Kshitiz Singh

Affiliations: 
Children’s Hospital of Philadelphia, Philadelphia, PA, United States 
Area:
Gene editing, myotonic dystrophy, tyrosinemia
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"Kshitiz Singh"
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Publications

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Singh K, Quinville BM, Mitchell M, et al. (2022) Gene Expression Profile in the Sandhoff Mouse Brain with Progression of Age. Genes. 13
Dastidar S, Majumdar D, Tipanee J, et al. (2021) Comprehensive transcriptome-wide analysis of spliceopathy correction using CRISPR/Cas9 in myotonic dystrophy patient specific iPSC-derived cardiomyocyte-like cells. Molecular Therapy : the Journal of the American Society of Gene Therapy
Bose SK, White BM, Kashyap MV, et al. (2021) In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease. Nature Communications. 12: 4291
Alapati D, Zacharias WJ, Hartman HA, et al. (2019) In utero gene editing for monogenic lung disease. Science Translational Medicine. 11
Rossidis AC, Stratigis JD, Chadwick AC, et al. (2018) In utero CRISPR-mediated therapeutic editing of metabolic genes. Nature Medicine. 24: 1513-1518
Dastidar S, Ardui S, Singh K, et al. (2018) Efficient CRISPR/Cas9-mediated editing of trinucleotide repeat expansion in myotonic dystrophy patient-derived iPS and myogenic cells. Nucleic Acids Research
Singh K, Evens H, Nair N, et al. (2018) Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9. Molecular Therapy : the Journal of the American Society of Gene Therapy
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