Tissue Engineering and Transplantation in the Fetus

Fauza, Dario O.

doi:10.1016/b978-0-12-398358-9.00026-4

Cited by 1 publication

(1 citation statement)

References 92 publications

(70 reference statements)

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“…As such, IUERT has the potential to enable better penetration of affected tissues, such as the CNS 12 and other. 13 Additionally, fetal cells in developing organs have a higher capacity to proliferate than adult cells, 27,28 which may increase the uptake and utilization of the therapeutic enzyme, achieving higher enzyme activity levels in those tissues. Conditions marked with "Yes" are associated with a higher risk NIHF.…”

Section: Potential To Penetrate Affected Tissuesmentioning

confidence: 99%

Intrauterine enzyme replacement therapies for lysosomal storage disorders: Current developments and promising future prospects

Herzeg,

Borges,

Lianoglou

et al. 2023

Prenatal Diagnosis

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Lysosomal storage disorders (LSDs) are a group of monogenic condition, with many characterized by an enzyme deficiency leading to the accumulation of an undegraded substrate within the lysosomes. For those LSDs, postnatal enzyme replacement therapy (ERT) represents the standard of care, but this treatment has limitations when administered only postnatally because, at that point, prenatal disease sequelae may be irreversible. Furthermore, most forms of ERT, specifically those administered systemically, are currently unable to access certain tissues, such as the central nervous system (CNS), and furthermore, may initiate an immune response. In utero enzyme replacement therapy (IUERT) is a novel approach to address these challenges evaluated in a first‐in‐human clinical trial for IUERT in LSDs (NCT04532047). IUERT has numerous advantages: in‐utero intervention may prevent early pathology; the CNS can be accessed before the blood‐brain barrier forms; and the unique fetal immune system enables exposure to new proteins with the potential to prevent an immune response and may induce sustained tolerance. However, there are challenges and limitations for any fetal procedure that involves two patients. This article reviews the current state of IUERT for LSDs, including its advantages, limitations, and potential future directions for definitive therapies.

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Section: Potential To Penetrate Affected Tissuesmentioning

confidence: 99%