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Akademický článek

Seamless Gene Correction in the Human Cystic Fibrosis Transmembrane Conductance Regulator Locus by Vector Replacement and Vector Insertion Events

Autor: Shingo Suzuki, Keisuke Chosa, Cristina Barillà, Michael Yao, Orsetta Zuffardi, Hirofumi Kai, Tsuyoshi Shuto, Mary Ann Suico, Yuet W. Kan, R. Geoffrey Sargent, Dieter C. Gruenert

Publikováno v: Frontiers in Genome Editing, Vol 4 (2022)

Background: Gene correction via homology directed repair (HDR) in patient-derived induced pluripotent stem (iPS) cells for regenerative medicine are becoming a more realistic approach to develop personalized and mutation-specific therapeutic strategi

Externí odkaz: https://doaj.org/article/98771079d2134f55b64cdc7d8d0cd6f4

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Akademický článek

TALENs Facilitate Single-step Seamless SDF Correction of F508del CFTR in Airway Epithelial Submucosal Gland Cell-derived CF-iPSCs

Publikováno v: Molecular Therapy: Nucleic Acids, Vol 5, Iss C (2016)

Cystic fibrosis (CF) is a recessive inherited disease associated with multiorgan damage that compromises epithelial and inflammatory cell function. Induced pluripotent stem cells (iPSCs) have significantly advanced the potential of developing a perso

Externí odkaz: https://doaj.org/article/aa7f6c58c90142c2a88dd3a6fd6f2d5f

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Induction of therapeutic levels of HbF in genome-edited primary β

Autor: Maura, Mingoia, Cristian A, Caria, Lin, Ye, Isadora, Asunis, M Franca, Marongiu, Laura, Manunza, M Carla, Sollaino, Jiaming, Wang, Annalisa, Cabriolu, Ryo, Kurita, Yukio, Nakamura, Francesco, Cucca, Yuet W, Kan, M Giuseppina, Marini, Paolo, Moi

Publikováno v: British journal of haematologyReferences. 192(2)

Hereditary persistence of fetal haemoglobin (HPFH) is the major modifier of the clinical severity of β-thalassaemia. The homozygous mutation c.-196 CT in the Aγ-globin (HBG1) promoter, which causes Sardinian δβ

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid________::a8e4b16955645de8560b0a91220a72f2
https://pubmed.ncbi.nlm.nih.gov/33216968

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Akademický článek

Genetically edited CD34⁺ cells derived from human iPS cells in vivo but not in vitro engraft and differentiate into HIV-resistant cells.

Autor: Morvan, Maelig G., Teque, Fernando, Lin Ye, Moreno, Mary E., Jiaming Wang, VandenBerg, Scott, Stoddart, Cheryl A., Yuet Wai Kan, Levy, Jay A.

Publikováno v: Proceedings of the National Academy of Sciences of the United States of America; 5/18/2021, Vol. 118 Issue 20, p1-7, 7p

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Akademický článek

A New Role for the Human Placenta as a Hematopoietic Site Throughout Gestation.

Autor: Bárcena, Alicia, Muench, Marcus O., Kapidzic, Mirhan, Fisher, Susan J.

Publikováno v: Reproductive Sciences; Feb2009, Vol. 16 Issue 2, p178-187, 10p, 5 Graphs

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Akademický článek

Sharpening the Tools: A summary of a National Institutes of Health workshop on new technologies for detection of fetal cells in maternal blood for early prenatal diagnosis.

Autor: Bianchi, Diana W., Hanson, James

Publikováno v: Journal of Maternal-Fetal & Neonatal Medicine; Apr2006, Vol. 19 Issue 4, p199-207, 9p

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