapy targeting blood stem cells, presenting an alternative to conventional treatments.
- Their in vivo approach involves directly administering lentiviral vectors into the bloodstream of newborn mice, leveraging the abundance of hematopoietic cells for gene transfer.
- Dr. Michela Milani highlighted the significance of this method, utilizing the natural migration of stem cells to enable genetic alterations without extensive processing.
- Mouse models with genetic disorders like ADA-SCID and Fanconi anemia showed therapeutic benefits from in vivo gene transfer, preventing bone marrow failure.
- By employing mobilizing agents and optimizing vectors, the team enhanced gene transfer efficiency and extended the treatment window to older mice.
- The discovery of circulating HSPCs in newborn humans reinforces the potential for in vivo gene therapy to be applicable in human populations.
- The distinct responsiveness of young stem cells to gene transfer highlights the need for further exploration into mechanisms governing this process for potential translation to older individuals.
- SR-Tiget's research offers a groundbreaking approach to genetic blood disorders, emphasizing timing and stem cell properties for improved therapeutic outcomes.
- This study signifies a significant advancement in gene therapy and may revolutionize treatment standards for patients with genetic abnormalities.
- The findings hold promise for enhancing gene therapy accessibility and efficacy in addressing a range of genetic blood disorders in both animal models and potentially in human neonates.
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