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Results: 1 to 20 of 38

1.

Erythrocyte-mediated delivery of phenylalanine ammonia lyase for the treatment of phenylketonuria in BTBR-Pahenu2 mice.

Rossi L, Pierigè F, Carducci C, Gabucci C, Pascucci T, Canonico B, Bell SM, Fitzpatrick PA, Leuzzi V, Magnani M.

J Control Release. 2014 Aug 21. pii: S0168-3659(14)00594-X. doi: 10.1016/j.jconrel.2014.08.012. [Epub ahead of print]

PMID:
25151978
[PubMed - as supplied by publisher]
2.

Charting a clear path: the ASGCT Standardized Pathways Conference.

Kiem HP, Baum C, Bushman FD, Byrne BJ, Carter BJ, Cavagnaro J, Malech HL, Mendell JR, Naldini LM, Sorrentino BP, Williams DA, Flotte TR.

Mol Ther. 2014 Jul;22(7):1235-8. doi: 10.1038/mt.2014.95. No abstract available.

PMID:
24981438
[PubMed - in process]
3.

Defining success in graduate school.

Bell SM, Blumstein J, Brose K, Carroll A, Chang J, Charles J, Haswell ES, Michelitsch M, Owens JC, Patil CK, Smith R, Tupy J, Walsh E, Ware T.

Mol Biol Cell. 2014 Jul 1;25(13):1942-4. doi: 10.1091/mbc.E14-03-0793.

PMID:
24970485
[PubMed - in process]
4.

Enzyme replacement therapy delays pupillary light reflex deficits in a canine model of late infantile neuronal ceroid lipofuscinosis.

Whiting RE, Narfström K, Yao G, Pearce JW, Coates JR, Castaner LJ, Jensen CA, Dougherty BN, Vuillemenot BR, Kennedy D, O'Neill CA, Katz ML.

Exp Eye Res. 2014 Aug;125:164-72. doi: 10.1016/j.exer.2014.06.008. Epub 2014 Jun 19.

PMID:
24954537
[PubMed - in process]
Free Article
5.

Single-dose, subcutaneous recombinant phenylalanine ammonia lyase conjugated with polyethylene glycol in adult patients with phenylketonuria: an open-label, multicentre, phase 1 dose-escalation trial.

Longo N, Harding CO, Burton BK, Grange DK, Vockley J, Wasserstein M, Rice GM, Dorenbaum A, Neuenburg JK, Musson DG, Gu Z, Sile S.

Lancet. 2014 Jul 5;384(9937):37-44. doi: 10.1016/S0140-6736(13)61841-3. Epub 2014 Apr 14.

PMID:
24743000
[PubMed - indexed for MEDLINE]
6.

A phase I dose escalation study of oral c-MET inhibitor tivantinib (ARQ 197) in combination with gemcitabine in patients with solid tumors.

Pant S, Saleh M, Bendell J, Infante JR, Jones S, Kurkjian CD, Moore KM, Kazakin J, Abbadessa G, Wang Y, Chen Y, Schwartz B, Camacho LH.

Ann Oncol. 2014 Jul;25(7):1416-21. doi: 10.1093/annonc/mdu157. Epub 2014 Apr 15.

PMID:
24737778
[PubMed - in process]
7.

Molecular testing of 163 patients with Morquio A (Mucopolysaccharidosis IVA) identifies 39 novel GALNS mutations.

Morrone A, Tylee KL, Al-Sayed M, Brusius-Facchin AC, Caciotti A, Church HJ, Coll MJ, Davidson K, Fietz MJ, Gort L, Hegde M, Kubaski F, Lacerda L, Laranjeira F, Leistner-Segal S, Mooney S, Pajares S, Pollard L, Ribeiro I, Wang RY, Miller N.

Mol Genet Metab. 2014 Jun;112(2):160-70. doi: 10.1016/j.ymgme.2014.03.004. Epub 2014 Mar 20.

PMID:
24726177
[PubMed - in process]
Free Article
8.

Sapropterin dihydrochloride use in pregnant women with phenylketonuria: an interim report of the PKU MOMS sub-registry.

Grange DK, Hillman RE, Burton BK, Yano S, Vockley J, Fong CT, Hunt J, Mahoney JJ, Cohen-Pfeffer JL; Phenylketonuria Demographics Outcomes and Safety (PKUDOS) registry; Maternal Phenylketonuria Observational Program (PKU MOMS) sub-registry.

Mol Genet Metab. 2014 May;112(1):9-16. doi: 10.1016/j.ymgme.2014.02.016. Epub 2014 Mar 12.

PMID:
24667082
[PubMed - in process]
Free Article
9.

Phenylketonuria Scientific Review Conference: state of the science and future research needs.

Camp KM, Parisi MA, Acosta PB, Berry GT, Bilder DA, Blau N, Bodamer OA, Brosco JP, Brown CS, Burlina AB, Burton BK, Chang CS, Coates PM, Cunningham AC, Dobrowolski SF, Ferguson JH, Franklin TD, Frazier DM, Grange DK, Greene CL, Groft SC, Harding CO, Howell RR, Huntington KL, Hyatt-Knorr HD, Jevaji IP, Levy HL, Lichter-Konecki U, Lindegren ML, Lloyd-Puryear MA, Matalon K, MacDonald A, McPheeters ML, Mitchell JJ, Mofidi S, Moseley KD, Mueller CM, Mulberg AE, Nerurkar LS, Ogata BN, Pariser AR, Prasad S, Pridjian G, Rasmussen SA, Reddy UM, Rohr FJ, Singh RH, Sirrs SM, Stremer SE, Tagle DA, Thompson SM, Urv TK, Utz JR, van Spronsen F, Vockley J, Waisbren SE, Weglicki LS, White DA, Whitley CB, Wilfond BS, Yannicelli S, Young JM.

Mol Genet Metab. 2014 Jun;112(2):87-122. doi: 10.1016/j.ymgme.2014.02.013. Epub 2014 Mar 6.

PMID:
24667081
[PubMed - in process]
10.

Recombinant human tripeptidyl peptidase-1 infusion to the monkey CNS: safety, pharmacokinetics, and distribution.

Vuillemenot BR, Kennedy D, Reed RP, Boyd RB, Butt MT, Musson DG, Keve S, Cahayag R, Tsuruda LS, O'Neill CA.

Toxicol Appl Pharmacol. 2014 May 15;277(1):49-57. doi: 10.1016/j.taap.2014.03.005. Epub 2014 Mar 15.

PMID:
24642058
[PubMed - indexed for MEDLINE]
11.

Poly (ADP-ribose) polymerase inhibitor LT-626: Sensitivity correlates with MRE11 mutations and synergizes with platinums and irinotecan in colorectal cancer cells.

McPherson LA, Shen Y, Ford JM.

Cancer Lett. 2014 Feb 28;343(2):217-23. doi: 10.1016/j.canlet.2013.10.034. Epub 2013 Nov 9.

PMID:
24215868
[PubMed - indexed for MEDLINE]
12.

Enzyme augmentation therapy enhances the therapeutic efficacy of bone marrow transplantation in mucopolysaccharidosis type II mice.

Akiyama K, Shimada Y, Higuchi T, Ohtsu M, Nakauchi H, Kobayashi H, Fukuda T, Ida H, Eto Y, Crawford BE, Brown JR, Ohashi T.

Mol Genet Metab. 2014 Feb;111(2):139-46. doi: 10.1016/j.ymgme.2013.09.013. Epub 2013 Sep 21.

PMID:
24100247
[PubMed - in process]
13.

Evaluation of the therapeutic potential of a CNP analog in a Fgfr3 mouse model recapitulating achondroplasia.

Lorget F, Kaci N, Peng J, Benoist-Lasselin C, Mugniery E, Oppeneer T, Wendt DJ, Bell SM, Bullens S, Bunting S, Tsuruda LS, O'Neill CA, Di Rocco F, Munnich A, Legeai-Mallet L.

Am J Hum Genet. 2012 Dec 7;91(6):1108-14. doi: 10.1016/j.ajhg.2012.10.014. Epub 2012 Nov 29.

PMID:
23200862
[PubMed - indexed for MEDLINE]
Free PMC Article
14.

A novel method for the large-scale production of PG-CNP37, a C-type natriuretic peptide analogue.

Long S, Wendt DJ, Bell SM, Taylor TW, Dewavrin JY, Vellard MC.

J Biotechnol. 2012 Dec 15;164(2):196-201. doi: 10.1016/j.jbiotec.2012.08.001. Epub 2012 Aug 28.

PMID:
22959861
[PubMed - indexed for MEDLINE]
15.

Biomarker analysis of Morquio syndrome: identification of disease state and drug responsive markers.

Martell L, Lau K, Mei M, Burnett V, Decker C, Foehr ED.

Orphanet J Rare Dis. 2011 Dec 16;6:84. doi: 10.1186/1750-1172-6-84.

PMID:
22176730
[PubMed - indexed for MEDLINE]
Free PMC Article
16.

Validation of an LC-MS/MS assay for detecting relevant disaccharides from keratan sulfate as a biomarker for Morquio A syndrome.

Martell LA, Cunico RL, Ohh J, Fulkerson W, Furneaux R, Foehr ED.

Bioanalysis. 2011 Aug;3(16):1855-66. doi: 10.4155/bio.11.172.

PMID:
21877895
[PubMed - indexed for MEDLINE]
17.

Intrathecal tripeptidyl-peptidase 1 reduces lysosomal storage in a canine model of late infantile neuronal ceroid lipofuscinosis.

Vuillemenot BR, Katz ML, Coates JR, Kennedy D, Tiger P, Kanazono S, Lobel P, Sohar I, Xu S, Cahayag R, Keve S, Koren E, Bunting S, Tsuruda LS, O'Neill CA.

Mol Genet Metab. 2011 Nov;104(3):325-37. doi: 10.1016/j.ymgme.2011.06.018. Epub 2011 Jun 30.

PMID:
21784683
[PubMed - indexed for MEDLINE]
18.

CFD-aided cell settler design optimization and scale-up: effect of geometric design and operational variables on separation performance.

Shen Y, Yanagimachi K.

Biotechnol Prog. 2011 Sep-Oct;27(5):1282-96. doi: 10.1002/btpr.636. Epub 2011 May 26.

PMID:
21618723
[PubMed - indexed for MEDLINE]
19.

The expression of soluble and active recombinant Haemophilus influenzae IgA1 protease in E. coli.

Long S, Phan E, Vellard MC.

J Biomed Biotechnol. 2010;2010:253983. doi: 10.1155/2010/253983. Epub 2010 Nov 30.

PMID:
21151648
[PubMed - indexed for MEDLINE]
Free PMC Article
20.

Clinical outcomes following hematopoietic stem cell transplantation for the treatment of mucopolysaccharidosis VI.

Turbeville S, Nicely H, Rizzo JD, Pedersen TL, Orchard PJ, Horwitz ME, Horwitz EM, Veys P, Bonfim C, Al-Seraihy A.

Mol Genet Metab. 2011 Feb;102(2):111-5. doi: 10.1016/j.ymgme.2010.09.010. Epub 2010 Oct 25.

PMID:
20980181
[PubMed - indexed for MEDLINE]
Free PMC Article

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