Generation of two induced pluripotent stem cell lines from spinal muscular atrophy type 1 patients carrying no functional copies of SMN1 gene
Author:
Funder
National Institutes of Health
Stanford University
Publisher
Elsevier BV
Subject
Cell Biology,Developmental Biology,General Medicine
Reference7 articles.
1. Comprehensive SMN1 and SMN2 profiling for spinal muscular atrophy analysis using long-read PacBio HiFi sequencing;Chen;Am. J. Hum. Genet.,2023
2. Generation of two heterozygous MYBPC3 mutation-carrying human iPSC lines, SCVIi001-A and SCVIi002-A, for modeling hypertrophic cardiomyopathy;Liu;Stem Cell Res.,2021
3. A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy;Lorson;Proc. Natl. Acad. Sci. U S A,1999
4. Spinal muscular atrophy;Mercuri;Nat. Rev. Dis. Primers,2022
5. A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2;Monani;Hum. Mol. Genet.,1999
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1. Generation of two induced pluripotent stem cell lines from Duchenne muscular dystrophy patients;Stem Cell Research;2023-10
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