Miaojin Zhou

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

Miaojin Zhou, Zhiqing Hu, Liyan Qiu et al. · Human Gene Therapy · 2018 · 67 citations

Life Sciences, Biotechnology, Genetics +17

ssODN-Mediated In-Frame Deletion with CRISPR/Cas9 Restores FVIII Function in Hemophilia A-Patient-Derived iPSCs and ECs

Zhiqing Hu, Miaojin Zhou, Yong Wu et al. · Molecular Therapy — Nucleic Acids · 2019 · 32 citations · Full text

Life Sciences, Genetics, Molecular Genetics +16

Targeting of the human F8 at the multicopy rDNA locus in Hemophilia a patient-derived iPSCs using TALENickases

Jialun Pang, Yong Wu, Zhuo Li et al. · Biochemical and Biophysical Research Communications · 2016 · 30 citations

Life Sciences, Genetics, Molecular Genetics +3

Amyotrophic lateral sclerosis (ALS) linked mutation in Ubiquilin 2 affects stress granule assembly via TIA‐1

Guangnan Peng, Ao Gu, Hongyan Niu et al. · CNS Neuroscience & Therapeutics · 2021 · 30 citations · Full text

Life Sciences, Molecular Biology, Neuroscience +16

Site-Specific Integration of <i>TRAIL</i> in iPSC-Derived Mesenchymal Stem Cells for Targeted Cancer Therapy

Zujia Wang, Hongting Chen, Peiyun Wang et al. · Stem Cells Translational Medicine · 2022 · 30 citations · Full text

Life Sciences, Cell Biology, Oncology +17