| Year |
Citation |
Score |
| 2024 |
Baruteau J, Keshavan N, Venditti CP. Mission possible: Gene therapy for inherited metabolic diseases. Journal of Inherited Metabolic Disease. 47: 5-6. PMID 38221761 DOI: 10.1002/jimd.12708 |
0.371 |
|
| 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 |
0.704 |
|
| 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 |
0.684 |
|
| 2023 |
Manoli I, Gebremariam A, McCoy S, Pass AR, Gagné J, Hall C, Ferry S, Van Ryzin C, Sloan JL, Sacchetti E, Catesini G, Rizzo C, Martinelli D, Spada M, Dionisi-Vici C, ... Venditti CP, et al. Biomarkers to predict disease progression and therapeutic response in isolated methylmalonic acidemia (MMA). Journal of Inherited Metabolic Disease. PMID 37243446 DOI: 10.1002/jimd.12636 |
0.316 |
|
| 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 |
0.62 |
|
| 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 |
0.711 |
|
| 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 |
0.717 |
|
| 2022 |
Head PE, Myung S, Chen Y, Schneller JL, Wang C, Duncan N, Hoffman P, Chang D, Gebremariam A, Gucek M, Manoli I, Venditti CP. Aberrant methylmalonylation underlies methylmalonic acidemia and is attenuated by an engineered sirtuin. Science Translational Medicine. 14: eabn4772. PMID 35613279 DOI: 10.1126/scitranslmed.abn4772 |
0.367 |
|
| 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 |
0.666 |
|
| 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 |
0.634 |
|
| 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 |
0.667 |
|
| 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 |
0.69 |
|
| 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, ... Venditti CP, 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 |
0.639 |
|
| 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 |
0.683 |
|
| 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 |
0.643 |
|
| 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 |
0.683 |
|
| 2020 |
Sloan JL, Achilly NP, Arnold ML, Catlett JL, Blake T, Bishop K, Jones M, Harper U, English MA, Anderson S, Trivedi NS, Elkahloun A, Hoffmann V, Brooks BP, Sood R, ... Venditti CP, et al. The vitamin B12 processing enzyme, mmachc, is essential for zebrafish survival, growth and retinal morphology. Human Molecular Genetics. PMID 32186706 DOI: 10.1093/Hmg/Ddaa044 |
0.435 |
|
| 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 |
0.664 |
|
| 2019 |
Pavuluri K, Manoli I, Pass A, Li Y, Vernon HJ, Venditti CP, McMahon MT. Noninvasive monitoring of chronic kidney disease using pH and perfusion imaging. Science Advances. 5: eaaw8357. PMID 31453331 DOI: 10.1126/Sciadv.Aaw8357 |
0.318 |
|
| 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 |
0.698 |
|
| 2019 |
Shchelochkov OA, Manoli I, Sloan JL, Ferry S, Pass A, Van Ryzin C, Myles J, Schoenfeld M, McGuire P, Rosing DR, Levin MD, Kopp JB, Venditti CP. Chronic kidney disease in propionic acidemia. Genetics in Medicine : Official Journal of the American College of Medical Genetics. PMID 31249402 DOI: 10.1038/S41436-019-0593-Z |
0.307 |
|
| 2019 |
Tarasenko TN, Jestin M, Matsumoto S, Saito K, Hwang S, Gavrilova O, Trivedi N, Zerfas PM, Barca E, DiMauro S, Senac J, Venditti CP, Cherukuri M, McGuire PJ. Macrophage derived TNFα promotes hepatic reprogramming to Warburg-like metabolism. Journal of Molecular Medicine (Berlin, Germany). PMID 31053970 DOI: 10.1007/S00109-019-01786-W |
0.36 |
|
| 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 |
0.624 |
|
| 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, ... ... Venditti CP, et al. FGF21 underlies a hormetic response to metabolic stress in methylmalonic acidemia. Jci Insight. 3. PMID 30518688 DOI: 10.1172/Jci.Insight.124351 |
0.685 |
|
| 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, ... ... Venditti CP, 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 |
0.554 |
|
| 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 |
0.664 |
|
| 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, ... ... Venditti CP, 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 |
0.703 |
|
| 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 |
0.697 |
|
| 2017 |
Schneller JL, Lee CM, Bao G, Venditti CP. Genome editing for inborn errors of metabolism: advancing towards the clinic. Bmc Medicine. 15: 43. PMID 28238287 DOI: 10.1186/S12916-017-0798-4 |
0.41 |
|
| 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 |
0.666 |
|
| 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 |
0.704 |
|
| 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 |
0.691 |
|
| 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 |
0.644 |
|
| 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 |
0.678 |
|
| 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 |
0.734 |
|
| 2016 |
Gagné J, Manoli I, Harrington E, Smyth S, Hattenbach JD, Sloan JL, Chen KY, Venditti CP. 353. 1- 13 C-Propionate Oxidation as a Measure of Methylmalonyl-CoA Mutase (MUT) Activity in Methylmalonic Acidemia Molecular Therapy. 24: S141. DOI: 10.1016/S1525-0016(16)33162-8 |
0.393 |
|
| 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 |
0.735 |
|
| 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 |
0.717 |
|
| 2016 |
Arnold M, Sloan JL, Achilly NP, Elliot G, Onojafe IF, Brooks BP, Venditti CP. 46. The First Viable Mouse Model of cblC Type Combined Methylmalonic Acidemia and Homocysteinemia: AAV Gene Therapy Rescues Neonatal Lethality and Provides Insight into Disease-Associated Retinal Degeneration Molecular Therapy. 24: S20-S21. DOI: 10.1016/S1525-0016(16)32855-6 |
0.534 |
|
| 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 |
0.662 |
|
| 2015 |
Ktena YP, Ramstad T, Baker EH, Sloan JL, Mannes AJ, Manoli I, Venditti CP. Propofol administration in patients with methylmalonic acidemia and intracellular cobalamin metabolism disorders: a review of theoretical concerns and clinical experiences in 28 patients. Journal of Inherited Metabolic Disease. PMID 25985870 DOI: 10.1007/S10545-015-9816-X |
0.337 |
|
| 2015 |
Sloan JL, Manoli I, Venditti CP. Liver or combined liver-kidney transplantation for patients with isolated methylmalonic acidemia: who and when? The Journal of Pediatrics. 166: 1346-50. PMID 25882873 DOI: 10.1016/J.Jpeds.2015.03.026 |
0.31 |
|
| 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 |
0.686 |
|
| 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 |
0.717 |
|
| 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 |
0.714 |
|
| 2014 |
Vernon HJ, Sperati CJ, King JD, Poretti A, Miller NR, Sloan JL, Cameron AM, Myers D, Venditti CP, Valle D. A detailed analysis of methylmalonic acid kinetics during hemodialysis and after combined liver/kidney transplantation in a patient with mut (0) methylmalonic acidemia. Journal of Inherited Metabolic Disease. 37: 899-907. PMID 24961826 DOI: 10.1007/S10545-014-9730-7 |
0.346 |
|
| 2014 |
Zsengellér ZK, Aljinovic N, Teot LA, Korson M, Rodig N, Sloan JL, Venditti CP, Berry GT, Rosen S. Methylmalonic acidemia: a megamitochondrial disorder affecting the kidney. Pediatric Nephrology (Berlin, Germany). 29: 2139-46. PMID 24865477 DOI: 10.1007/S00467-014-2847-Y |
0.343 |
|
| 2014 |
Trakadis YJ, Alfares A, Bodamer OA, Buyukavci M, Christodoulou J, Connor P, Glamuzina E, Gonzalez-Fernandez F, Bibi H, Echenne B, Manoli I, Mitchell J, Nordwall M, Prasad C, Scaglia F, ... ... Venditti CP, et al. Update on transcobalamin deficiency: clinical presentation, treatment and outcome. Journal of Inherited Metabolic Disease. 37: 461-73. PMID 24305960 DOI: 10.1007/S10545-013-9664-5 |
0.36 |
|
| 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 |
0.731 |
|
| 2013 |
Yu HC, Sloan JL, Scharer G, Brebner A, Quintana AM, Achilly NP, Manoli I, Coughlin CR, Geiger EA, Schneck U, Watkins D, Suormala T, Van Hove JL, Fowler B, Baumgartner MR, ... ... Venditti CP, et al. An X-linked cobalamin disorder caused by mutations in transcriptional coregulator HCFC1. American Journal of Human Genetics. 93: 506-14. PMID 24011988 DOI: 10.1016/J.Ajhg.2013.07.022 |
0.358 |
|
| 2013 |
Tanpaiboon P, Venditti CP. Noncompaction of the ventricular myocardium and hydrops fetalis in cobalamin C disease: Response to letter "Disappearance of congenital noncompaction in hereditary cobalamin-C-deficiency 2.5 years after birth" by J. Finsterer and Claudia Stöllberger, DOI 10.1007/s10545-013-9612-4. Journal of Inherited Metabolic Disease. 36: 1085. PMID 23974651 DOI: 10.1007/S10545-013-9644-9 |
0.355 |
|
| 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, ... ... Venditti CP, 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 |
0.647 |
|
| 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 |
0.677 |
|
| 2012 |
O'Shea CJ, Sloan JL, Wiggs EA, Pao M, Gropman A, Baker EH, Manoli I, Venditti CP, Snow J. Neurocognitive phenotype of isolated methylmalonic acidemia. Pediatrics. 129: e1541-51. PMID 22614770 DOI: 10.1542/Peds.2011-1715 |
0.306 |
|
| 2012 |
Chapman KA, Gropman A, MacLeod E, Stagni K, Summar ML, Ueda K, Ah Mew N, Franks J, Island E, Matern D, Pena L, Smith B, Sutton VR, Urv T, Venditti C, et al. Acute management of propionic acidemia. Molecular Genetics and Metabolism. 105: 16-25. PMID 22000903 DOI: 10.1016/J.Ymgme.2011.09.026 |
0.335 |
|
| 2012 |
Pena L, Franks J, Chapman KA, Gropman A, Ah Mew N, Chakrapani A, Island E, MacLeod E, Matern D, Smith B, Stagni K, Sutton VR, Ueda K, Urv T, Venditti C, et al. Natural history of propionic acidemia. Molecular Genetics and Metabolism. 105: 5-9. PMID 21986446 DOI: 10.1016/J.Ymgme.2011.09.022 |
0.348 |
|
| 2012 |
Sutton VR, Chapman KA, Gropman AL, MacLeod E, Stagni K, Summar ML, Ueda K, Ah Mew N, Franks J, Island E, Matern D, Peña L, Smith B, Urv T, Venditti C, et al. Chronic management and health supervision of individuals with propionic acidemia. Molecular Genetics and Metabolism. 105: 26-33. PMID 21963082 DOI: 10.1016/J.Ymgme.2011.08.034 |
0.31 |
|
| 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 |
0.734 |
|
| 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 |
0.641 |
|
| 2012 |
Carrillo-Carrasco N, Venditti CP. Combined methylmalonic acidemia and homocystinuria, cblC type. II. Complications, pathophysiology, and outcomes. Journal of Inherited Metabolic Disease. 35: 103-14. PMID 21748408 DOI: 10.1007/S10545-011-9365-X |
0.361 |
|
| 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, ... ... Venditti CP, 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 |
0.611 |
|
| 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 |
0.69 |
|
| 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 |
0.731 |
|
| 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 |
0.707 |
|
| 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 |
0.746 |
|
| 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 |
0.58 |
|
| 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 |
0.73 |
|
| 2010 |
Carrillo-Carrasco N, Venditti CP. Hydroxocobalamin for cobalamin C: Which dosage? by V. Wiwanitkit Journal of Inherited Metabolic Disease. 33: 455. DOI: 10.1007/S10545-010-9092-8 |
0.331 |
|
| 2009 |
Carrillo-Carrasco N, Sloan J, Valle D, Hamosh A, Venditti CP. Hydroxocobalamin dose escalation improves metabolic control in cblC. Journal of Inherited Metabolic Disease. 32: 728-31. PMID 19821145 DOI: 10.1007/S10545-009-1257-Y |
0.347 |
|
| 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 |
0.643 |
|
| 2008 |
Buccafusca R, Venditti CP, Kenyon LC, Johanson RA, Van Bockstaele E, Ren J, Pagliardini S, Minarcik J, Golden JA, Coady MJ, Greer JJ, Berry GT. Characterization of the null murine sodium/myo-inositol cotransporter 1 (Smit1 or Slc5a3) phenotype: myo-inositol rescue is independent of expression of its cognate mitochondrial ribosomal protein subunit 6 (Mrps6) gene and of phosphatidylinositol levels in neonatal brain. Molecular Genetics and Metabolism. 95: 81-95. PMID 18675571 DOI: 10.1016/J.Ymgme.2008.05.008 |
0.434 |
|
| 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 |
0.73 |
|
| 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 |
0.671 |
|
| 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 |
0.708 |
|
| 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 |
0.624 |
|
| 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 |
0.613 |
|
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