Batten disease

StemCells files IND to begin Batten disease stem cell study. StemCells, Inc. PRESS RELEASE 2005 January 04. 

Neurodegenerative disease The neuronal ceroid lipofuscinoses (Batten disease). 

StemCells, Inc. receives IRB approval from Oregon Health Science University to begin phase I clinical trial in Batten disease first ever clinical trial using purified human neural stem cells. StemCells, Inc. PRESS RELEASE 2006 March 09. 

Rakheja, D. et al. (2004). CLN3P, the Batten disease protein, localizes to membrane lipid rafts (detergent-resistant membranes). Biochem. Biophys. Res. Commun. 317(4), 988-991. 

Pears MR, Cooper JD, Mitchison HM, Mortishire-Smith RJ, Pearce DA, Griffin JL. High resolntion IH NMR-based metabolomics indicates a nenrotransmitter cycling deficit in cerebral tissne from a monse model of Batten disease. J. Biol. Chem. 2005 280 42508-42514. 

Prenatal screening Haemophilia Sickle cell anaemia 3-Thalassaemia Duchenne muscular dystrophy Batten disease Sex determination... 

Pearce DA, Atkinson M, Tagle DA (2004) Glutamic acid decarboxylase autoimmunity in Batten disease and other disorders. Neurology 63 2001-2005. 

Kryndushkin D, Shewmaker F, Wickner R (2008) Curing of the URP3J prion by Btn2p, a Batten disease-related protein. EMBO J 27 2725-2735... 

Already, many microarray studies have been published and validate the use of this expensive technology. Perhaps the best examples include human cancer (13-16) encompassing the monitoring of tumor progression (17,18) and leading to the reclassification of tumors (17,19). Other diseases have also been subject to array analysis such as rheumatoid arthritis (20) and Battens disease (21). 

Bennett, M.J., Hofmann, S.L., 1999. The neuronal ceroid-lipofiiscinoses (Batten disease) a new class of lysosomal storage diseases. J. Inherit. Metab. Dis. 22 (4), 535—544. 

Batten disease Mutations in CLN3 and protein palmitoylthioesterase Rylova et at., 2002... 

The neuronal ceroid lipofuscinoses (CLN), also referred to as Batten s disease, are a group of disorders characterized by the accumulation of autofluorescent lipopigments. Clinical hallmarks include blindness, seizures, cognitive and motor decline and early death. Age of onset varies from infancy to adulthood. Eight genetic forms have been identified [4]. Two involve lysosomal acid hydrolases. CLN1 codes for palmitoyl protein thioesterase 1. Clinically it presents most often in infancy and leads to loss of active movement and visual contact by 3 years of age. It is most common in Finland, where its incidence is 1 20,000. CLN2 codes for a lysosomal pepstatin-insensitive acid protease. 

Volume III, Pharmacology and Therapy, addresses developments in basic science, translational and clinical research that are underway to bring stem cell research to therapy, particularly for the treatment of Batten s diseases, graft-versus-host disease and adenosine deaminase deficiency. This volume covers the importance of stem cell research for the understanding of drug activities and design. It also addresses the ethical issues and constraints involved in stem cell research, and its commercial applications. 

Brain transplantation of human neural stem cells in mouse model of Batten s disease. Basu, S B., Belichenko, P.V., Uchida, N., Tamaki, S., Eckert, K., Udani, V., Tsukamoto, A., Mobley, W.C. (2003). Abstr SocNeurosci, 335.7. 

Reviews - The experimental aspects of the chemotherapy of tuberculosis were reviewed by Batten, clinical aspects were discussed editorially. Browne reviewed the treatment of leprosy. A favorable editorial comment was made on the use of an antimutagen (quinacrlne) with standard treatment of urinary tract Infection to prevent the rise of resistant bacterial strains. Lampe examined the changing concepts in the therapy of urinary tract disease. The not unrelated fields of sulfonamides, and nitro-furans were also reviewed. The resistance development as a consequence of antimicrobial drugs in animal feeds was examined. Topical antimicrobials were reviewed in several contexts antiseptics and disinfectants, soap bacteriostats,deodorants and antlperspirants,and antimicrobial biguanides. The topical treatment of burns and the renaissance of mafenide for such use was discussed by Moncrief. Veterinary ophthalmic antimicrobials were reviewed. 

Dawson G, Cho S. Batten s disease clues to neuronal protein catabolism in lysosomes. J Neurosci Res 2000 60(2) 133... 

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