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Items: 50


Targeting Root Cause by Systemic scAAV9-hIDS Gene Delivery: Functional Correction and Reversal of Severe MPS II in Mice.

Fu H, Zaraspe K, Murakami N, Meadows AS, Pineda RJ, McCarty DM, Muenzer J.

Mol Ther Methods Clin Dev. 2018 Sep 4;10:327-340. doi: 10.1016/j.omtm.2018.07.005. eCollection 2018 Sep 21.


Differential Prevalence of Antibodies Against Adeno-Associated Virus in Healthy Children and Patients with Mucopolysaccharidosis III: Perspective for AAV-Mediated Gene Therapy.

Fu H, Meadows AS, Pineda RJ, Kunkler KL, Truxal KV, McBride KL, Flanigan KM, McCarty DM.

Hum Gene Ther Clin Dev. 2017 Dec;28(4):187-196. doi: 10.1089/humc.2017.109. Epub 2017 Oct 24.


Serum global metabolomics profiling reveals profound metabolic impairments in patients with MPS IIIA and MPS IIIB.

Fu H, Meadows AS, Pineda RJ, Mohney RP, Stirdivant S, McCarty DM.

Metab Brain Dis. 2017 Oct;32(5):1403-1415. doi: 10.1007/s11011-017-0009-1. Epub 2017 Apr 5.


Effective Depletion of Pre-existing Anti-AAV Antibodies Requires Broad Immune Targeting.

Velazquez VM, Meadows AS, Pineda RJ, Camboni M, McCarty DM, Fu H.

Mol Ther Methods Clin Dev. 2017 Jan 25;4:159-168. doi: 10.1016/j.omtm.2017.01.003. eCollection 2017 Mar 17.


Near-Complete Correction of Profound Metabolomic Impairments Corresponding to Functional Benefit in MPS IIIB Mice after IV rAAV9-hNAGLU Gene Delivery.

Fu H, Meadows AS, Ware T, Mohney RP, McCarty DM.

Mol Ther. 2017 Mar 1;25(3):792-802. doi: 10.1016/j.ymthe.2016.12.025. Epub 2017 Jan 28.


A prospective one-year natural history study of mucopolysaccharidosis types IIIA and IIIB: Implications for clinical trial design.

Truxal KV, Fu H, McCarty DM, McNally KA, Kunkler KL, Zumberge NA, Martin L, Aylward SC, Alfano LN, Berry KM, Lowes LP, Corridore M, McKee C, McBride KL, Flanigan KM.

Mol Genet Metab. 2016 Nov;119(3):239-248. doi: 10.1016/j.ymgme.2016.08.002. Epub 2016 Aug 18.


Crossing the blood-brain-barrier with viral vectors.

Fu H, McCarty DM.

Curr Opin Virol. 2016 Dec;21:87-92. doi: 10.1016/j.coviro.2016.08.006. Epub 2016 Sep 1. Review.


Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery.

Fu H, Cataldi MP, Ware TA, Zaraspe K, Meadows AS, Murrey DA, McCarty DM.

Mol Ther Methods Clin Dev. 2016 Jun 8;3:16036. doi: 10.1038/mtm.2016.36. eCollection 2016.


A GLP-Compliant Toxicology and Biodistribution Study: Systemic Delivery of an rAAV9 Vector for the Treatment of Mucopolysaccharidosis IIIB.

Meadows AS, Duncan FJ, Camboni M, Waligura K, Montgomery C, Zaraspe K, Naughton BJ, Bremer WG, Shilling C, Walker CM, Bolon B, Flanigan KM, McBride KL, McCarty DM, Fu H.

Hum Gene Ther Clin Dev. 2015 Dec;26(4):228-42. doi: 10.1089/humc.2015.132.


Broad functional correction of molecular impairments by systemic delivery of scAAVrh74-hSGSH gene delivery in MPS IIIA mice.

Duncan FJ, Naughton BJ, Zaraspe K, Murrey DA, Meadows AS, Clark KR, Newsom DE, White P, Fu H, McCarty DM.

Mol Ther. 2015 Apr;23(4):638-47. doi: 10.1038/mt.2015.9. Epub 2015 Jan 16.


Blood genome-wide transcriptional profiles reflect broad molecular impairments and strong blood-brain links in Alzheimer's disease.

Naughton BJ, Duncan FJ, Murrey DA, Meadows AS, Newsom DE, Stoicea N, White P, Scharre DW, Mccarty DM, Fu H.

J Alzheimers Dis. 2015;43(1):93-108. doi: 10.3233/JAD-140606.


Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates.

Murrey DA, Naughton BJ, Duncan FJ, Meadows AS, Ware TA, Campbell KJ, Bremer WG, Walker CM, Goodchild L, Bolon B, La Perle K, Flanigan KM, McBride KL, McCarty DM, Fu H.

Hum Gene Ther Clin Dev. 2014 Jun;25(2):72-84. doi: 10.1089/humc.2013.208. Epub 2014 Apr 10.


Amyloidosis, synucleinopathy, and prion encephalopathy in a neuropathic lysosomal storage disease: the CNS-biomarker potential of peripheral blood.

Naughton BJ, Duncan FJ, Murrey D, Ware T, Meadows A, McCarty DM, Fu H.

PLoS One. 2013 Nov 21;8(11):e80142. doi: 10.1371/journal.pone.0080142. eCollection 2013.


Hairpin-end conformation of adeno-associated virus genome determines interactions with DNA-repair pathways.

Cataldi MP, McCarty DM.

Gene Ther. 2013 Jun;20(6):686-93. doi: 10.1038/gt.2012.86. Epub 2012 Nov 15.


Peripheral nervous system neuropathology and progressive sensory impairments in a mouse model of Mucopolysaccharidosis IIIB.

Fu H, Bartz JD, Stephens RL Jr, McCarty DM.

PLoS One. 2012;7(9):e45992. doi: 10.1371/journal.pone.0045992. Epub 2012 Sep 25.


Patterns of scAAV vector insertion associated with oncogenic events in a mouse model for genotoxicity.

Rosas LE, Grieves JL, Zaraspe K, La Perle KM, Fu H, McCarty DM.

Mol Ther. 2012 Nov;20(11):2098-110. doi: 10.1038/mt.2012.197. Epub 2012 Sep 18.


Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery.

Fu H, Dirosario J, Killedar S, Zaraspe K, McCarty DM.

Mol Ther. 2011 Jun;19(6):1025-33. doi: 10.1038/mt.2011.34. Epub 2011 Mar 8.


Differential distribution of heparan sulfate glycoforms and elevated expression of heparan sulfate biosynthetic enzyme genes in the brain of mucopolysaccharidosis IIIB mice.

McCarty DM, DiRosario J, Gulaid K, Killedar S, Oosterhof A, van Kuppevelt TH, Martin PT, Fu H.

Metab Brain Dis. 2011 Mar;26(1):9-19. doi: 10.1007/s11011-010-9230-x. Epub 2011 Jan 12.


Mucopolysaccharidosis IIIB, a lysosomal storage disease, triggers a pathogenic CNS autoimmune response.

Killedar S, Dirosario J, Divers E, Popovich PG, McCarty DM, Fu H.

J Neuroinflammation. 2010 Jul 16;7:39. doi: 10.1186/1742-2094-7-39.


Differential effects of DNA double-strand break repair pathways on single-strand and self-complementary adeno-associated virus vector genomes.

Cataldi MP, McCarty DM.

J Virol. 2010 Sep;84(17):8673-82. doi: 10.1128/JVI.00641-10. Epub 2010 Jun 10.


Mannitol-facilitated CNS entry of rAAV2 vector significantly delayed the neurological disease progression in MPS IIIB mice.

McCarty DM, DiRosario J, Gulaid K, Muenzer J, Fu H.

Gene Ther. 2009 Nov;16(11):1340-52. doi: 10.1038/gt.2009.85. Epub 2009 Jul 9.


Innate and adaptive immune activation in the brain of MPS IIIB mouse model.

DiRosario J, Divers E, Wang C, Etter J, Charrier A, Jukkola P, Auer H, Best V, Newsom DL, McCarty DM, Fu H.

J Neurosci Res. 2009 Mar;87(4):978-90. doi: 10.1002/jnr.21912.


Self-complementary AAV vectors; advances and applications.

McCarty DM.

Mol Ther. 2008 Oct;16(10):1648-56. doi: 10.1038/mt.2008.171. Epub 2008 Aug 5. Review.


Significantly increased lifespan and improved behavioral performances by rAAV gene delivery in adult mucopolysaccharidosis IIIB mice.

Fu H, Kang L, Jennings JS, Moy SS, Perez A, Dirosario J, McCarty DM, Muenzer J.

Gene Ther. 2007 Jul;14(14):1065-77. Epub 2007 Apr 26.


Host cell DNA repair pathways in adeno-associated viral genome processing.

Choi VW, McCarty DM, Samulski RJ.

J Virol. 2006 Nov;80(21):10346-56.


Intrathecal long-term gene expression by self-complementary adeno-associated virus type 1 suitable for chronic pain studies in rats.

Storek B, Harder NM, Banck MS, Wang C, McCarty DM, Janssen WG, Morrison JH, Walsh CE, Beutler AS.

Mol Pain. 2006 Jan 30;2:4.


AAV hybrid serotypes: improved vectors for gene delivery.

Choi VW, McCarty DM, Samulski RJ.

Curr Gene Ther. 2005 Jun;5(3):299-310. Review.


Adeno-associated virus serotypes 1 to 5 mediated tumor cell directed gene transfer and improvement of transduction efficiency.

Hacker UT, Wingenfeld L, Kofler DM, Schuhmann NK, Lutz S, Herold T, King SB, Gerner FM, Perabo L, Rabinowitz J, McCarty DM, Samulski RJ, Hallek M, B√ľning H.

J Gene Med. 2005 Nov;7(11):1429-38.


Effects of adeno-associated virus DNA hairpin structure on recombination.

Choi VW, Samulski RJ, McCarty DM.

J Virol. 2005 Jun;79(11):6801-7.


Integration of adeno-associated virus (AAV) and recombinant AAV vectors.

McCarty DM, Young SM Jr, Samulski RJ.

Annu Rev Genet. 2004;38:819-45. Review.


Analysis of the interaction between adeno-associated virus and heparan sulfate using atomic force microscopy.

Negishi A, Chen J, McCarty DM, Samulski RJ, Liu J, Superfine R.

Glycobiology. 2004 Nov;14(11):969-77. Epub 2004 Jun 23.


Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo.

McCarty DM, Fu H, Monahan PE, Toulson CE, Naik P, Samulski RJ.

Gene Ther. 2003 Dec;10(26):2112-8.


Development and characterization of novel empty adenovirus capsids and their impact on cellular gene expression.

Stilwell JL, McCarty DM, Negishi A, Superfine R, Samulski RJ.

J Virol. 2003 Dec;77(23):12881-5.


Lentivirus vector purification using anion exchange HPLC leads to improved gene transfer.

Yamada K, McCarty DM, Madden VJ, Walsh CE.

Biotechniques. 2003 May;34(5):1074-8, 1080.


Roles of adeno-associated virus Rep protein and human chromosome 19 in site-specific recombination.

Young SM Jr, McCarty DM, Degtyareva N, Samulski RJ.

J Virol. 2000 May;74(9):3953-66.


Adeno-associated virus vectors for gene transfer into erythroid cells.

McCarty DM, Samulski RJ.

Curr Top Microbiol Immunol. 1996;218:75-91. Review. No abstract available.


Identification of linear DNA sequences that specifically bind the adeno-associated virus Rep protein.

McCarty DM, Pereira DJ, Zolotukhin I, Zhou X, Ryan JH, Muzyczka N.

J Virol. 1994 Aug;68(8):4988-97.


In vitro replication of adeno-associated virus DNA.

Ni TH, Zhou X, McCarty DM, Zolotukhin I, Muzyczka N.

J Virol. 1994 Feb;68(2):1128-38.


Analysis of mutations in adeno-associated virus Rep protein in vivo and in vitro.

McCarty DM, Ni TH, Muzyczka N.

J Virol. 1992 Jul;66(7):4050-7.


Nucleotide sequence of the Zea mays mitochondrial cytochrome oxidase subunit III gene.

McCarty DM, Hehman GL, Hauswirth WW.

Nucleic Acids Res. 1988 Oct 25;16(20):9873. No abstract available.


Sequence and transcriptional analysis of a chloroplast insert in the mitochondrial genome of Zea mays.

Fejes E, Masters BS, McCarty DM, Hauswirth WW.

Curr Genet. 1988 Jun;13(6):509-15.


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