Almeida-Porada Lab

Research focus

NIH R01 · 2025

Targeted conditioning to maximize prenatal HSC engraftment for SCD

PROJECT ABSTRACT: Treatment of genetic disorders by in utero transplantation (IUTx) has safely been performed for decades in humans. The first IUTx cure, in the US, used hematopoietic stem cells (HSC) and corrected a child with X-SCID. Since this groundbreaking moment >25 yrs. ago, >50 patients have now been treated with this procedure for 14 different genetic disorders. However, for reasons that are still not well understood, full therapeutic success has only been achieved in X-SCID patients. Thus, a better understanding of the mechanisms by which HSC engraftment is hindered after IUTx is required, so that strategies can be developed to achieve therapeutic levels of HSC engraftment in…

From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.