Generation of CRISPR/Cas9-edited human iPSC lines carrying homozygous and heterozygous SAMD9 p.I983S mutations

Generation of CRISPR/Cas9-edited human iPSC lines carrying homozygous and heterozygous SAMD9 p.I983S mutations

Induced pluripotent stem cells (iPSCs) harboring patient derived SAMD9 mutation offer a unique platform to study the multi-organ involvement observed in this rare disease, referred to as myelodysplasia, infections, restriction of growth, adrenal hypoplasia, genital phenotypes, and enteropathy (MIRAG...

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Bibliographic Details
Main Authors: Majd Khiami, Yan Ju, Lei Han, Jonathan Klein, Min-Joon Han, Shondra M. Pruett-Miller, Marcin W. Wlodarski
Format: Article
Language:English
Published: Elsevier 2024-12-01
Series:Stem Cell Research
Subjects:
SAMD9
Bone marrow failure
Hematology
iPSC
CRISPR/Cas9
Online Access:http://www.sciencedirect.com/science/article/pii/S1873506124002307
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