Randy J. Chandler, Ph.D. - Publications

2012 Biochemistry and Molecular Biology The George Washington University, Washington, DC, United States 
Molecular Biology, Genetics, Medicine and Surgery

33 high-probability publications. We are testing a new system for linking publications to authors. You can help! If you notice any inaccuracies, please sign in and mark papers as correct or incorrect matches. If you identify any major omissions or other inaccuracies in the publication list, please let us know.

Year Citation  Score
2020 Brooks P, Ottinger E, Portero D, Lomash RM, Alimardanov A, Terse P, Xu X, Chandler RJ, Geist Hauserman J, Esposito E, Bönnemann CG, Venditti CP, Austin C, Pariser A, Lo DC. The Platform Vector Gene Therapies (PaVe-GT) Project: Increasing the efficiency of adeno-associated virus (AAV) gene therapy clinical trial startup. Human Gene Therapy. PMID 32993373 DOI: 10.1089/hum.2020.259  0.92
2020 Chandler RJ, Venturoni LE, Liao J, Hubbard BT, Schneller JL, Hoffmann V, Gordo S, Zang S, Ko CW, Chau N, Chiang K, Kay MA, Barzel A, Venditti CP. Promoterless, nuclease-free genome editing confers a growth advantage for corrected hepatocytes in mice with methylmalonic acidemia. Hepatology (Baltimore, Md.). PMID 32976669 DOI: 10.1002/hep.31570  0.92
2019 Kang HR, Gjorgjieva M, Smith SN, Brooks ED, Chen Z, Burgess SM, Chandler RJ, Waskowicz LR, Grady KM, Li S, Mithieux G, Venditti CP, Rajas F, Koeberl DD. Pathogenesis of Hepatic Tumors following Gene Therapy in Murine and Canine Models of Glycogen Storage Disease. Molecular Therapy. Methods & Clinical Development. 15: 383-391. PMID 31890731 DOI: 10.1016/j.omtm.2019.10.016  0.92
2019 Chandler RJ, Venditti CP. Gene Therapy for Methylmalonic Acidemia: Past, Present, and Future. Human Gene Therapy. PMID 31303064 DOI: 10.1089/hum.2019.113  0.92
2018 Manoli I, Sysol JR, Epping MW, Li L, Wang C, Sloan JL, Pass A, Gagné J, Ktena YP, Li L, Trivedi NS, Ouattara B, Zerfas PM, Hoffmann V, Abu-Asab M, ... ... Chandler RJ, et al. FGF21 underlies a hormetic response to metabolic stress in methylmalonic acidemia. Jci Insight. 3. PMID 30518688 DOI: 10.1172/jci.insight.124351  0.92
2018 An D, Schneller JL, Frassetto A, Liang S, Zhu X, Park JS, Theisen M, Hong SJ, Zhou J, Rajendran R, Levy B, Howell R, Besin G, Presnyak V, Sabnis S, ... ... Chandler RJ, et al. Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia. Cell Reports. 24: 2520. PMID 30157442 DOI: 10.1016/j.celrep.2018.08.049  0.92
2017 An D, Schneller JL, Frassetto A, Liang S, Zhu X, Park JS, Theisen M, Hong SJ, Zhou J, Rajendran R, Levy B, Howell R, Besin G, Presnyak V, Sabnis S, ... ... Chandler RJ, et al. Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia. Cell Reports. 21: 3548-3558. PMID 29262333 DOI: 10.1016/j.celrep.2017.11.081  0.92
2017 Chandler RJ, Sands M, Venditti CP. rAAV integration and genotoxicity: insights from animal models. Human Gene Therapy. PMID 28293963 DOI: 10.1089/hum.2017.009  0.92
2016 Chandler RJ, Venditti CP. Gene Therapy for Metabolic Diseases. Translational Science of Rare Diseases. 1: 73-89. PMID 27853673 DOI: 10.3233/TRD-160007  0.92
2016 Chandler RJ, Williams IM, Gibson AL, Davidson CD, Incao AA, Hubbard BT, Porter FD, Pavan WJ, Venditti CP. Systemic AAV9 gene therapy improves the lifespan of mice with Niemann-Pick disease, type C1. Human Molecular Genetics. PMID 27798114 DOI: 10.1093/hmg/ddw367  0.92
2016 Chandler RJ, LaFave MC, Varshney GK, Burgess SM, Venditti CP. Genotoxicity in Mice Following AAV Gene Delivery: A Safety Concern for Human Gene Therapy? Molecular Therapy : the Journal of the American Society of Gene Therapy. 24: 198-201. PMID 26906613 DOI: 10.1038/mt.2016.17  0.84
2016 Harrington EA, Sloan JL, Manoli I, Chandler RJ, Schneider M, McGuire PJ, Calcedo R, Wilson JM, Venditti CP. Neutralizing Antibodies against AAV Capsids in Patients with mut Methylmalonic Acidemia (MMA). Human Gene Therapy. PMID 26790480 DOI: 10.1089/hum.2015.092  0.84
2015 Caterino M, Chandler RJ, Sloan JL, Dorko K, Cusmano-Ozog K, Ingenito L, Strom SC, Imperlini E, Scolamiero E, Venditti CP, Ruoppolo M. The proteome of methylmalonic acidemia (MMA): the elucidation of altered pathways in patient livers. Molecular Biosystems. PMID 26672496 DOI: 10.1039/c5mb00736d  0.84
2015 Chandler RJ, LaFave MC, Varshney GK, Trivedi NS, Carrillo-Carrasco N, Senac JS, Wu W, Hoffmann V, Elkahloun AG, Burgess SM, Venditti CP. Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy. The Journal of Clinical Investigation. 125: 870-80. PMID 25607839 DOI: 10.1172/JCI79213  0.92
2013 Chandler RJ, Tarasenko TN, Cusmano-Ozog K, Sun Q, Sutton VR, Venditti CP, McGuire PJ. Liver-directed adeno-associated virus serotype 8 gene transfer rescues a lethal murine model of citrullinemia type 1. Gene Therapy. 20: 1188-91. PMID 24131980 DOI: 10.1038/gt.2013.53  0.84
2013 Manoli I, Sysol JR, Li L, Houillier P, Garone C, Wang C, Zerfas PM, Cusmano-Ozog K, Young S, Trivedi NS, Cheng J, Sloan JL, Chandler RJ, Abu-Asab M, Tsokos M, et al. Targeting proximal tubule mitochondrial dysfunction attenuates the renal disease of methylmalonic acidemia. Proceedings of the National Academy of Sciences of the United States of America. 110: 13552-7. PMID 23898205 DOI: 10.1073/pnas.1302764110  0.84
2012 Chandler RJ, Venditti CP. Pre-clinical efficacy and dosing of an AAV8 vector expressing human methylmalonyl-CoA mutase in a murine model of methylmalonic acidemia (MMA). Molecular Genetics and Metabolism. 107: 617-9. PMID 23046887 DOI: 10.1016/j.ymgme.2012.09.019  0.84
2012 Sénac JS, Chandler RJ, Sysol JR, Li L, Venditti CP. Gene therapy in a murine model of methylmalonic acidemia using rAAV9-mediated gene delivery. Gene Therapy. 19: 385-91. PMID 21776024 DOI: 10.1038/gt.2011.108  0.84
2012 Carrillo-Carrasco N, Chandler RJ, Venditti CP. Combined methylmalonic acidemia and homocystinuria, cblC type. I. Clinical presentations, diagnosis and management. Journal of Inherited Metabolic Disease. 35: 91-102. PMID 21748409 DOI: 10.1007/s10545-011-9364-y  0.84
2011 Sloan JL, Johnston JJ, Manoli I, Chandler RJ, Krause C, Carrillo-Carrasco N, Chandrasekaran SD, Sysol JR, O'Brien K, Hauser NS, Sapp JC, Dorward HM, Huizing M, Barshop BA, et al. Exome sequencing identifies ACSF3 as a cause of combined malonic and methylmalonic aciduria. Nature Genetics. 43: 883-6. PMID 21841779 DOI: 10.1038/ng.908  0.84
2011 Chandler RJ, Venditti CP. A barrel of monkeys: scAAV8 gene therapy for hemophilia in nonhuman primates. Molecular Therapy : the Journal of the American Society of Gene Therapy. 19: 826-7. PMID 21532610 DOI: 10.1038/mt.2011.73  0.84
2011 Chandler RJ, Chandrasekaran S, Carrillo-Carrasco N, Senac JS, Hofherr SE, Barry MA, Venditti CP. Adeno-associated virus serotype 8 gene transfer rescues a neonatal lethal murine model of propionic acidemia. Human Gene Therapy. 22: 477-81. PMID 20950151 DOI: 10.1089/hum.2010.164  0.84
2010 Chandler RJ, Venditti CP. Long-term Rescue of a Lethal Murine Model of Methylmalonic Acidemia Using Adeno associated Viral Gene Therapy. Molecular Therapy : the Journal of the American Society of Gene Therapy. 18: 11-16. PMID 28178549 DOI: 10.1038/mt.2009.247  0.92
2010 Carrillo-Carrasco N, Chandler RJ, Chandrasekaran S, Venditti CP. Liver-directed recombinant adeno-associated viral gene delivery rescues a lethal mouse model of methylmalonic acidemia and provides long-term phenotypic correction. Human Gene Therapy. 21: 1147-54. PMID 20486773 DOI: 10.1089/hum.2010.008  0.84
2010 Murphy GE, Lowekamp BC, Zerfas PM, Chandler RJ, Narasimha R, Venditti CP, Subramaniam S. Ion-abrasion scanning electron microscopy reveals distorted liver mitochondrial morphology in murine methylmalonic acidemia. Journal of Structural Biology. 171: 125-32. PMID 20399866 DOI: 10.1016/j.jsb.2010.04.005  0.84
2010 Chandler RJ, Venditti CP. Long-term rescue of a lethal murine model of methylmalonic acidemia using adeno-associated viral gene therapy. Molecular Therapy : the Journal of the American Society of Gene Therapy. 18: 11-6. PMID 19861951 DOI: 10.1038/mt.2009.247  0.84
2009 Chandler RJ, Zerfas PM, Shanske S, Sloan J, Hoffmann V, DiMauro S, Venditti CP. Mitochondrial dysfunction in mut methylmalonic acidemia. Faseb Journal : Official Publication of the Federation of American Societies For Experimental Biology. 23: 1252-61. PMID 19088183 DOI: 10.1096/fj.08-121848  0.84
2008 Chandler RJ, Venditti CP. Adenovirus-mediated gene delivery rescues a neonatal lethal murine model of mut(0) methylmalonic acidemia. Human Gene Therapy. 19: 53-60. PMID 18052792 DOI: 10.1089/hum.2007.0118  0.84
2007 Chandler RJ, Sloan J, Fu H, Tsai M, Stabler S, Allen R, Kaestner KH, Kazazian HH, Venditti CP. Metabolic phenotype of methylmalonic acidemia in mice and humans: the role of skeletal muscle. Bmc Medical Genetics. 8: 64. PMID 17937813 DOI: 10.1186/1471-2350-8-64  0.84
2007 Chandler RJ, Tsai MS, Dorko K, Sloan J, Korson M, Freeman R, Strom S, Venditti CP. Adenoviral-mediated correction of methylmalonyl-CoA mutase deficiency in murine fibroblasts and human hepatocytes. Bmc Medical Genetics. 8: 24. PMID 17470278 DOI: 10.1186/1471-2350-8-24  0.84
2006 Chandler RJ, Aswani V, Tsai MS, Falk M, Wehrli N, Stabler S, Allen R, Sedensky M, Kazazian HH, Venditti CP. Propionyl-CoA and adenosylcobalamin metabolism in Caenorhabditis elegans: evidence for a role of methylmalonyl-CoA epimerase in intermediary metabolism. Molecular Genetics and Metabolism. 89: 64-73. PMID 16843692 DOI: 10.1016/j.ymgme.2006.06.001  0.84
2005 Chandler RJ, Venditti CP. Genetic and genomic systems to study methylmalonic acidemia. Molecular Genetics and Metabolism. 86: 34-43. PMID 16182581 DOI: 10.1016/j.ymgme.2005.07.020  0.84
2004 Sarkar R, Tetreault R, Gao G, Wang L, Bell P, Chandler R, Wilson JM, Kazazian HH. Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype. Blood. 103: 1253-60. PMID 14551134 DOI: 10.1182/blood-2003-08-2954  0.84
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