Year |
Citation |
Score |
2023 |
Chandler RJ, Di Pasquale G, Choi EY, Chang D, Smith SN, Sloan JL, Hoffmann V, Li L, Chiorini JA, Venditti CP. Systemic gene therapy using an AAV44.9 vector rescues a neonatal lethal mouse model of propionic acidemia. Molecular Therapy. Methods & Clinical Development. 30: 181-190. PMID 37746248 DOI: 10.1016/j.omtm.2023.06.008 |
0.71 |
|
2023 |
Chandler RJ, Venditti CP. Gene therapy for organic acidemias: Lessons learned from methylmalonic and propionic acidemia. Journal of Inherited Metabolic Disease. PMID 37530705 DOI: 10.1002/jimd.12665 |
0.688 |
|
2023 |
Lomash RM, Shchelochkov O, Chandler RJ, Venditti CP, Pariser A, Ottinger E. Successfully Navigating FDA Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The NIH Pave-GT Experience. Human Gene Therapy. PMID 36694456 DOI: 10.1089/hum.2022.232 |
0.621 |
|
2022 |
Chandler RJ, Di Pasquale G, Sloan JL, McCoy S, Hubbard BT, Kilts TM, Manoli I, Chiorini JA, Venditti CP. Systemic gene therapy for methylmalonic acidemia using the novel adeno-associated viral vector 44.9. Molecular Therapy. Methods & Clinical Development. 27: 61-72. PMID 36186952 DOI: 10.1016/j.omtm.2022.09.001 |
0.725 |
|
2022 |
Venturoni LE, Chandler RJ, Liao J, Hoffmann V, Ramesh N, Gordo S, Chau N, Venditti CP. Growth advantage of corrected hepatocytes in a juvenile model of methylmalonic acidemia following liver directed adeno-associated viral mediated nuclease-free genome editing. Molecular Genetics and Metabolism. 137: 1-8. PMID 35868241 DOI: 10.1016/j.ymgme.2022.06.011 |
0.728 |
|
2022 |
Sabatino DE, Bushman CFD, Chandler RJ, Crystal RG, Davidson BL, Dolmetsch R, Eggan KC, Gao G, Gil-Farina I, Kay MA, McCarty DM, Montini E, Ndu A, Yuan J. Evaluating the State of the Science for Adeno-Associated Virus (AAV) Integration:An Integrated Perspective. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 35690906 DOI: 10.1016/j.ymthe.2022.06.004 |
0.343 |
|
2021 |
Schneller JL, Lee CM, Venturoni LE, Chandler RJ, Li A, Myung S, Cradick TJ, Hurley AE, Lagor WR, Bao G, Venditti CP. genome editing at the albumin locus to treat methylmalonic acidemia. Molecular Therapy. Methods & Clinical Development. 23: 619-632. PMID 34901307 DOI: 10.1016/j.omtm.2021.11.004 |
0.67 |
|
2021 |
Ilyinskii PO, Michaud AM, Rizzo GL, Roy CJ, Leung SS, Elkins SL, Capela T, Chowdhury A, Li L, Chandler RJ, Manoli I, Andres-Mateos E, Johnston LPM, Vandenberghe LH, Venditti CP, et al. ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia. Molecular Therapy. Methods & Clinical Development. 22: 279-292. PMID 34485611 DOI: 10.1016/j.omtm.2021.06.015 |
0.637 |
|
2021 |
Davidson CD, Gibson AL, Gu T, Baxter LL, Deverman BE, Beadle K, Incao AA, Rodriguez-Gil JL, Fujiwara H, Jiang X, Chandler RJ, Ory DS, Gradinaru V, Venditti CP, Pavan WJ. Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann-Pick disease type C1 mice. Life Science Alliance. 4. PMID 34407999 DOI: 10.26508/lsa.202101040 |
0.675 |
|
2021 |
May FJ, Head PE, Venturoni LE, Chandler RJ, Venditti CP. Central nervous system-targeted adeno-associated virus gene therapy in methylmalonic acidemia. Molecular Therapy. Methods & Clinical Development. 21: 765-776. PMID 34169115 DOI: 10.1016/j.omtm.2021.04.005 |
0.695 |
|
2021 |
Ferla R, Alliegro M, Dell'Anno M, Nusco E, Cullen JM, Smith SN, Wolfsberg TG, O'Donnell P, Wang P, Nguyen AD, Chandler RJ, Chen Z, Burgess SM, Vite CH, Haskins ME, et al. Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors. Molecular Therapy. Methods & Clinical Development. 20: 247-257. PMID 33473358 DOI: 10.1016/j.omtm.2020.11.015 |
0.647 |
|
2020 |
Li Y, Miller CA, Shea LK, Jiang X, Guzman MA, Chandler RJ, Ramakrishnan SM, Smith SN, Venditti CP, Vogler CA, Ory DS, Ley TJ, Sands MS. Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe disease. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 33388420 DOI: 10.1016/j.ymthe.2020.12.031 |
0.694 |
|
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 |
0.653 |
|
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 |
0.693 |
|
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 |
0.669 |
|
2019 |
Chandler RJ. Messenger RNA therapy as an option for treating metabolic disorders. Proceedings of the National Academy of Sciences of the United States of America. PMID 31537746 DOI: 10.1073/Pnas.1914673116 |
0.484 |
|
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 |
0.702 |
|
2019 |
Davidson C, Gibson A, Gu T, Ali N, Chandler R, Venditti C, Walkley SU, Pavan W. Improved disease amelioration with combination therapy for Niemann-Pick disease type C1 Molecular Genetics and Metabolism. 126: S46. DOI: 10.1016/J.Ymgme.2018.12.100 |
0.621 |
|
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 |
0.682 |
|
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 |
0.551 |
|
2018 |
Davidson C, Gibson A, Gu T, Chandler R, Deverman B, Gradinaru V, Venditti C, Pavan W. Gene therapy for the treatment of Niemann-Pick disease type C1: Comparison of AAV9 to a novel serotype, AAV-PHP.B Molecular Genetics and Metabolism. 123: S36-S37. DOI: 10.1016/J.Ymgme.2017.12.075 |
0.667 |
|
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 |
0.708 |
|
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 |
0.705 |
|
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 |
0.67 |
|
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 |
0.713 |
|
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 |
0.703 |
|
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 |
0.639 |
|
2016 |
Gibson AL, Chandler RJ, Williams IM, Incao AA, Porter FD, Pavan WJ, Venditti CP. 613. A Comparison of CNS Transduction After Systemic versus Cranial Delivery of an AAV2/9 CamKII Promoter-eGFP Vector in Mice Molecular Therapy. 24: S243. DOI: 10.1016/S1525-0016(16)33421-9 |
0.684 |
|
2016 |
Schneller JL, Chandler RJ, Venditti CP. 359. Targeted Genome Editing Using TALENs to Correct a Mouse Model of Methylmalonic Acidemia (MMA) Molecular Therapy. 24: S144. DOI: 10.1016/S1525-0016(16)33168-9 |
0.746 |
|
2016 |
Hubbard BT, Chandler RJ, Venditti CP. 168. Assaying Hepatic Correction Mediated by Varied AAV Vectors in a Knock-Out Transgenic Mouse Model of Methylmalonic Acidemia (MMA) Molecular Therapy. 24: S65-S66. DOI: 10.1016/S1525-0016(16)32977-X |
0.743 |
|
2016 |
Chandler RJ, Barzel A, Kay MA, Venditti CP. 48. Treatment of Methylmalonic Acidemia by Promoterless Gene-Targeting Using Adeno-Associated Viral (AAV) Mediated Homologous Recombination Molecular Therapy. 24: S21-S22. DOI: 10.1016/S1525-0016(16)32857-X |
0.727 |
|
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 |
0.654 |
|
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 |
0.697 |
|
2015 |
Chandler RJ, Williams IM, Incao AA, Porter FD, Pavan WJ, Venditti CP. 198. Adeno-Associated Viral Gene Therapy To Treat Niemann-Pick Disease, Type C1 Molecular Therapy. 23: S79. DOI: 10.1016/S1525-0016(16)33803-5 |
0.726 |
|
2015 |
Schneller J, Senac J, Chandler R, Venditti CP. 176. An Internally Tagged Methylmalonyl-CoA Mutase (Mut) Displays In Vivo Activity after AAV9 Mediated Gene Delivery Molecular Therapy. 23: S70. DOI: 10.1016/S1525-0016(16)33781-9 |
0.721 |
|
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 |
0.742 |
|
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 |
0.643 |
|
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 |
0.683 |
|
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 |
0.744 |
|
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 |
0.628 |
|
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 |
0.601 |
|
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 |
0.699 |
|
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 |
0.742 |
|
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 |
0.72 |
|
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 |
0.755 |
|
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 |
0.57 |
|
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 |
0.741 |
|
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 |
0.637 |
|
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 |
0.739 |
|
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 |
0.665 |
|
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 |
0.711 |
|
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 |
0.611 |
|
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 |
0.601 |
|
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 |
0.526 |
|
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