In utero stem cell transplantation and gene therapy: Recent progress and the potential for clinical application

McClain, Lauren E.; Flake, Alan W.

doi:10.1016/j.bpobgyn.2015.08.006

Cited by 40 publications

(38 citation statements)

References 80 publications

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“…1,2 It has the potential to treat a number of congenital immune, metabolic, and hematologic disorders, including sickle cell disease and thalassemia. [3][4][5][6] IUHCT has been successful in preclinical studies in the murine, canine, ovine, and porcine models. 1,2,7,8 The clinical translation of IUHCT, however, has been heretofore disappointing.…”

Section: Introductionmentioning

confidence: 99%

Regulatory T cells promote alloengraftment in a model of late-gestation in utero hematopoietic cell transplantation

et al. 2020

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In utero hematopoietic cell transplantation (IUHCT) has the potential to cure congenital hematologic disorders including sickle cell disease. However, the window of opportunity for IUHCT closes with the acquisition of T-cell immunity, beginning at approximately 14 weeks gestation, posing significant technical challenges and excluding from treatment fetuses evaluated after the first trimester. Here we report that regulatory T cells can promote alloengraftment and preserve allograft tolerance after the acquisition of T-cell immunity in a mouse model of late-gestation IUHCT. We show that allografts enriched with regulatory T cells harvested from either IUHCT-tolerant or naive mice engraft at 20 days post coitum (DPC) with equal frequency to unenriched allografts transplanted at 14 DPC. Long-term, multilineage donor cell chimerism was achieved in the absence of graft-versus-host disease or mortality. Decreased alloreactivity among recipient T cells was observed consistent with donor-specific tolerance. These findings suggest that donor graft enrichment with regulatory T cells could be used to successfully perform IUHCT later in gestation.

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Section: Introductionmentioning

confidence: 99%

Regulatory T cells promote alloengraftment in a model of late-gestation in utero hematopoietic cell transplantation

et al. 2020

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“…In the largest review from 12 fetal centers from the North American Fetal Therapy Network registry data, identified 98 patients who underwent percutaneous radio frequency ablation of a cardiac twin. In this series, the overall survival of the normal (pump) twin to 30 days was 80% [46][47][48][49][50].…”

Section: Twin Reversed Arterial Perfusionmentioning

confidence: 66%

Principles of Fetal Surgery

Helal¹

2019

Pediatric Surgery, Flowcharts and Clinical Algorithms

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Fetal therapy (in utero therapy) is a type of special therapy which aims to prevent or correct congenital anomalies in fetus, and prevents their severe consequences on later fetal development. It includes the use of in utero human fetal stem cell transplantation, fetal gene therapy and gene-editing technology as a new treatment for fetal genetic disorders. It started with open fetal surgery and then significantly advancing with innovations, toward minimally invasive fetal procedures, which are undoubtedly the future of fetal surgery, with the goal of providing the best possible fetal outcome, while minimizing the morbidity and mortality to the mother. The goal of fetal treatments is to decrease both fetal and maternal risks and prevent premature rupture of membranes. Fetal ultrasound and MRI are crucial for successful fetal interventions. Moreover, multidisciplinary fetal teams, including fetal surgeon, ultrasonographer, perinatologist, and anesthesiologist, are essential for optimum care to both mother and fetus. Finally, any new modality of fetal therapy must be thoroughly evaluated in animal models before clinical practice. In this chapter, we discuss the basic principles of fetal surgery, milestones of fetal surgery, specific fetal anomalies that are amenable for fetal surgery, successful fetal surgery criteria and future of fetal surgery.

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“…One of the challenges of any new technology is the "the pacing problem". Marchant (2011 [68] ) describes this issue as the "inability of legal and regulatory frameworks to keep pace with technologies" due to their rapid emergence into the marketplace (Charo, 2015 [69] ; Wallach, 2015 [70] ). The US National Academies has recently recommended that "existing regulatory infrastructure and processes for reviewing and evaluating somatic gene therapy to treat or prevent disease and disability should be used to evaluate somatic gene therapy that uses gene editing" (National Academies of Sciences, 2017 [9] ).…”

Section: Legal Lag or The Pacing Problemmentioning

confidence: 99%

Gene editing for advanced therapies

Garden¹,

Winickoff²

2018

OECD Science, Technology and Industry Working Papers

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OECD Working Papers should not be reported as representing the official views of the OECD or of its member countries. The opinions expressed and arguments employed are those of the authors. Working Papers describe preliminary results or research in progress by the author(s) and are published to stimulate discussion on a broad range of issues on which the OECD works. Comments on Working Papers are welcomed, and may be sent

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In utero stem cell transplantation and gene therapy: Recent progress and the potential for clinical application

Cited by 40 publications

References 80 publications

Regulatory T cells promote alloengraftment in a model of late-gestation in utero hematopoietic cell transplantation

Regulatory T cells promote alloengraftment in a model of late-gestation in utero hematopoietic cell transplantation

Principles of Fetal Surgery

Gene editing for advanced therapies

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