Regenerative medicine: prenatal approaches

Coppi, Paolo De; Loukogeorgakis, Stavros; David, Anna L.; Almeida‐Porada, Graça; Chan, Jerry Kok Yen; Deprest, Jan; Wong, Kenneth Kak Yuen; Tam, Paul Kwong Hang

doi:10.1016/s2352-4642(22)00192-4

Cited by 13 publications

(4 citation statements)

References 118 publications

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“…Increasingly, fetal blood transfusion is used to treat α‐thalassemia major 134 and other rare anemias although transfusion of diagnosed metabolic disease (e.g., pyruvate Kinase deficiency 135 ) is still uncommon. Fetal stem cell transplantation of fetal stem cells has been applied to a range of genetic diseases, including metabolic diseases such as acute neuronopathic (type II) Gaucher disease, Hurler syndrome, and Niemann Pick type A (reviewed extensively in reference 136).…”

Section: Why Perform Fetal Gene Therapy: In Which Circumstances Might...mentioning

confidence: 99%

Fetal gene therapy

Waddington

Peranteau

Rahim

et al. 2023

J of Inher Metab Disea

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Fetal gene therapy was first proposed toward the end of the 1990s when the field of gene therapy was, to quote the Gartner hype cycle, at its “peak of inflated expectations.” Gene therapy was still an immature field but over the ensuing decade, it matured and is now a clinical and market reality. The trajectory of treatment for several genetic diseases is toward earlier intervention. The ability, capacity, and the will to diagnose genetic disease early—in utero—improves day by day. A confluence of clinical trials now signposts a trajectory toward fetal gene therapy. In this review, we recount the history of fetal gene therapy in the context of the broader field, discuss advances in fetal surgery and diagnosis, and explore the full ambit of preclinical gene therapy for inherited metabolic disease.

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Section: Why Perform Fetal Gene Therapy: In Which Circumstances Might...mentioning

confidence: 99%

Fetal gene therapy

Waddington

Peranteau

Rahim

et al. 2023

J of Inher Metab Disea

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“…This review will first briefly overview technological platforms available for regenerative medicine both after birth and prenatally [ 2 ]. Tissue stem and progenitor cells of each organ will be discussed in the later section on clinical application.…”

Section: Introductionmentioning

confidence: 99%

Regenerative medicine: current research and perspective in pediatric surgery

2023

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The field of regenerative medicine, encompassing several disciplines including stem cell biology and tissue engineering, continues to advance with the accumulating research on cell manipulation technologies, gene therapy and new materials. Recent progress in preclinical and clinical studies may transcend the boundaries of regenerative medicine from laboratory research towards clinical reality. However, for the ultimate goal to construct bioengineered transplantable organs, a number of issues still need to be addressed. In particular, engineering of elaborate tissues and organs requires a fine combination of different relevant aspects; not only the repopulation of multiple cell phenotypes in an appropriate distribution but also the adjustment of the host environmental factors such as vascularisation, innervation and immunomodulation. The aim of this review article is to provide an overview of the recent discoveries and development in stem cells and tissue engineering, which are inseparably interconnected. The current status of research on tissue stem cells and bioengineering, and the possibilities for application in specific organs relevant to paediatric surgery have been specifically focused and outlined.

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“…Access to fetal tissues, however, has ethical and legal restrictions in many parts of the world that prohibit their use in research 27 . Until now, primary fetal organoids have only been derived with destructive methods, limiting their use for autologous disease modelling, prenatal functional diagnostics and personalised therapeutics 28 . In this manuscript, we present the derivation of primary human fetal epithelial organoids of multiple tissue identities (intestinal, renal and pulmonary) from fetal fluids collected during the second and third trimester of gestation.…”

Section: Introductionmentioning

confidence: 99%

Single cell-guided prenatal derivation of primary epithelial organoids from the human amniotic and tracheal fluids

Gerli

Calà

Beesley

et al. 2023

Preprint

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Despite advances in prenatal diagnosis, it is still difficult to predict severity and outcomes of many congenital malformations. New patient-specific prenatal disease modelling may optimise personalised prediction. We and others have described the presence of mesenchymal stem cells in amniotic fluid (AFSC) that can generate induced pluripotent stem cells (iPSCs). The lengthy reprogramming processes, however, limits the ability to define individual phenotypes or plan prenatal treatment. Therefore, it would be advantageous if fetal stem cells could be obtained during pregnancy and expanded without reprogramming. Using single cell analysis, we characterised the cellular identities in amniotic fluid (AF) and identified viable epithelial stem/progenitor cells of fetal intestinal, renal and pulmonary origin. With relevance for prenatal disease modelling, these cells could be cultured to form clonal epithelial organoids manifesting small intestine, kidney and lung identity. To confirm this, we derived lung organoids from AF and tracheal fluid (TF) cells of Congenital Diaphragmatic Hernia (CDH) fetuses and found that they show differences to non-CDH controls and can recapitulate some pathological features of the disease. Amniotic Fluid Organoids (AFO) allow investigation of fetal epithelial tissues at clinically relevant developmental stages and may enable the development of therapeutic tools tailored to the fetus, as well as to predicting the effects of such therapies.

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Regenerative medicine: prenatal approaches

Cited by 13 publications

References 118 publications

Fetal gene therapy

Fetal gene therapy

Regenerative medicine: current research and perspective in pediatric surgery

Single cell-guided prenatal derivation of primary epithelial organoids from the human amniotic and tracheal fluids

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