Seamless Genetic Conversion of SMN2 to SMN1 via CRISPR/Cpf1 and Single-Stranded Oligodeoxynucleotides in Spinal Muscular Atrophy Patient-Specific Induced Pluripotent Stem Cells

2018 ◽  
Vol 29 (11) ◽  
pp. 1252-1263 ◽  
Author(s):  
Miaojin Zhou ◽  
Zhiqing Hu ◽  
Liyan Qiu ◽  
Tao Zhou ◽  
Mai Feng ◽  
...  
Keyword(s):  
Stem Cells ◽  
Spinal Muscular Atrophy ◽  
Induced Pluripotent Stem Cells ◽  
Pluripotent Stem Cells ◽  
Muscular Atrophy ◽  
Patient Specific ◽  
Spinal Muscular Atrophy Patient ◽  
Induced Pluripotent ◽  
Genetic Conversion

scholarly journals Induced pluripotent stem cells from a spinal muscular atrophy patient

Nature ◽  
2008 ◽  
Vol 457 (7227) ◽  
pp. 277-280 ◽  
Author(s):  
Allison D. Ebert ◽  
Junying Yu ◽  
Ferrill F. Rose ◽  
Virginia B. Mattis ◽  
Christian L. Lorson ◽  
...  
Keyword(s):  
Stem Cells ◽  
Spinal Muscular Atrophy ◽  
Induced Pluripotent Stem Cells ◽  
Pluripotent Stem Cells ◽  
Muscular Atrophy ◽  
Spinal Muscular Atrophy Patient ◽  
Induced Pluripotent

Gene Corrected Spinal Muscular Atrophy-Induced Pluripotent Stem Cells and Motoneuron as a Model and Cell Source for Transplantation (P03.176)

Neurology ◽  
2012 ◽  
Vol 78 (Meeting Abstracts 1) ◽  
pp. P03.176-P03.176
Author(s):  
S. Corti ◽  
M. Nizzardo ◽  
C. Simone ◽  
M. Falcone ◽  
M. Nardini ◽  
...  
Keyword(s):  
Stem Cells ◽  
Spinal Muscular Atrophy ◽  
Induced Pluripotent Stem Cells ◽  
Pluripotent Stem Cells ◽  
Muscular Atrophy ◽  
Cell Source ◽  
Induced Pluripotent

Gene Corrected Spinal Muscular Atrophy-Induced Pluripotent Stem Cells and Motoneuron as a Model and Cell Source for Transplantation (IN8-2.002)

Neurology ◽  
2012 ◽  
Vol 78 (Meeting Abstracts 1) ◽  
pp. IN8-2.002-IN8-2.002
Author(s):  
S. Corti ◽  
M. Nizzardo ◽  
C. Simone ◽  
M. Falcone ◽  
M. Nardini ◽  
...  
Keyword(s):  
Stem Cells ◽  
Spinal Muscular Atrophy ◽  
Induced Pluripotent Stem Cells ◽  
Pluripotent Stem Cells ◽  
Muscular Atrophy ◽  
Cell Source ◽  
Induced Pluripotent

scholarly journals Genetic Correction of Human Induced Pluripotent Stem Cells from Patients with Spinal Muscular Atrophy

2012 ◽  
Vol 4 (165) ◽  
pp. 165ra162-165ra162 ◽  
Author(s):  
S. Corti ◽  
M. Nizzardo ◽  
C. Simone ◽  
M. Falcone ◽  
M. Nardini ◽  
...  
Keyword(s):  
Stem Cells ◽  
Spinal Muscular Atrophy ◽  
Induced Pluripotent Stem Cells ◽  
Pluripotent Stem Cells ◽  
Muscular Atrophy ◽  
Induced Pluripotent
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