| In utero CRISPR-mediated therapeutic editing of metabolic genes AC Rossidis, JD Stratigis, AC Chadwick, HA Hartman, NJ Ahn, H Li, ... Nature medicine 24 (10), 1513-1518, 2018 | 206 | 2018 |
| In utero gene editing for monogenic lung disease D Alapati, WJ Zacharias, HA Hartman, AC Rossidis, JD Stratigis, NJ Ahn, ... Science translational medicine 11 (488), eaav8375, 2019 | 93 | 2019 |
| Efficient CRISPR/Cas9-mediated editing of trinucleotide repeat expansion in myotonic dystrophy patient-derived iPS and myogenic cells S Dastidar, S Ardui, K Singh, D Majumdar, N Nair, Y Fu, D Reyon, ... Nucleic acids research 46 (16), 8275-8298, 2018 | 89 | 2018 |
| Efficient in vivo liver-directed gene editing using CRISPR/Cas9 K Singh, H Evens, N Nair, MY Rincón, S Sarcar, E Samara-Kuko, ... Molecular Therapy 26 (5), 1241-1254, 2018 | 78 | 2018 |
| In Utero Adenine Base Editing Corrects Multi-Organ Pathology in a Lethal Lysosomal Storage Disease KS Sourav K., Brandon M. White, Meghana V. Kashyap, Apeksha Dave, Felix R ... Nature Communications 12 (1), 2021 | 48 | 2021 |
| Comprehensive transcriptome-wide analysis of spliceopathy correction of myotonic dystrophy using CRISPR-Cas9 in iPSCs-derived cardiomyocytes S Dastidar, D Majumdar, J Tipanee, K Singh, AF Klein, D Furling, ... Molecular Therapy 30 (1), 75-91, 2022 | 14 | 2022 |
| Combined targeting of EGFR/HER promotes anti-tumor efficacy in subsets of KRAS mutant lung cancer resistant to single EGFR blockade IA Umelo, O De Wever, P Kronenberger, J Van Deun, A Noor, K Singh, ... Oncotarget 6 (24), 20132, 2015 | 10 | 2015 |
| Hemophilia gene therapy: the end of the beginning? D De Wolf, K Singh, MK Chuah, T VandenDriessche Human Gene Therapy 34 (17-18), 782-792, 2023 | 9 | 2023 |
| In utero gene editing for monogenic lung disease. Sci Transl Med 11 D Alapati, WJ Zacharias, HA Hartman, AC Rossidis, JD Stratigis, NJ Ahn, ... | 5 | 2019 |
| AZ-3102 significantly increases survival and decreases neuroinflammation in a mouse model of Sandhoff disease K Landskroner, K Singh, M Mitchell, J Walia Molecular Genetics and Metabolism 138 (2), 107193, 2023 | 1 | 2023 |
| Gene Expression Profile in the Sandhoff Mouse Brain with Progression of Age K Singh, BM Quinville, M Mitchell, Z Chen, JS Walia Genes 13 (11), 2020, 2022 | 1 | 2022 |
| CRISPR/Cas9-mediated editing for dominant genetic disorders: efficient excision of trinucleotide repeat expansion in myotonic dystrophy S Dastidar, S Ardui, K Singh, N Nair, Y Fu, D Reyon, E Samara, MFM Gerli, ... Human Gene Therapy 28 (12), A7-A7, 2017 | 1 | 2017 |
| Prenatal Interventions for the Treatment of Congenital Disorders K Singh Regenerative Medicine, 259–268, 2023 | | 2023 |
| Liver-directed gene editing with crispr-cas9: Comprehensive on-target and off-target analyses K Singh | | 2023 |
| Efficient In utero RNA lipid nanoparticle-mediated gene editing for correction of type 1 tyrosinemia without the AAV-associated genotoxicity K Singh, R Riley, M Kashyap, B White, S Bose, H Li, A Dave, R Palanki, ... HUMAN GENE THERAPY 32 (19-20), A68-A68, 2021 | | 2021 |
| In Utero Lipid Nanoparticle Delivery of CRISPR Technology to Correct Hereditary Tyrosinemia Type 1 K Singh, RS Riley, MVV Kashyap, B White, SK Bose, H Li, R Palanki, ... MOLECULAR THERAPY 29 (4), 10-10, 2021 | | 2021 |
| Molecular Tools for Editing Globin Genes K Singh Fetal Hemoglobin: The Panacea for Major β-Hemoglobinopathies, 2020 | | 2020 |
| IN UTERO GENE EDITING FOR MONOGENIC LUNG DISEASE WJ ZACHARIAS, S ZHOU, J KATZEN, Y TOMER, AC CHADWICK, ... SCIENCE TRANSLATIONAL MEDICINE 11 (488), 2019 | | 2019 |
| Truncated gRNAs result in efficient in vivo liver-directed gene inactivation using CRISPR/Cas9 K Singh, H Evens, N Nair, MY Rincon, S Sarcar, E Samara, MK Chuah, ... Human Gene Therapy 29 (12), A112-A112, 2018 | | 2018 |
| CRISPR/Cas9-Mediated Editing of Trinucleotide Repeat Expansion in Myotonic Dystrophy S Dastidar, K Singh, N Nair, Y Fu, D Reyon, E Samara, MFM Gerli, ... Molecular Therapy 25 (5), 85-85, 2017 | | 2017 |
William J ZachariasCincinnati Children's Hospital在 cchmc.org 的电子邮件经过验证
