Bibliography |
Nephropathic Cystinosis (RP103)
Cystagon Package Insert Accessed Feb 22, 2011.
Dohil R, Fidler M, Barshop BA, Martin M, Gangoiti J, Deutsch R, Schneider JA. Understanding intestinal cysteamine bitartrate absorption. J Pediatr. 2006;148:764-769.
Dohil R, Fidler M, Gangoiti JA, Kaskel F, Schneider JA, Barshop BA. Twice-daily cysteamine bitartrate therapy for children with cystinosis. J Pediatr. 2010 Jan;156(1):71-75.e1-3.
Gahl WA, Balog JZ, Kleta R. Nephropathic cystinosis in adults: natural history and effects of oral cysteamine therapy. Ann Intern Med. 2007;147:242-250.
Kleta R, Bernardini IM, Ueda M, Varade WS, Phornphutkul C, Krasnewich D, Gahl WA. Long-term follow-up of well-treated nephropathic cystinosis patients. J Pediatr. 2004; 145:555-60.
Kleta R, Gahl WA. Pharmacological treatment of nephropathic cystinosis with cysteamine. Expert Opin. Pharmacother. 2004;5(11):2255-2262.
Levtchenko EN, van Dael CM, de Graaf-Hess AC, Wilmer MJ, van den Heuvel LP, Monnens LA, Blom HJ. Strict cysteamine dose regimen is required to prevent nocturnal cystine accumulation in cystinosis. Pediatr Nephrol. 2006;21:110–113.
Markello TC, Bernardini IM, Gahl WA. Improved renal function in children with cystinosis treated with cysteamine. N Engl J Med. 1993;328:1157-1162.
Non-alcoholic Steatohepatitis-NASH (RP104)
Contos MJ, Sanyal AJ. The clinicopathologic spectrum and management of nonalcoholic fatty liver disease. Adv Anat Pathol. 2002;9(1):37-51.
Franzese A, Vajro P, Argenziano A, Puzziello A, Iannucci MP, Saviano MC, et al. Liver involvement in obese children. Ultrasonography and liver enzyme levels at diagnosis and during follow-up in an Italian population. Dig Dis Sci. 1997;42(7):1428-32.
Hashimoto E, Tokushige K. Prevalence, gender, ethnic variations, and prognosis of NASH. J Gastroenterol. 2011 Jan;46 Suppl 1:63-69.
Ko JS, Yoon JM, Yang HR, Myung JK, Kim HR, Kang GH, Cheon JE, Seo JK. Clinical and histological features of nonalcoholic fatty liver disease in children. Dig Dis Sci. 2009 54:2225-2230.
Lavine JE. Vitamin E treatment of nonalcoholic steatohepatitis in children: a pilot study. J Pediatr. 2000;136(6):734-8.
Loomba R, Sirlin CB, Schwimmer JB, Lavine JE. Advances in pediatric nonalcoholic fatty liver disease. Hepatology. 2009;50:1282-1293.
National Digestive Diseases Information Clearinghouse. Nonalcoholic Steatohepatitis. Accessed October 14, 2008.
Papandreou D, Rousso I, Mavromichalis I. Update on non-alcoholic fatty liver disease in children. Clin Nutr. 2007;26:409-415.
Prescott LF, Critchley JA. The treatment of acetaminophen poisoning. Annu Rev Pharmacol Toxicol. 1983;23:87-101.
Strauss R, Barlow SE, Dietz WH. Prevalence of abnormal serum aminotransferase values in overweight and obese adolescents. J Pediatr. 2000;136:727-733.
Targher G, Day CP, Bonora E. Risk of cardiovascular disease in patients with nonalcoholic fatty liver disease. N Engl J Med. 2010;363(14):1341-1350.
Te Sligte K, Bourass I, Sels JP, Driessen A, Stockbrugger RW, Kock GH. Non-alcoholic steatohepatitis: review of a growing problem. Eur J Intern Med. 2004;15:10-21.
Huntington’s Disease (RP103)
Borrell-Pages M, Canals JM, Cordelieres FP, Parker JA, Pineda JR, Grange G, Bryson EA, Guillermier M, Hirsch E, Hantraye P, Cheetham ME, Neri C, Alberch J, Brouillet E, Saudou F, Humbert S. Cysteamine and cysteamine increase in brain levels of BDNF in Huntington disease via HSJ1b and transglutaminase. J Clin Invest. 2006 May;116(5):1410-24.
Dubinsky R, Gray C. CTYE-I-HD: phase I dose finding and tolerability study of cysteamine (Cystagon®) in Huntington’s disease. Mov Disord. 2006 April;21(4):530-3.
Ferrer I, Goutan E, Marin C, Rey MJ, Ribalta T. Brain-derived neurotrophic factor in Huntington disease. Brain Res. 2000;866:257-261.
Pinto JT, Van Raamsdonk JM, Leavitt BR, Hayden MR, Jeitner TM, Thaler HT, Krasnikov BF, Cooper AJ. Treatment of YAC128 mice and their wild-type littermates with cysteamine does not lead to its accumulation in plasma or brain: implications in the treatment of Huntington disease. Neurochem. 2005 Aug;94(4):1087-101.
Shults C, Steardo L, Barone P, Mohr E, Juncos J, Serrati C, Fedio P, Tamminga CA, Chase TN. Huntington's Disease: effect of cysteamine, a somatostatin-depleting agent. Neurology. 1986 Aug;36(8):1099-102.
ALDH2 Deficiency (Convivia™)
Blushing from alcohol signals cancer risk for East Asians. USA Today. March 23, 2009.
Seitz HK, Hoffman N. p;The role of acetaldehyde in alcohol-associated cancer of the gastrointestinal tract. Novartis Foundation Symposium 285 - Acetaldehyde-Related Pathology: Bridging the Trans-Disciplinary Divide. John Wiley & Sons; 2007:110-124.
Hepatocellular Carcinoma and Other Liver Diseases (HepTide™)
Isbell SL, Haslam SB, Dennis SJ, Nash JA, Zankel TC. Functional mimicry of the LDL receptor-associated protein through multimerization of a minimized third domain. Biochem Biophys Res Comm. 2007;364(3):614-619.
Isbell SL, Haslam SB, Zankel TC. Minimization of the third domain of the LDL receptor-associated protein. Biochem Biophys Res Comm. 2007a;361:758-762.
Isbell SL, Haslam SB, Zankel TC. Peptide for Improved Drug Delivery to the Liver. Poster presented at: International Liver Cancer Association (ILCA) conference; November 2007b; Barcelona, Spain.
Blood-Brain Barrier (NeuroTrans™)
Pan W, Kastin AJ, Zankel TC, van Kerkhoff P, Terasaki T, Bu G. Efficient transfer of RAP across the blood-brain barrier. J Cell Sci. 2004;117:5071-5078.
Prince WS, McCormick LM, Wendt DJ, Fitzpatrick PA, Schwartz Aguilera AI, Koppaka V, Christianson TM, Vellard MC, Pavloff N, Lemontt JF, Qin M, Starr CM, Bu G, Zankel TC. Lipoprotein receptor binding, cellular uptake, and lysosomal delivery of fusions between the receptor-associated protein (RAP) and alpha-L-iduronidase or acid alpha-glucosidase. J Biol Chem. 2004 Aug 13;279(33):35037-46.
Breast Cancer Research (WntTide™)
Li Y, Bu G. LRP5/6 in Wnt signaling and tumorigenesis. Future Oncol. 2005 Oct;1(5):673-81.
Li Y, Chen J, Lu W, McCormick LM, Wang J, Bu G. d binds to mature LDL-receptor-related protein-6 and antagonizes ligand binding. J Cell Sci. 2005 Nov 15;118(Pt 22):5305-14.
Liu CC, Prior J, Piwnica-Worms D, Bu G., LRP6 overexpression defines a class of breast cancer subtype and is a target for therapy. Proc Natl Acad Sci U S A. 2010 Mar 16;107(11):5136-41.