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US61561440B   [0102] 

Nature Biotechnology   [0032] 
Nature Biotechnology   [0032] 
Cancer Gene Ther.   [0054] 
Diabetes   [0054] 
Pharm. Research   [0054] 
PNAS   [0054] 
Adv. Appl. Math   [0065] 
J. Mol. Biol.   [0065] 
Proc. Natl. Acad. Sci. USA   [0065] 
J. Mol. Biol.   [0065] 
Proc. Natl. Acad. Sci. USA   [0065] 
Current Protocols in Molecular Biology   [0066] 
Laboratory Techniques in Biochemistry and Molecular Biology -- Hybridization with Nucleic Acid Probes   [0066] 
GAA repeat expansion mutation mouse models of Friedreich ataxia exhibit oxidative stress leading to progressive neuronal and cardiac pathology   [0101] 
Advancements in the pathophysiology of Friedreich's Ataxia and new prospects for treatments   [0101] 
Atypical Friedreich ataxia caused by compound heterozygosity for a novel missense mutation and the GAA triplet-repeat expansion   [0101] 
Xanthomonas AvrBs3 family-type III effectors: discovery and function   [0101] 
Breaking the code of DNA binding specificity of TAL-type III effectors   [0101] 
Two new complete genome sequences offer insight into host and tissue specificity of plant pathogenic Xanthomonas spp   [0101] 
The first cellular models based on frataxin missense mutations that reproduce spontaneously the defects associated with Friedreich ataxia   [0101] 
Friedreich's ataxia: autosomal recessive disease caused by an intronic GAA triplet repeat expansion   [0101] 
Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting   [0101] 
The GAA triplet-repeat is unstable in the context of the human FXN locus and displays age-dependent expansions in cerebellum and DRG in a transgenic mouse model   [0101] 
A pool of extramitochondrial frataxin that promotes cell survival   [0101] 
Friedreich's Ataxia: disease mechanisms, antioxidant and Coenzyme Q10 therapy   [0101] 
Gene expression profiling in frataxin deficient mice: microarray evidence for significant expression changes without detectable neurodegeneration   [0101] 
Functional genomic analysis of frataxin deficiency reveals tissue-specific alterations and identifies the PPARgamma pathway as a therapeutic target in Friedreich's ataxia   [0101] 
Friedreich's ataxia: point mutations and clinical presentation of compound heterozygotes   [0101] 
Frataxin deficiency causes upregulation of mitochondrial Lon and CIpP proteases and severe loss of mitochondrial Fe-S proteins   [0101] 
Rational selection of small molecules that increase transcription through the GAA repeats found in Friedreich's ataxia   [0101] 
Friedreich's ataxia: a clinical and genetic study of 90 families with an analysis of early diagnostic criteria and intrafamilial clustering of clinical features   [0101] 
Expression of Human frataxin is regulated by transcription factors SRF and TFAP2   [0101] 
Friedreich ataxia: effects of genetic understanding on clinical evaluation and therapy   [0101] 
Frataxin point mutations in two patients with Friedreich's ataxia and unusual clinical features   [0101] 
Frataxin knockin mouse   [0101] 
Frataxin overexpressing mice   [0101] 
A simple cipher governs DNA recognition by TAL effectors   [0101] 
Molecular pathogenesis of Friedreich ataxia   [0101] 
Mouse models for Friedreich ataxia exhibit cardiomyopathy, sensory nerve defect and Fe-S enzyme deficiency followed by intramitochondrial iron deposits   [0101] 
Frataxin deficiency in pancreatic islets causes diabetes due to loss of beta cell mass   [0101] 
Evaluation of an FRDA-EGFP genomic reporter assay in transgenic mice   [0101] 
Upregulation of expression from the FRDA genomic locus for the therapy of Friedreich ataxia   [0101] 
Frataxin deficiency alters heme pathway transcripts and decreases mitochondrial heme metabolites in mammalian cells   [0101] 
Electroconvulsive therapy and Friedreich's ataxia   [0101] 
Colonization of rice leaf blades by an African strain of Xanthomonas oryzae pv. oryzae depends on a new TAL effector that induces the rice nodulin-3 Os11 N3 gene   [0101] 
Efficient construction of sequence-specific TAL effectors for modulating mammalian transcription   [0101]