TY  - GEN
AU  - Zhou,Miaojin
AU  - Hu,Zhiqing
AU  - Qiu,Liyan
AU  - Zhou,Tao
AU  - Feng,Mai
AU  - Hu,Qian
AU  - Zeng,Baitao
AU  - Li,Zhuo
AU  - Sun,Qianru
AU  - Wu,Yong
AU  - Liu,Xionghao
AU  - Wu,Lingqian
AU  - Liang,Desheng
TI  - Seamless Genetic Conversion of SMN2 to SMN1 via CRISPR/Cpf1 and Single-Stranded Oligodeoxynucleotides in Spinal Muscular Atrophy Patient-Specific Induced Pluripotent Stem Cells
SN  - 1557-7422
PY  - 2019///1010
KW  - Base Sequence
KW  - Cell Differentiation
KW  - Clone Cells
KW  - Clustered Regularly Interspaced Short Palindromic Repeats
KW  - genetics
KW  - Gene Conversion
KW  - Genotype
KW  - Humans
KW  - Induced Pluripotent Stem Cells
KW  - metabolism
KW  - Male
KW  - Motor Neurons
KW  - Muscular Atrophy, Spinal
KW  - Oligodeoxyribonucleotides
KW  - Proto-Oncogene Proteins c-myc
KW  - RNA, Guide, CRISPR-Cas Systems
KW  - Survival of Motor Neuron 1 Protein
KW  - Survival of Motor Neuron 2 Protein
KW  - Young Adult
N1  - Publication Type: Journal Article; Research Support, Non-U.S. Gov't
UR  - https://doi.org/10.1089/hum.2017.255
ER  -