| Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy RJ Chandler, MC LaFave, GK Varshney, NS Trivedi, N Carrillo-Carrasco, ... The Journal of clinical investigation 125 (2), 870-880, 2015 | 372 | 2015 |
| Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype R Sarkar, R Tetreault, G Gao, L Wang, P Bell, R Chandler, JM Wilson, ... Blood 103 (4), 1253-1260, 2004 | 271 | 2004 |
| Combined methylmalonic acidemia and homocystinuria, cblC type. I. Clinical presentations, diagnosis and management N Carrillo-Carrasco, RJ Chandler, CP Venditti Journal of inherited metabolic disease 35, 91-102, 2012 | 248 | 2012 |
| Systemic messenger RNA therapy as a treatment for methylmalonic acidemia D An, JL Schneller, A Frassetto, S Liang, X Zhu, JS Park, M Theisen, ... Cell reports 21 (12), 3548-3558, 2017 | 233 | 2017 |
| Recombinant adeno-associated viral integration and genotoxicity: insights from animal models RJ Chandler, MS Sands, CP Venditti Human gene therapy 28 (4), 314-322, 2017 | 204 | 2017 |
| Mitochondrial dysfunction in mut methylmalonic acidemia RJ Chandler, PM Zerfas, S Shanske, J Sloan, V Hoffmann, S DiMauro, ... The FASEB Journal 23 (4), 1252, 2009 | 202 | 2009 |
| Targeting proximal tubule mitochondrial dysfunction attenuates the renal disease of methylmalonic acidemia I Manoli, JR Sysol, L Li, P Houillier, C Garone, C Wang, PM Zerfas, ... Proceedings of the National Academy of Sciences 110 (33), 13552-13557, 2013 | 125 | 2013 |
| Exome sequencing identifies ACSF3 as a cause of combined malonic and methylmalonic aciduria JL Sloan, JJ Johnston, I Manoli, RJ Chandler, C Krause, ... Nature genetics 43 (9), 883-886, 2011 | 119 | 2011 |
| Systemic AAV9 gene therapy improves the lifespan of mice with Niemann-Pick disease, type C1 RJ Chandler, IM Williams, AL Gibson, CD Davidson, AA Incao, ... Human molecular genetics 26 (1), 52-64, 2017 | 92 | 2017 |
| Evaluating the state of the science for adeno-associated virus integration: An integrated perspective DE Sabatino, FD Bushman, RJ Chandler, RG Crystal, BL Davidson, ... Molecular Therapy 30 (8), 2646-2663, 2022 | 82 | 2022 |
| Liver-directed adeno-associated virus serotype 8 gene transfer rescues a lethal murine model of citrullinemia type 1 RJ Chandler, TN Tarasenko, K Cusmano-Ozog, Q Sun, VR Sutton, ... Gene therapy 20 (12), 1188-1191, 2013 | 77 | 2013 |
| Metabolic phenotype of methylmalonic acidemia in mice and humans: the role of skeletal muscle RJ Chandler, J Sloan, H Fu, M Tsai, S Stabler, R Allen, KH Kaestner, ... BMC medical genetics 8, 1-11, 2007 | 73 | 2007 |
| Genetic and genomic systems to study methylmalonic acidemia RJ Chandler, CP Venditti Molecular genetics and metabolism 86 (1-2), 34-43, 2005 | 73 | 2005 |
| Liver-directed recombinant adeno-associated viral gene delivery rescues a lethal mouse model of methylmalonic acidemia and provides long-term phenotypic correction N Carrillo-Carrasco, RJ Chandler, S Chandrasekaran, CP Venditti Human gene therapy 21 (9), 1147-1154, 2010 | 69 | 2010 |
| Genotoxicity in mice following AAV gene delivery: a safety concern for human gene therapy? RJ Chandler, MC LaFave, GK Varshney, SM Burgess, CP Venditti Molecular Therapy 24 (2), 198-201, 2016 | 61 | 2016 |
| Long-term rescue of a lethal murine model of methylmalonic acidemia using adeno associated viral gene therapy RJ Chandler, CP Venditti Molecular Therapy 18 (1), 11-16, 2010 | 61 | 2010 |
| FGF21 underlies a hormetic response to metabolic stress in methylmalonic acidemia I Manoli, JR Sysol, MW Epping, L Li, C Wang, JL Sloan, A Pass, J Gagné, ... JCI insight 3 (23), 2018 | 57 | 2018 |
| Adeno-associated virus serotype 8 gene transfer rescues a neonatal lethal murine model of propionic acidemia RJ Chandler, S Chandrasekaran, N Carrillo-Carrasco, JS Senac, ... Human gene therapy 22 (4), 477-481, 2011 | 51 | 2011 |
| Adenovirus-mediated gene delivery rescues a neonatal lethal murine model of mut 0 methylmalonic acidemia RJ Chandler, CP Venditti Human gene therapy 19 (1), 53-60, 2008 | 51 | 2008 |
| Promoterless, nuclease‐free genome editing confers a growth advantage for corrected hepatocytes in mice with methylmalonic acidemia RJ Chandler, LE Venturoni, J Liao, BT Hubbard, JL Schneller, ... Hepatology 73 (6), 2223-2237, 2021 | 46 | 2021 |
Irini Manoli, MD, PhDClinician Associate Investigator, NHGRI, NIHVerified email at mail.nih.gov
