# Biomimetic intervention strategies for endogenous in utero repair of Spina Bifida

> **NIH NIH R01** · BAYLOR COLLEGE OF MEDICINE · 2024 · $562,740

## Abstract

ABSTRACT
Neural tube defects (NTDs), including spina bifida (SB), have a devastating impact on the health and
development of infants and children, with economic, social, and physical demands or hardships placed on
caregivers. SB affects approximately 2,500 live births per year in the United States, with environmental and
genetic factors playing a role in its etiology. By combining bioengineering and placental-derived stem cell
approaches, the CuRe (Cellular Therapy for In Utero Repair of Myelomeningocele) trial has recently advanced
surgical effectiveness and improved clinical outcomes. However, in utero repair requiring fetal exposure at mid-
pregnancy to reduce the ongoing damage of the exposed spinal cord represents an invasive procedure. By
leveraging our expertise in the synthesis of biomimetic therapeutic strategies able to induce tissue repair by
inducing a regenerative cascade at the site of lesion and in the generation of genetically induced model systems,
this project aims to devise less invasive, novel intervention strategies for endogenous in utero repair of
SB. The overall hypothesis is that by creating a pro-regenerative environment within the amniotic cavity, it is
possible to activate the cellular and molecular cascades required to reduce the severity of SB lesions. To test
these hypotheses, we will i) determine the therapeutic efficacy of amniotic fluid-based cell free strategies to
modulate the in utero environment and reduce the severity of SB in a clinically relevant in-house mouse model
(Fbpk8 knockout mice) (Aim 1) and ii) evaluate the protective and regenerative potential of a biomimetic
thermogel for in utero repair of SB lesions in mouse and rabbit models (Aim 2). The proposed research is
expected to enhance our understanding with respect to the impact of biomimetic strategies inducive of
regeneration on the development of neural tube tissues following genetic and mechanical disruption. The
implications of these therapeutic intervention strategies are timely and critical, as stem cell-based, and
bioengineering approaches are already being deployed clinically in other areas, but a minimally invasive, early
in utero intervention is still needed for families and infants affected by SB.

## Key facts

- **NIH application ID:** 10979551
- **Project number:** 1R01HD113702-01A1
- **Recipient organization:** BAYLOR COLLEGE OF MEDICINE
- **Principal Investigator:** Bruna Corradetti
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $562,740
- **Award type:** 1
- **Project period:** 2024-09-01 → 2029-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10979551

## Citation

> US National Institutes of Health, RePORTER application 10979551, Biomimetic intervention strategies for endogenous in utero repair of Spina Bifida (1R01HD113702-01A1). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10979551. Licensed CC0.

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