The Theoretical Basis of In Utero Hematopoietic Stem Cell Transplantation and Its Use in the Treatment of Blood Disorders

Tai-MacArthur, Sarah; Lombardi, Giovanna; Shangaris, Panicos

doi:10.1089/scd.2020.0181

Cited by 6 publications

(2 citation statements)

References 60 publications

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“…Despite the obvious therapeutic improvement, this approach was risky and likely to increase the rate of fetal loss. Intrauterine hematopoietic stem cell (HSC) transplantation was deliberated as another option for the treatment; however, in contrast to postnatal HSC transplantation, many trials failed to succeed (Songdej et al., 2017; Tai‐MacArthur et al., 2021). This suggests that there is no current effective treatment for Hb Bart's hydrops fetalis.…”

Section: Introductionmentioning

confidence: 99%

Development of molecular diagnostic platform for α⁰‐thalassemia 44.6 kb (Chiang Rai, ‐‐^CR) deletion in individuals with microcytic red blood cells across Thailand

Khamphikham

Hanmanoviriya

Wongpalee

et al. 2023

Annals of Human Genetics

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Introduction:The α 0 -thalassemia 44.6 kb or Chiang Rai (--CR ) deletion has been reported in northern Thailand and is capable of causing hemoglobin (Hb) H disease and a lethal α-thalassemia genotype, Hb Bart's hydrops fetalis, in this region.However, there are no current data regarding the frequency of --CR nationwide due to a lack of effective diagnostic assay. Therefore, this study aimed to develop a reliable platform for simultaneous genotyping of --CR and two common α 0thalassemias in Thailand (--SEA and --THAI ) and investigate the frequency of --CR across Thailand.Methods: Multiplex gap-PCR assay and five renewable plasmid DNA controls for --CR , --SEA , --THAI , α2-globin (HBA2), and β-actin (ACTB) were newly developed and validated with reference methods. The developed assay was further tested on 1046 unrelated individuals with a reduced mean corpuscular volume (MCV) of less than 75 fl for investigating genotypic and allelic spectrum of --CR . Results: Our developed assay showed 100% concordance with reference methods. The results were valid and reproducible throughout hundreds of reactions.Comparison of the genotypic and allelic spectra revealed that heterozygous --SEA (--SEA /αα) and --SEA alleles were dominant with the frequency of 22.85%

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Section: Introductionmentioning

confidence: 99%

Development of molecular diagnostic platform for α⁰‐thalassemia 44.6 kb (Chiang Rai, ‐‐^CR) deletion in individuals with microcytic red blood cells across Thailand

Khamphikham

Hanmanoviriya

Wongpalee

et al. 2023

Annals of Human Genetics

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“…However, the thymic deletion is incomplete, self-reactive T cells that escape the deletion are suppressed by regulatory T cells (Tregs) mediated peripheral tolerance (Vrecenak and Flake, 2013). Central and peripheral tolerance leaves the fetus to recognize self-antigens and eliminate foreign antigens (Tai-MacArthur et al, 2021). Theoretically, introduction of allogenic cells by IUHCT ahead of the formation of functional fetal adaptive immune system could result in deletion of alloreactive T cells and induction of Treg cells, resulting in complete donor-specific immune tolerance (Vrecenak and Flake, 2013).…”

Section: Rationale For In Utero Hematopoietic Cell Transplantationmentioning

confidence: 99%

Experimental and clinical progress of in utero hematopoietic cell transplantation therapy for congenital disorders

Shi

Pan

2022

Front. Pharmacol.

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In utero hematopoietic cell transplantation (IUHCT) is considered a potentially efficient therapeutic approach with relatively few side effects, compared to adult hematopoietic cell transplantation, for various hematological genetic disorders. The principle of IUHCT has been extensively studied in rodent models and in some large animals with close evolutionary similarities to human beings. However, IUHCT has only been used to rebuild human T cell immunity in certain patients with inherent immunodeficiencies. This review will first summarize the animal models utilized for IUHCT investigations and describe the associated outcomes. Recent advances and potential barriers for successful IUHCT are discussed, followed by possible strategies to overcome these barriers experimentally. Lastly, we will outline the progress made towards utilizing IUHCT to treat inherent disorders for patients, list out associated limitations and propose feasible means to promote the efficacy of IUHCT clinically.

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Transamniotic Delivery of Hematopoietic Stem Cells Genetically Modified to Carry a Human Hemoglobin Subunit Beta Gene (HBB) in a Healthy Rodent Model

Moskowitzova,

Scire,

Kycia

et al. 2024

Journal of Pediatric Surgery

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The Theoretical Basis of In Utero Hematopoietic Stem Cell Transplantation and Its Use in the Treatment of Blood Disorders

Cited by 6 publications

References 60 publications

Development of molecular diagnostic platform for α⁰‐thalassemia 44.6 kb (Chiang Rai, ‐‐^CR) deletion in individuals with microcytic red blood cells across Thailand

Development of molecular diagnostic platform for α⁰‐thalassemia 44.6 kb (Chiang Rai, ‐‐^CR) deletion in individuals with microcytic red blood cells across Thailand

Experimental and clinical progress of in utero hematopoietic cell transplantation therapy for congenital disorders

Transamniotic Delivery of Hematopoietic Stem Cells Genetically Modified to Carry a Human Hemoglobin Subunit Beta Gene (HBB) in a Healthy Rodent Model

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The Theoretical Basis of In Utero Hematopoietic Stem Cell Transplantation and Its Use in the Treatment of Blood Disorders

Cited by 6 publications

References 60 publications

Development of molecular diagnostic platform for α0‐thalassemia 44.6 kb (Chiang Rai, ‐‐CR) deletion in individuals with microcytic red blood cells across Thailand

Development of molecular diagnostic platform for α0‐thalassemia 44.6 kb (Chiang Rai, ‐‐CR) deletion in individuals with microcytic red blood cells across Thailand

Experimental and clinical progress of in utero hematopoietic cell transplantation therapy for congenital disorders

Transamniotic Delivery of Hematopoietic Stem Cells Genetically Modified to Carry a Human Hemoglobin Subunit Beta Gene (HBB) in a Healthy Rodent Model

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Product

Resources

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Development of molecular diagnostic platform for α⁰‐thalassemia 44.6 kb (Chiang Rai, ‐‐^CR) deletion in individuals with microcytic red blood cells across Thailand

Development of molecular diagnostic platform for α⁰‐thalassemia 44.6 kb (Chiang Rai, ‐‐^CR) deletion in individuals with microcytic red blood cells across Thailand