Intra‑amniotic transplantation of brain‑derived neurotrophic factor‑modified mesenchymal stem cells treatment for rat fetuses with spina bifida aperta

Stem Cell Research & Therapy, 2022 · DOI: https://doi.org/10.1186/s13287-022-03105-6 · Published: August 3, 2022

Neurology Women's Health Regenerative Medicine & Stem Cells

Simple Explanation

Spina bifida aperta (SBA) is a birth defect where the spinal cord doesn't close completely. This study explores a new way to treat SBA before birth by transplanting modified stem cells into the amniotic fluid, the liquid surrounding the baby in the womb. The stem cells are modified with a special protein called brain-derived neurotrophic factor (BDNF), which helps nerve cells grow and survive. The goal is to improve nerve repair in babies with SBA. The study found that the modified stem cells were more effective than regular stem cells in reducing nerve cell death, promoting nerve cell growth, and improving the environment around the spinal cord.

Study Duration

Not specified

Participants

Rat fetuses with spina bifida aperta

Evidence Level

Not specified

Key Findings

1
BDNF-BMSCs sustained the characteristic of directional migration, engrafted at the SBA lesion area, increased the expression of BDNF in the defective spinal cords, alleviated the apoptosis of spinal cord cells, differentiated into neurons and skin-like cells, reduced the area of skin lesions, and improved the amniotic fluid microenvironment.
2
The BDNF-modified BMSCs showed a better effect than pure BMSCs on the inhibition of apoptosis and promotion of neural differentiation.
3
Intra-amniotic transplanted BDNF-BMSCs have an advantage of promoting the recovery of defective neural tissue of SBA fetuses.

Research Summary

This study investigates the therapeutic potential of intra-amniotic transplantation of brain-derived neurotrophic factor (BDNF)-modified bone marrow mesenchymal stem cells (BMSCs) for rat fetuses with spina bifida aperta (SBA). The results demonstrate that BDNF-BMSCs can migrate to the lesion area, increase BDNF expression, reduce apoptosis, differentiate into neurons and skin-like cells, decrease skin lesion area, and improve the amniotic fluid microenvironment. The study concludes that intra-amniotic transplantation of BDNF-modified BMSCs shows promise for promoting the recovery of injured neural tissue in SBA fetuses.

Practical Implications

Potential Prenatal Treatment

Intra-amniotic transplantation of BDNF-modified BMSCs could be a potential prenatal treatment for spina bifida aperta.

Improved Neurological Outcomes

The use of BDNF-modified BMSCs may lead to improved neurological outcomes in children with spina bifida aperta.

Novel Therapeutic Approach

This study introduces a novel therapeutic approach for congenital malformations by targeting lesion-specific engraftment and regeneration.

Study Limitations

1
The study is limited by the use of an animal model, making it difficult to assess long-term neural function recovery after birth.
2
Nerve repair of SBA takes a long time, and large animal models may be needed to identify the long-term effects of intra-amniotic stem cell transplantation.
3
Whether intra-amniotic genetically modified stem cell transplantation can be used as an independent minimally invasive method or as an adjunct to the existing prenatal surgical treatment in the clinic for SBA remains to be further studied and evaluated.

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