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Clinical advances of RNA therapeutics for treatment of neurological and neuromuscular diseases.
Holm A, Hansen SN, Klitgaard H, Kauppinen S. Holm A, et al. RNA Biol. 2022;19(1):594-608. doi: 10.1080/15476286.2022.2066334. Epub 2021 Dec 31. RNA Biol. 2022. PMID: 35482908 Free PMC article.
Furthermore, RNA-based therapeutics have the potential to modulate entire disease pathways, and thereby represent a new modality with unprecedented potential for generating disease-modifying drugs for a wide variety of human diseases, including central nervou …
Furthermore, RNA-based therapeutics have the potential to modulate entire disease pathways, and thereby represent a new modali …
Strategies for treatment in Alexander disease.
Messing A, LaPash Daniels CM, Hagemann TL. Messing A, et al. Neurotherapeutics. 2010 Oct;7(4):507-15. doi: 10.1016/j.nurt.2010.05.013. Neurotherapeutics. 2010. PMID: 20880512 Free PMC article. Review.
The pathological hallmark of Alexander disease is the inclusion body, known as Rosenthal fiber, within the cell bodies and processes of astrocytes. ...Ideas are beginning to emerge that suggest several compelling therapeutic targets for interventions that mig …
The pathological hallmark of Alexander disease is the inclusion body, known as Rosenthal fiber, within the cell bodies and pro …
Nationwide survey of Alexander disease in Japan and proposed new guidelines for diagnosis.
Yoshida T, Sasaki M, Yoshida M, Namekawa M, Okamoto Y, Tsujino S, Sasayama H, Mizuta I, Nakagawa M; Alexander Disease Study Group in Japan. Yoshida T, et al. J Neurol. 2011 Nov;258(11):1998-2008. doi: 10.1007/s00415-011-6056-3. Epub 2011 May 1. J Neurol. 2011. PMID: 21533827
Alexander disease (AxD) is a rare neurodegenerative disorder characterized by white matter degeneration and formation of cytoplasmic inclusions. ...We gathered information regarding prevalence, neurological findings, magnetic resonance imaging (MRI) findings, electr …
Alexander disease (AxD) is a rare neurodegenerative disorder characterized by white matter degeneration and formation of cytop …
Antisense suppression of glial fibrillary acidic protein as a treatment for Alexander disease.
Hagemann TL, Powers B, Mazur C, Kim A, Wheeler S, Hung G, Swayze E, Messing A. Hagemann TL, et al. Ann Neurol. 2018 Jan;83(1):27-39. doi: 10.1002/ana.25118. Epub 2018 Jan 14. Ann Neurol. 2018. PMID: 29226998 Free PMC article.
Here we investigate whether suppressing GFAP with antisense oligonucleotides could provide a therapeutic strategy for treating Alexander disease. METHODS: In this study, we use GFAP mutant mouse models of Alexander disease to test the efficacy o …
Here we investigate whether suppressing GFAP with antisense oligonucleotides could provide a therapeutic strategy for treating Ale …
Infantile Alexander disease: spectrum of GFAP mutations and genotype-phenotype correlation.
Rodriguez D, Gauthier F, Bertini E, Bugiani M, Brenner M, N'guyen S, Goizet C, Gelot A, Surtees R, Pedespan JM, Hernandorena X, Troncoso M, Uziel G, Messing A, Ponsot G, Pham-Dinh D, Dautigny A, Boespflug-Tanguy O. Rodriguez D, et al. Am J Hum Genet. 2001 Nov;69(5):1134-40. doi: 10.1086/323799. Epub 2001 Sep 20. Am J Hum Genet. 2001. PMID: 11567214 Free PMC article.
Heterozygous, de novo mutations in the glial fibrillary acidic protein (GFAP) gene have recently been reported in 12 patients affected by neuropathologically proved Alexander disease. We searched for GFAP mutations in a series of patients who had heterogeneous clini …
Heterozygous, de novo mutations in the glial fibrillary acidic protein (GFAP) gene have recently been reported in 12 patients affected by ne …