Transamniotic Delivery of Hematopoietic Stem Cells Genetically Modified to Carry a Human Hemoglobin Subunit Beta Gene (HBB) in a Healthy Rodent Model

jpedsurg.org

We sought to determine whether transamniotic stem cell therapy (TRASCET) could be a viable alternative for the fetal administration of genetically modified hematopoietic stem cells (HSCs) carrying a human hemoglobin subunit beta gene (hHBB) in a healthy syngeneic rat model.

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Article · Dec 2024 · 1 min read

In brief

This preclinical study evaluates transamniotic delivery of genetically modified hematopoietic stem cells carrying the human hemoglobin beta gene in a rat model. The research explores a minimally invasive fetal intervention approach that could potentially treat hemoglobinopathies like sickle cell disease before birth.

Transamniotic stem cell therapy (TRASCET) offers a minimally invasive route for delivering genetically modified HSCs to the fetus.
Study tested delivery of hematopoietic stem cells carrying human hemoglobin subunit beta gene (hHBB) via amniotic fluid in rats.
TRASCET may provide an alternative to direct intravascular fetal injection for gene therapy in hemoglobinopathies like sickle cell disease.
Healthy syngeneic rat model used to establish proof-of-concept for transamniotic delivery of gene-modified stem cells.

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