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Items: 33

1.

Messenger RNA therapy as an option for treating metabolic disorders.

Chandler RJ.

Proc Natl Acad Sci U S A. 2019 Oct 15;116(42):20804-20806. doi: 10.1073/pnas.1914673116. Epub 2019 Sep 19. No abstract available.

PMID:
31537746
2.

Gene Therapy for Methylmalonic Acidemia: Past, Present, and Future.

Chandler RJ, Venditti CP.

Hum Gene Ther. 2019 Oct;30(10):1236-1244. doi: 10.1089/hum.2019.113. Epub 2019 Aug 16.

PMID:
31303064
3.

FGF21 underlies a hormetic response to metabolic stress in methylmalonic acidemia.

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, Tsokos MG, Kleiner DE, Garone C, Cusmano-Ozog K, Enns GM, Vernon HJ, Andersson HC, Grunewald S, Elkahloun AG, Girard CL, Schnermann J, DiMauro S, Andres-Mateos E, Vandenberghe LH, Chandler RJ, Venditti CP.

JCI Insight. 2018 Dec 6;3(23). pii: 124351. doi: 10.1172/jci.insight.124351.

4.

Compliance in autism: Self-report in action.

Chandler RJ, Russell A, Maras KL.

Autism. 2019 May;23(4):1005-1017. doi: 10.1177/1362361318795479. Epub 2018 Aug 31.

PMID:
30168344
5.

Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia.

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, Murphy-Benenato KE, Kumarasinghe ES, Salerno T, Mihai C, Lukacs CM, Chandler RJ, Guey LT, Venditti CP, Martini PGV.

Cell Rep. 2018 Aug 28;24(9):2520. doi: 10.1016/j.celrep.2018.08.049. No abstract available.

6.

Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia.

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, Murphy-Benenato KE, Kumarasinghe ES, Salerno T, Mihai C, Lukacs CM, Chandler RJ, Guey LT, Venditti CP, Martini PGV.

Cell Rep. 2017 Dec 19;21(12):3548-3558. doi: 10.1016/j.celrep.2017.11.081. Erratum in: Cell Rep. 2018 Aug 28;24(9):2520.

7.

Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights from Animal Models.

Chandler RJ, Sands MS, Venditti CP.

Hum Gene Ther. 2017 Apr;28(4):314-322. doi: 10.1089/hum.2017.009.

8.

Gene Therapy for Metabolic Diseases.

Chandler RJ, Venditti CP.

Transl Sci Rare Dis. 2016;1(1):73-89.

9.

Systemic AAV9 gene therapy improves the lifespan of mice with Niemann-Pick disease, type C1.

Chandler RJ, Williams IM, Gibson AL, Davidson CD, Incao AA, Hubbard BT, Porter FD, Pavan WJ, Venditti CP.

Hum Mol Genet. 2017 Jan 1;26(1):52-64. doi: 10.1093/hmg/ddw367.

10.

Genotoxicity in Mice Following AAV Gene Delivery: A Safety Concern for Human Gene Therapy?

Chandler RJ, LaFave MC, Varshney GK, Burgess SM, Venditti CP.

Mol Ther. 2016 Feb;24(2):198-201. doi: 10.1038/mt.2016.17. No abstract available.

11.

Neutralizing Antibodies Against Adeno-Associated Viral Capsids in Patients with mut Methylmalonic Acidemia.

Harrington EA, Sloan JL, Manoli I, Chandler RJ, Schneider M, McGuire PJ, Calcedo R, Wilson JM, Venditti CP.

Hum Gene Ther. 2016 May;27(5):345-53. doi: 10.1089/hum.2015.092. Epub 2016 Mar 22.

12.

The proteome of methylmalonic acidemia (MMA): the elucidation of altered pathways in patient livers.

Caterino M, Chandler RJ, Sloan JL, Dorko K, Cusmano-Ozog K, Ingenito L, Strom SC, Imperlini E, Scolamiero E, Venditti CP, Ruoppolo M.

Mol Biosyst. 2016 Feb;12(2):566-74. doi: 10.1039/c5mb00736d.

13.

Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy.

Chandler RJ, LaFave MC, Varshney GK, Trivedi NS, Carrillo-Carrasco N, Senac JS, Wu W, Hoffmann V, Elkahloun AG, Burgess SM, Venditti CP.

J Clin Invest. 2015 Feb;125(2):870-80. doi: 10.1172/JCI79213. Epub 2015 Jan 20.

14.

Liver-directed adeno-associated virus serotype 8 gene transfer rescues a lethal murine model of citrullinemia type 1.

Chandler RJ, Tarasenko TN, Cusmano-Ozog K, Sun Q, Sutton VR, Venditti CP, McGuire PJ.

Gene Ther. 2013 Dec;20(12):1188-91. doi: 10.1038/gt.2013.53. Epub 2013 Oct 17.

15.

Targeting proximal tubule mitochondrial dysfunction attenuates the renal disease of methylmalonic acidemia.

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, Elkahloun AG, Rosen S, Enns GM, Berry GT, Hoffmann V, DiMauro S, Schnermann J, Venditti CP.

Proc Natl Acad Sci U S A. 2013 Aug 13;110(33):13552-7. doi: 10.1073/pnas.1302764110. Epub 2013 Jul 29.

16.

Pre-clinical efficacy and dosing of an AAV8 vector expressing human methylmalonyl-CoA mutase in a murine model of methylmalonic acidemia (MMA).

Chandler RJ, Venditti CP.

Mol Genet Metab. 2012 Nov;107(3):617-9. doi: 10.1016/j.ymgme.2012.09.019. Epub 2012 Sep 25.

17.

Exome sequencing identifies ACSF3 as a cause of combined malonic and methylmalonic aciduria.

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; NIH Intramural Sequencing Center Group, Barshop BA, Berry SA, James PM, Champaigne NL, de Lonlay P, Valayannopoulos V, Geschwind MD, Gavrilov DK, Nyhan WL, Biesecker LG, Venditti CP.

Nat Genet. 2011 Aug 14;43(9):883-6. doi: 10.1038/ng.908.

18.

Gene therapy in a murine model of methylmalonic acidemia using rAAV9-mediated gene delivery.

Sénac JS, Chandler RJ, Sysol JR, Li L, Venditti CP.

Gene Ther. 2012 Apr;19(4):385-91. doi: 10.1038/gt.2011.108. Epub 2011 Jul 21.

19.

Combined methylmalonic acidemia and homocystinuria, cblC type. I. Clinical presentations, diagnosis and management.

Carrillo-Carrasco N, Chandler RJ, Venditti CP.

J Inherit Metab Dis. 2012 Jan;35(1):91-102. doi: 10.1007/s10545-011-9364-y. Epub 2011 Jul 12. Review.

20.

A barrel of monkeys: scAAV8 gene therapy for hemophilia in nonhuman primates.

Chandler RJ, Venditti CP.

Mol Ther. 2011 May;19(5):826-7. doi: 10.1038/mt.2011.73. No abstract available.

21.

Adeno-associated virus serotype 8 gene transfer rescues a neonatal lethal murine model of propionic acidemia.

Chandler RJ, Chandrasekaran S, Carrillo-Carrasco N, Senac JS, Hofherr SE, Barry MA, Venditti CP.

Hum Gene Ther. 2011 Apr;22(4):477-81. doi: 10.1089/hum.2010.164. Epub 2011 Feb 16.

22.

Liver-directed recombinant adeno-associated viral gene delivery rescues a lethal mouse model of methylmalonic acidemia and provides long-term phenotypic correction.

Carrillo-Carrasco N, Chandler RJ, Chandrasekaran S, Venditti CP.

Hum Gene Ther. 2010 Sep;21(9):1147-54. doi: 10.1089/hum.2010.008.

23.

Ion-abrasion scanning electron microscopy reveals distorted liver mitochondrial morphology in murine methylmalonic acidemia.

Murphy GE, Lowekamp BC, Zerfas PM, Chandler RJ, Narasimha R, Venditti CP, Subramaniam S.

J Struct Biol. 2010 Aug;171(2):125-32. doi: 10.1016/j.jsb.2010.04.005. Epub 2010 Apr 23.

24.

A system-level view of optimizing high-channel-count wireless biosignal telemetry.

Chandler RJ, Gibson S, Karkare V, Farshchi S, Marković D, Judy JW.

Conf Proc IEEE Eng Med Biol Soc. 2009;2009:5525-30. doi: 10.1109/IEMBS.2009.5333187.

PMID:
19964127
25.

Long-term rescue of a lethal murine model of methylmalonic acidemia using adeno-associated viral gene therapy.

Chandler RJ, Venditti CP.

Mol Ther. 2010 Jan;18(1):11-6. doi: 10.1038/mt.2009.247. Epub 2009 Oct 27.

26.

Mitochondrial dysfunction in mut methylmalonic acidemia.

Chandler RJ, Zerfas PM, Shanske S, Sloan J, Hoffmann V, DiMauro S, Venditti CP.

FASEB J. 2009 Apr;23(4):1252-61. doi: 10.1096/fj.08-121848. Epub 2008 Dec 16.

27.
28.

Metabolic phenotype of methylmalonic acidemia in mice and humans: the role of skeletal muscle.

Chandler RJ, Sloan J, Fu H, Tsai M, Stabler S, Allen R, Kaestner KH, Kazazian HH, Venditti CP.

BMC Med Genet. 2007 Oct 15;8:64.

29.

Adenoviral-mediated correction of methylmalonyl-CoA mutase deficiency in murine fibroblasts and human hepatocytes.

Chandler RJ, Tsai MS, Dorko K, Sloan J, Korson M, Freeman R, Strom S, Venditti CP.

BMC Med Genet. 2007 Apr 30;8:24.

30.

Propionyl-CoA and adenosylcobalamin metabolism in Caenorhabditis elegans: evidence for a role of methylmalonyl-CoA epimerase in intermediary metabolism.

Chandler RJ, Aswani V, Tsai MS, Falk M, Wehrli N, Stabler S, Allen R, Sedensky M, Kazazian HH, Venditti CP.

Mol Genet Metab. 2006 Sep-Oct;89(1-2):64-73. Epub 2006 Jul 14.

31.

Genetic and genomic systems to study methylmalonic acidemia.

Chandler RJ, Venditti CP.

Mol Genet Metab. 2005 Sep-Oct;86(1-2):34-43. Epub 2005 Sep 22. Review.

32.

Creep movements in low gradient clay slopes since the late glacial.

Chandler RJ, Pook MJ.

Nature. 1971 Feb 5;229(5284):399-400. No abstract available.

PMID:
16059261
33.

Studies on protozoa. Part II: The golden hamster (Cricetus auratus) and cotton rat (Sigmodon hispidus) as experimental hosts for Leishmania donovani.

FULTON JD, JOYNER LP, CHANDLER RJ.

Trans R Soc Trop Med Hyg. 1950 Jul;44(1):105-12. No abstract available.

PMID:
15443044

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