Spinal Muscular Atrophy Modeling and Treatment Advances by Induced Pluripotent Stem Cells Studies

Pluripotent Stem Cells ◽

Muscular Atrophy ◽

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

Human Gene Therapy ◽

10.1089/hum.2017.255 ◽

2018 ◽

Vol 29 (11) ◽

pp. 1252-1263 ◽

Cited By ~ 16

Author(s):

Miaojin Zhou ◽

Zhiqing Hu ◽

Liyan Qiu ◽

Tao Zhou ◽

Mai Feng ◽

...

Keyword(s):

Stem Cells ◽

Spinal Muscular Atrophy ◽

Pluripotent Stem Cells ◽

Muscular Atrophy ◽

Patient Specific ◽

Spinal Muscular Atrophy Patient ◽

Induced Pluripotent ◽

Genetic Conversion

Induced pluripotent stem cells from a spinal muscular atrophy patient

Nature ◽

10.1038/nature07677 ◽

2008 ◽

Vol 457 (7227) ◽

pp. 277-280 ◽

Cited By ~ 1019

Author(s):

Allison D. Ebert ◽

Junying Yu ◽

Ferrill F. Rose ◽

Virginia B. Mattis ◽

Christian L. Lorson ◽

...

Keyword(s):

Stem Cells ◽

Spinal Muscular Atrophy ◽

Pluripotent Stem Cells ◽

Muscular Atrophy ◽

Spinal Muscular Atrophy Patient ◽

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

Neurology ◽

10.1212/wnl.78.1_meetingabstracts.p03.176 ◽

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 ◽

Pluripotent Stem Cells ◽

Muscular Atrophy ◽

Cell Source ◽

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

Neurology ◽

10.1212/wnl.78.1_meetingabstracts.in8-2.002 ◽

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 ◽

Pluripotent Stem Cells ◽

Muscular Atrophy ◽

Cell Source ◽

Edaravone is a candidate agent for spinal muscular atrophy: In vitro analysis using a human induced pluripotent stem cells-derived disease model

European Journal of Pharmacology ◽

10.1016/j.ejphar.2017.08.005 ◽

2017 ◽

Vol 814 ◽

pp. 161-168 ◽

Cited By ~ 10

Author(s):

Shiori Ando ◽

Michinori Funato ◽

Kazuki Ohuchi ◽

Tsubasa Kameyama ◽

Satoshi Inagaki ◽

...

Keyword(s):

Stem Cells ◽

Spinal Muscular Atrophy ◽

Pluripotent Stem Cells ◽

Muscular Atrophy ◽

Disease Model ◽

Induced Pluripotent ◽

Vitro Analysis ◽

In Vitro Analysis

Modeling the differential phenotypes of spinal muscular atrophy with high-yield generation of motor neurons from human induced pluripotent stem cells

Oncotarget ◽

10.18632/oncotarget.14925 ◽

2017 ◽

Vol 8 (26) ◽

pp. 42030-42042 ◽

Cited By ~ 6

Author(s):

Xiang Lin ◽

Jin-Jing Li ◽

Wen-Jing Qian ◽

Qi-Jie Zhang ◽

Zhong-Feng Wang ◽

...

Keyword(s):

Stem Cells ◽

Spinal Muscular Atrophy ◽

Motor Neurons ◽

Pluripotent Stem Cells ◽

Muscular Atrophy ◽

High Yield ◽

Induced pluripotent stem cells for modeling of spinal muscular atrophy

iPSCs for Modeling Central Nervous System Disorders ◽

10.1016/b978-0-323-85764-2.00010-7 ◽

2021 ◽

pp. 147-170

Author(s):

Emily Welby ◽

Allison D. Ebert

Keyword(s):

Stem Cells ◽

Spinal Muscular Atrophy ◽

Pluripotent Stem Cells ◽

Muscular Atrophy ◽

P3.15 A model for motor neuron degeneration and treatment of Spinal Muscular Atrophy using human induced pluripotent stem cells

Neuromuscular Disorders ◽

10.1016/j.nmd.2011.06.909 ◽

2011 ◽

Vol 21 (9-10) ◽

pp. 686

Author(s):

S. Corti ◽

F. Magri ◽

M. Nizzardo ◽

C. Simone ◽

M. Falcone ◽

...

Keyword(s):

Stem Cells ◽

Spinal Muscular Atrophy ◽

Motor Neuron ◽

Pluripotent Stem Cells ◽

Muscular Atrophy ◽

Motor Neuron Degeneration ◽

Neuron Degeneration ◽

Faculty Opinions recommendation of Induced pluripotent stem cells from a spinal muscular atrophy patient.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.1147093.604216 ◽

2009 ◽

Author(s):

Steven Dowdy

Keyword(s):

Stem Cells ◽

Spinal Muscular Atrophy ◽

Pluripotent Stem Cells ◽

Muscular Atrophy ◽

Spinal Muscular Atrophy Patient ◽

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

Science Translational Medicine ◽

10.1126/scitranslmed.3004108 ◽

2012 ◽

Vol 4 (165) ◽

pp. 165ra162-165ra162 ◽

Cited By ~ 127

Author(s):

S. Corti ◽

M. Nizzardo ◽

C. Simone ◽

M. Falcone ◽

M. Nardini ◽

...

Keyword(s):

Stem Cells ◽

Spinal Muscular Atrophy ◽

Pluripotent Stem Cells ◽

Muscular Atrophy ◽