Innovative Gene Therapies Target Blood Cells for Treating Diseases in Newborns
TL;DR Summary
Researchers have discovered a critical early postnatal period when circulating blood stem cells can be effectively targeted with gene therapy directly in the body, opening new possibilities for treating genetic blood disorders without stem cell transplantation or chemotherapy, with promising results in mouse models and potential applicability to humans.
Topics:health#blood-stem-cells#gene-therapy#genetic-disorders#in-vivo-treatment#postnatal-window#science
- No More Transplants? New Gene Therapy Targets Blood Stem Cells Directly SciTechDaily
- New Gene Therapy Reverses Three Diseases With Shots to the Bloodstream SingularityHub
- Gene Therapies for Newborns themedicinemaker.com
- Newborn Mice May Hold the Key to Simpler Gene Therapy the-scientist.com
- Newborns May Have Window for In Vivo Gene Therapy Inside Precision Medicine
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