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Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatment of Sanfilippo type B.

Grover A, Crippen-Harmon D, Nave L, Vincelette J, Wait JCM, Melton AC, Lawrence R, Brown JR, Webster KA, Yip BK, Baridon B, Vitelli C, Rigney S, Christianson TM, Tiger PMN, Lo MJ, Holtzinger J, Shaywitz AJ, Crawford BE, Fitzpatrick PA, LeBowitz JH, Bullens S, Aoyagi-Scharber M, Bunting S, O'Neill CA, Pinkstaff J, Bagri A.

Drug Deliv Transl Res. 2020 Jan 15. doi: 10.1007/s13346-019-00683-6. [Epub ahead of print]


Osmolyte transporter expression is reduced in photoaged human skin: Implications for skin hydration in aging.

Foster AR, El Chami C, O'Neill CA, Watson REB.

Aging Cell. 2020 Jan;19(1):e13058. doi: 10.1111/acel.13058. Epub 2019 Nov 26.


Consumer Safety Considerations of Skin and Oral Microbiome Perturbation.

McBain AJ, O'Neill CA, Amezquita A, Price LJ, Faust K, Tett A, Segata N, Swann JR, Smith AM, Murphy B, Hoptroff M, James G, Reddy Y, Dasgupta A, Ross T, Chapple IL, Wade WG, Fernandez-Piquer J.

Clin Microbiol Rev. 2019 Jul 31;32(4). pii: e00051-19. doi: 10.1128/CMR.00051-19. Print 2019 Sep 18. Review.


CRISPR/Cas9 mediated generation of an ovine model for infantile neuronal ceroid lipofuscinosis (CLN1 disease).

Eaton SL, Proudfoot C, Lillico SG, Skehel P, Kline RA, Hamer K, Rzechorzek NM, Clutton E, Gregson R, King T, O'Neill CA, Cooper JD, Thompson G, Whitelaw CB, Wishart TM.

Sci Rep. 2019 Jul 9;9(1):9891. doi: 10.1038/s41598-019-45859-9.


The Impact of Pre-existing Immunity on the Non-clinical Pharmacodynamics of AAV5-Based Gene Therapy.

Long BR, Sandza K, Holcomb J, Crockett L, Hayes GM, Arens J, Fonck C, Tsuruda LS, Schweighardt B, O'Neill CA, Zoog S, Vettermann C.

Mol Ther Methods Clin Dev. 2019 Apr 11;13:440-452. doi: 10.1016/j.omtm.2019.03.006. eCollection 2019 Jun 14.


Ex Vivo Gene Therapy: Graft-versus-host Disease (GVHD) in NSG™ (NOD.Cg-Prkdcscid Il2rgtm1Wjl/SzJ) Mice Transplanted with CD34+ Human Hematopoietic Stem Cells.

Chandra S, Cristofori P, Fonck C, O'Neill CA.

Toxicol Pathol. 2019 Jul;47(5):656-660. doi: 10.1177/0192623319844484. Epub 2019 May 7.


Immunogenicity to cerliponase alfa intracerebroventricular enzyme replacement therapy for CLN2 disease: Results from a Phase 1/2 study.

Cherukuri A, Cahan H, de Hart G, Van Tuyl A, Slasor P, Bray L, Henshaw J, Ajayi T, Jacoby D, O'Neill CA, Schweighardt B.

Clin Immunol. 2018 Dec;197:68-76. doi: 10.1016/j.clim.2018.09.003. Epub 2018 Sep 8.


Organic osmolytes preserve the function of the developing tight junction in ultraviolet B-irradiated rat epidermal keratinocytes.

El-Chami C, Haslam IS, Steward MC, O'Neill CA.

Sci Rep. 2018 Mar 26;8(1):5167. doi: 10.1038/s41598-018-22533-0.


Gene Therapy with BMN 270 Results in Therapeutic Levels of FVIII in Mice and Primates and Normalization of Bleeding in Hemophilic Mice.

Bunting S, Zhang L, Xie L, Bullens S, Mahimkar R, Fong S, Sandza K, Harmon D, Yates B, Handyside B, Sihn CR, Galicia N, Tsuruda L, O'Neill CA, Bagri A, Colosi P, Long S, Vehar G, Carter B.

Mol Ther. 2018 Feb 7;26(2):496-509. doi: 10.1016/j.ymthe.2017.12.009. Epub 2017 Dec 14.


Maternal phenylketonuria syndrome: studies in mice suggest a potential approach to a continuing problem.

Zeile WL, McCune HC, Musson DG, O'Donnell B, O'Neill CA, Tsuruda LS, Zori RT, Laipis PJ.

Pediatr Res. 2018 Apr;83(4):889-896. doi: 10.1038/pr.2017.323. Epub 2018 Jan 31.


The Impact of Ultraviolet Radiation on Barrier Function in Human Skin: Molecular Mechanisms and Topical Therapeutics.

Alhasaniah A, Sherratt MJ, O'Neill CA.

Curr Med Chem. 2018;25(40):5503-5511. doi: 10.2174/0929867324666171106164916. Review.


Partial rescue of neuropathology in the murine model of PKU following administration of recombinant phenylalanine ammonia lyase (pegvaliase).

Goldfinger M, Zeile WL, Corado CR, O'Neill CA, Tsuruda LS, Laipis PJ, Cooper JD.

Mol Genet Metab. 2017 Sep;122(1-2):33-35. doi: 10.1016/j.ymgme.2017.04.013. Epub 2017 Apr 29.


Long-term Immunogenicity of Elosulfase Alfa in the Treatment of Morquio A Syndrome: Results From MOR-005, a Phase III Extension Study.

Long B, Tompkins T, Decker C, Jesaitis L, Khan S, Slasor P, Harmatz P, O'Neill CA, Schweighardt B.

Clin Ther. 2017 Jan;39(1):118-129.e3. doi: 10.1016/j.clinthera.2016.11.017. Epub 2016 Dec 10.


Reveglucosidase alfa (BMN 701), an IGF2-Tagged rhAcid α-Glucosidase, Improves Respiratory Functional Parameters in a Murine Model of Pompe Disease.

Peng J, Dalton J, Butt M, Tracy K, Kennedy D, Haroldsen P, Cahayag R, Zoog S, O'Neill CA, Tsuruda LS.

J Pharmacol Exp Ther. 2017 Feb;360(2):313-323. doi: 10.1124/jpet.116.235952. Epub 2016 Nov 16.


Osteochondral Allograft Donor-Host Matching by the Femoral Condyle Radius of Curvature.

Bernstein DT, O'Neill CA, Kim RS, Jones HL, Noble PC, Harris JD, McCulloch PC.

Am J Sports Med. 2017 Feb;45(2):403-409. doi: 10.1177/0363546516671519. Epub 2016 Oct 29.


Antibodies that neutralize cellular uptake of elosulfase alfa are not associated with reduced efficacy or pharmacodynamic effect in individuals with Morquio A syndrome.

Melton AC, Soon RK Jr, Tompkins T, Long B, Schweighardt B, Qi Y, Vitelli C, Bagri A, Decker C, O'Neill CA, Zoog SJ, Jesaitis L.

J Immunol Methods. 2017 Jan;440:41-51. doi: 10.1016/j.jim.2016.10.006. Epub 2016 Oct 24.


The gut-skin axis in health and disease: A paradigm with therapeutic implications.

O'Neill CA, Monteleone G, McLaughlin JT, Paus R.

Bioessays. 2016 Nov;38(11):1167-1176. doi: 10.1002/bies.201600008. Epub 2016 Aug 24. Review.


Workshop Proceedings: Streamlined Development of Safety Assessment Programs Supporting Orphan/Rare Diseases-Are We There Yet?

Allamneni KP, Parker S, O'Neill CA, Wright TL, King S, Andrews L.

Int J Toxicol. 2016 Jul;35(4):393-409. doi: 10.1177/1091581816644709. Epub 2016 Jun 6.


Pharmacodynamics, pharmacokinetics and biodistribution of recombinant human N-acetylgalactosamine 4-sulfatase after 6months of therapy in cats using different IV infusion durations.

Ruane T, Haskins M, Cheng A, Wang P, Aguirre G, Knox VW 4th, Qi Y, Tompkins T, O'Neill CA.

Mol Genet Metab. 2016 Feb;117(2):157-63. doi: 10.1016/j.ymgme.2015.10.006. Epub 2015 Oct 21.


Lactobacillus rhamnosus GG Lysate Increases Re-Epithelialization of Keratinocyte Scratch Assays by Promoting Migration.

Mohammedsaeed W, Cruickshank S, McBain AJ, O'Neill CA.

Sci Rep. 2015 Nov 5;5:16147. doi: 10.1038/srep16147.


Effects of Food Intake on the Relative Bioavailability of Amifampridine Phosphate Salt in Healthy Adults.

Haroldsen PE, Musson DG, Hanson B, Quartel A, O'Neill CA.

Clin Ther. 2015 Jul 1;37(7):1555-63. doi: 10.1016/j.clinthera.2015.05.498. Epub 2015 Jun 20.


Genetic variation in aryl N-acetyltransferase results in significant differences in the pharmacokinetic and safety profiles of amifampridine (3,4-diaminopyridine) phosphate.

Haroldsen PE, Garovoy MR, Musson DG, Zhou H, Tsuruda L, Hanson B, O'Neill CA.

Pharmacol Res Perspect. 2015 Feb;3(1):e00099. doi: 10.1002/prp2.99. Epub 2014 Dec 9.


Neutral endopeptidase-resistant C-type natriuretic peptide variant represents a new therapeutic approach for treatment of fibroblast growth factor receptor 3-related dwarfism.

Wendt DJ, Dvorak-Ewell M, Bullens S, Lorget F, Bell SM, Peng J, Castillo S, Aoyagi-Scharber M, O'Neill CA, Krejci P, Wilcox WR, Rimoin DL, Bunting S.

J Pharmacol Exp Ther. 2015 Apr;353(1):132-49. doi: 10.1124/jpet.114.218560. Epub 2015 Feb 3.


Immunogenicity of Elosulfase Alfa, an Enzyme Replacement Therapy in Patients With Morquio A Syndrome: Results From MOR-004, a Phase III Trial.

Schweighardt B, Tompkins T, Lau K, Jesaitis L, Qi Y, Musson DG, Farmer P, Haller C, Shaywitz AJ, Yang K, O'Neill CA.

Clin Ther. 2015 May 1;37(5):1012-1021.e6. doi: 10.1016/j.clinthera.2014.11.005. Epub 2014 Dec 6.


Differential expression and functionality of ATP-binding cassette transporters in the human hair follicle.

Haslam IS, El-Chami C, Faruqi H, Shahmalak A, O'Neill CA, Paus R.

Br J Dermatol. 2015 Jun;172(6):1562-1572. doi: 10.1111/bjd.13549. Epub 2015 Mar 19.


Nonclinical evaluation of CNS-administered TPP1 enzyme replacement in canine CLN2 neuronal ceroid lipofuscinosis.

Vuillemenot BR, Kennedy D, Cooper JD, Wong AM, Sri S, Doeleman T, Katz ML, Coates JR, Johnson GC, Reed RP, Adams EL, Butt MT, Musson DG, Henshaw J, Keve S, Cahayag R, Tsuruda LS, O'Neill CA.

Mol Genet Metab. 2015 Feb;114(2):281-93. doi: 10.1016/j.ymgme.2014.09.004. Epub 2014 Sep 16.


Pharmacokinetic and pharmacodynamic evaluation of elosulfase alfa, an enzyme replacement therapy in patients with Morquio A syndrome.

Qi Y, Musson DG, Schweighardt B, Tompkins T, Jesaitis L, Shaywitz AJ, Yang K, O'Neill CA.

Clin Pharmacokinet. 2014 Dec;53(12):1137-47. doi: 10.1007/s40262-014-0173-y.


Lactobacillus rhamnosus GG inhibits the toxic effects of Staphylococcus aureus on epidermal keratinocytes.

Mohammedsaeed W, McBain AJ, Cruickshank SM, O'Neill CA.

Appl Environ Microbiol. 2014 Sep;80(18):5773-81. doi: 10.1128/AEM.00861-14. Epub 2014 Jul 11.


Influence of podiatry on orthopedic surgery at a level I trauma center.

Jakoi AM, Old AB, O'Neill CA, Stein BE, Stander EP, Rosenblatt J, Herman MJ.

Orthopedics. 2014 Jun;37(6):e571-5. doi: 10.3928/01477447-20140528-58.


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.


Role of organic osmolytes in water homoeostasis in skin.

El-Chami C, Haslam IS, Steward MC, O'Neill CA.

Exp Dermatol. 2014 Aug;23(8):534-7. doi: 10.1111/exd.12473.


Enzyme replacement therapy attenuates disease progression in a canine model of late-infantile neuronal ceroid lipofuscinosis (CLN2 disease).

Katz ML, Coates JR, Sibigtroth CM, Taylor JD, Carpentier M, Young WM, Wininger FA, Kennedy D, Vuillemenot BR, O'Neill CA.

J Neurosci Res. 2014 Nov;92(11):1591-8. doi: 10.1002/jnr.23423. Epub 2014 Jun 17.


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.


Strain-dependent augmentation of tight-junction barrier function in human primary epidermal keratinocytes by Lactobacillus and Bifidobacterium lysates.

Sultana R, McBain AJ, O'Neill CA.

Appl Environ Microbiol. 2013 Aug;79(16):4887-94. doi: 10.1128/AEM.00982-13. Epub 2013 Jun 14.


Rab3Gap1 mediates exocytosis of Claudin-1 and tight junction formation during epidermal barrier acquisition.

Youssef G, Gerner L, Naeem AS, Ralph O, Ono M, O'Neill CA, O'Shaughnessy RF.

Dev Biol. 2013 Aug 15;380(2):274-85. doi: 10.1016/j.ydbio.2013.04.034. Epub 2013 May 16.


Corneal inflammation is inhibited by the LFA-1 antagonist, lifitegrast (SAR 1118).

Sun Y, Zhang R, Gadek TR, O'Neill CA, Pearlman E.

J Ocul Pharmacol Ther. 2013 May;29(4):395-402. doi: 10.1089/jop.2012.0102. Epub 2012 Dec 5.


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.


Lactobacillus reuteri protects epidermal keratinocytes from Staphylococcus aureus-induced cell death by competitive exclusion.

Prince T, McBain AJ, O'Neill CA.

Appl Environ Microbiol. 2012 Aug;78(15):5119-26. doi: 10.1128/AEM.00595-12. Epub 2012 May 11.


Intrathecal recombinant human 4-sulfatase reduces accumulation of glycosaminoglycans in dura of mucopolysaccharidosis VI cats.

Auclair D, Finnie J, Walkley SU, White J, Nielsen T, Fuller M, Cheng A, O'Neill CA, Hopwood JJ.

Pediatr Res. 2012 Jan;71(1):39-45. doi: 10.1038/pr.2011.13.


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.


Bi-modal dose-dependent cardiac response to tetrahydrobiopterin in pressure-overload induced hypertrophy and heart failure.

Moens AL, Ketner EA, Takimoto E, Schmidt TS, O'Neill CA, Wolin MS, Alp NJ, Channon KM, Kass DA.

J Mol Cell Cardiol. 2011 Oct;51(4):564-9. doi: 10.1016/j.yjmcc.2011.05.017. Epub 2011 May 30.


Biodistribution and pharmacodynamics of recombinant human alpha-L-iduronidase (rhIDU) in mucopolysaccharidosis type I-affected cats following multiple intrathecal administrations.

Vite CH, Wang P, Patel RT, Walton RM, Walkley SU, Sellers RS, Ellinwood NM, Cheng AS, White JT, O'Neill CA, Haskins M.

Mol Genet Metab. 2011 Jul;103(3):268-74. doi: 10.1016/j.ymgme.2011.03.011. Epub 2011 Mar 21.


The pharmacologic assessment of a novel lymphocyte function-associated antigen-1 antagonist (SAR 1118) for the treatment of keratoconjunctivitis sicca in dogs.

Murphy CJ, Bentley E, Miller PE, McIntyre K, Leatherberry G, Dubielzig R, Giuliano E, Moore CP, Phillips TE, Smith PB, Prescott E, Miller JM, Thomas P, Scagliotti R, Esson D, Gadek T, O'Neill CA.

Invest Ophthalmol Vis Sci. 2011 May 16;52(6):3174-80. doi: 10.1167/iovs.09-5078.


Tight junction proteins and the epidermis.

O'Neill CA, Garrod D.

Exp Dermatol. 2011 Feb;20(2):88-91. doi: 10.1111/j.1600-0625.2010.01206.x.


Delivery of SAR 1118 to the retina via ophthalmic drops and its effectiveness in a rat streptozotocin (STZ) model of diabetic retinopathy (DR).

Rao VR, Prescott E, Shelke NB, Trivedi R, Thomas P, Struble C, Gadek T, O'Neill CA, Kompella UB.

Invest Ophthalmol Vis Sci. 2010 Oct;51(10):5198-204. doi: 10.1167/iovs.09-5144. Epub 2010 May 5.


Tetrahydrobiopterin supplementation reduces atherosclerosis and vascular inflammation in apolipoprotein E-knockout mice.

Schmidt TS, McNeill E, Douglas G, Crabtree MJ, Hale AB, Khoo J, O'Neill CA, Cheng A, Channon KM, Alp NJ.

Clin Sci (Lond). 2010 May 6;119(3):131-42. doi: 10.1042/CS20090559.


Safety and pharmacokinetics of a novel lymphocyte function-associated antigen-1 antagonist ophthalmic solution (SAR 1118) in healthy adults.

Semba CP, Swearingen D, Smith VL, Newman MS, O'Neill CA, Burnier JP, Haughey DB, Gadek TR.

J Ocul Pharmacol Ther. 2011 Feb;27(1):99-104. doi: 10.1089/jop.2009.0105. Epub 2010 Mar 24.


Repeated intrathecal injections of recombinant human 4-sulphatase remove dural storage in mature mucopolysaccharidosis VI cats primed with a short-course tolerisation regimen.

Auclair D, Finnie J, White J, Nielsen T, Fuller M, Kakkis E, Cheng A, O'Neill CA, Hopwood JJ.

Mol Genet Metab. 2010 Feb;99(2):132-41. doi: 10.1016/j.ymgme.2009.10.002. Epub 2009 Oct 13.


The mycotoxin patulin, modulates tight junctions in caco-2 cells.

McLaughlin J, Lambert D, Padfield PJ, Burt JP, O'Neill CA.

Toxicol In Vitro. 2009 Feb;23(1):83-9. doi: 10.1016/j.tiv.2008.10.009. Epub 2008 Oct 29.


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