Prenatal and Postnatal Therapies for Down's Syndrome and Associated Developmental Anomalies and Degenerative Deficits: A Systematic Review of Guidelines and Trials

Autor: Zinnat Hasina, Chi Chiu Wang

Publikováno v: Frontiers in Medicine, Vol 9 (2022)

Down's syndrome (DS) is the most common genetic disorder at birth. Multiple developmental abnormalities before birth and early onset of degenerative deficits after birth are features of DS. Early treatment for the manifestations associated with DS in

Externí odkaz: https://doaj.org/article/77897161d71049749a19bc6409f12593

Intra-Amniotic Delivery of CRMP4 siRNA Improves Mesenchymal Stem Cell Therapy in a Rat Spina Bifida Model

Autor: Xiaowei Wei, Songying Cao, Wei Ma, Chaonan Zhang, Hui Gu, Dan Liu, Wenting Luo, Yuzuo Bai, Weilin Wang, Zhengwei Yuan

Publikováno v: Molecular Therapy: Nucleic Acids, Vol 20, Iss , Pp 502-517 (2020)

Neural tube defects (NTDs) result in prenatal mortality and lifelong morbidity, and available treatments have limited efficacy. We previously suggested that prenatal bone marrow-derived mesenchymal stem cell (BMSC) transplantation could treat neuron

Externí odkaz: https://doaj.org/article/160a0897feea43b4952029632dfa4422

In utero Therapy for the Treatment of Sickle Cell Disease: Taking Advantage of the Fetal Immune System

Autor: Alba Saenz de Villaverde Cortabarria, Laura Makhoul, John Strouboulis, Giovanna Lombardi, Eugene Oteng-Ntim, Panicos Shangaris

Publikováno v: Frontiers in Cell and Developmental Biology, Vol 8 (2021)

Sickle Cell Disease (SCD) is an autosomal recessive disorder resulting from a β-globin gene missense mutation and is among the most prevalent severe monogenic disorders worldwide. Haematopoietic stem cell transplantation remains the only curative op

Externí odkaz: https://doaj.org/article/656f2479f6474e3fae576a05a8e132d9

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A Causal Treatment for X-Linked Hypohidrotic Ectodermal Dysplasia: Long-Term Results of Short-Term Perinatal Ectodysplasin A1 Replacement

Autor: Holm Schneider, Christine Schweikl, Florian Faschingbauer, Smail Hadj-Rabia, Pascal Schneider

Publikováno v: International journal of molecular sciences, vol. 24, no. 8, pp. 7155
International Journal of Molecular Sciences; Volume 24; Issue 8; Pages: 7155

X-linked hypohidrotic ectodermal dysplasia (XLHED), caused by a genetic deficiency of ectodysplasin A1 (EDA1), is a rare developmental disorder of ectodermal derivatives such as hair, sweat glands, and teeth. The absence of sweat glands and perspirat

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a6935ca73489ed826b8ed8cf19bab4c7
https://opus4.kobv.de/opus4-fau/files/22840/ijms-24-07155-v2.pdf