| An improved technique for chromosomal analysis of human ES and iPS cells D Moralli, M Yusuf, MA Mandegar, S Khoja, ZL Monaco, EV Volpi Stem Cell Reviews and Reports 7, 471-477, 2011 | 59 | 2011 |
| Genetic analysis of HIV-1 subtypes in Nairobi, Kenya S Khoja, P Ojwang, S Khan, N Okinda, R Harania, S Ali PloS one 3 (9), e3191, 2008 | 52 | 2008 |
| CD13 and ROR2 permit isolation of highly enriched cardiac mesoderm from differentiating human embryonic stem cells RJP Skelton, B Brady, S Khoja, D Sahoo, J Engel, D Arasaratnam, ... Stem Cell Reports 6 (1), 95-108, 2016 | 29 | 2016 |
| Evidence for a "Founder Effect" among HIV-infected injection drug users (IDUs) in Pakistan MA Rai, VR Nerurkar, S Khoja, S Khan, R Yanagihara, A Rehman, ... BMC infectious diseases 10 (1), 7, 2010 | 28 | 2010 |
| Functional human artificial chromosomes are generated and stably maintained in human embryonic stem cells MA Mandegar, D Moralli, S Khoja, S Cowley, DYL Chan, M Yusuf, ... Human molecular genetics 20 (15), 2905-2913, 2011 | 27 | 2011 |
| Noncoding centromeric RNA expression impairs chromosome stability in human and murine stem cells DYL Chan, D Moralli, S Khoja, ZL Monaco Disease markers 2017, 2017 | 26 | 2017 |
| Conditional disruption of hepatic carbamoyl phosphate synthetase 1 in mice results in hyperammonemia without orotic aciduria and can be corrected by liver-directed gene therapy S Khoja, M Nitzahn, K Hermann, B Truong, R Borzone, B Willis, M Rudd, ... Molecular genetics and metabolism 124 (4), 243-253, 2018 | 23 | 2018 |
| Magnetic resonance imaging of iron oxide-labeled human embryonic stem cell-derived cardiac progenitors RJP Skelton, S Khoja, S Almeida, S Rapacchi, F Han, J Engel, P Zhao, ... Stem cells translational medicine 5 (1), 67-74, 2016 | 23 | 2016 |
| Human hepatocyte transplantation corrects the inherited metabolic liver disorder arginase deficiency in mice SAK Angarita, B Truong, S Khoja, M Nitzahn, AK Rajbhandari, I Zhuravka, ... Molecular genetics and metabolism 124 (2), 114-123, 2018 | 14 | 2018 |
| Intermittent lipid nanoparticle mRNA administration prevents cortical dysmyelination associated with arginase deficiency S Khoja, XB Liu, B Truong, M Nitzahn, J Lambert, A Eliav, E Nasser, ... Molecular Therapy-Nucleic Acids 28, 859-874, 2022 | 13 | 2022 |
| Split AAV-mediated gene therapy restores ureagenesis in a murine model of carbamoyl phosphate synthetase 1 deficiency M Nitzahn, G Allegri, S Khoja, B Truong, G Makris, J Häberle, GS Lipshutz Molecular Therapy 28 (7), 1717-1730, 2020 | 13 | 2020 |
| Hepatic glutamine synthetase augmentation enhances ammonia detoxification LR Soria, M Nitzahn, A De Angelis, S Khoja, S Attanasio, P Annunziata, ... Journal of inherited metabolic disease 42 (6), 1128-1135, 2019 | 13 | 2019 |
| A constitutive knockout of murine carbamoyl phosphate synthetase 1 results in death with marked hyperglutaminemia and hyperammonemia S Khoja, M Nitzahn, B Truong, J Lambert, B Willis, G Allegri, V Rüfenacht, ... Journal of inherited metabolic disease 42 (6), 1044-1053, 2019 | 11 | 2019 |
| Focus: Rare Disease: CRISPR-Mediated Genomic Addition to CPS1 Deficient iPSCs is Insufficient to Restore Nitrogen Homeostasis M Nitzahn, B Truong, S Khoja, A Vega-Crespo, C Le, A Eliav, G Makris, ... The Yale Journal of Biology and Medicine 94 (4), 545, 2021 | 7 | 2021 |
| Gene therapy for guanidinoacetate methyltransferase deficiency restores cerebral and myocardial creatine while resolving behavioral abnormalities S Khoja, J Lambert, M Nitzahn, A Eliav, YC Zhang, M Tamboline, CT Le, ... Molecular Therapy-Methods & Clinical Development 25, 278-296, 2022 | 5 | 2022 |
| Gene Therapy for Guanidinoacetate Methyltransferase Deficiency Restores Cerebral and Myocardial Creatine While Resolving Behavioral Abnormalities YC Zhang, GS Lipshutz, S Khoja, M Nitzahn MOLECULAR THERAPY 30 (4), 118-119, 2022 | | 2022 |
| Developing an Improved Understanding for the Neurophenotype of SLC6A8 Deficiency CT Le, M Nitzahn, S Khoja, J Rodriguez, G Lipshutz MOLECULAR THERAPY 30 (4), 469-470, 2022 | | 2022 |
| Developing a Gene Therapy Approach for Guanidinoacetate Methyltransferase Deficiency, a Creatine Deficiency Disorder S Khoja, M Nitzahn, J Lambert, A Eliav, E Nasser, GS Lipshutz MOLECULAR THERAPY 29 (4), 237-237, 2021 | | 2021 |
| Development of Cell-Based Therapies for the Urea Cycle Disorder CPS1 Deficiency M Nitzahn, B Truong, S Khoja, G Lipshutz MOLECULAR THERAPY 29 (4), 243-244, 2021 | | 2021 |
| Minimal Protein from DNA Mini Circles Provides Therapeutic Benefit in CPS1 Deficiency M Nitzahn, S Khoja, J Lambert, HM Grisch-Chan, B Thony, GS Lipshutz MOLECULAR THERAPY 28 (4), 125-125, 2020 | | 2020 |
Mohammad A. MandegarGladstone Institutes / UCSFVerified email at gladstone.ucsf.edu
