GBA-associated PD: chances and obstacles for targeted treatment strategies.; ; et al in Journal of neural transmission (Vienna, Austria : 1996) (2022), 129(9), 1219-1233 Given the clear role of GBA in the pathogenesis of Parkinson's disease (PD) and its impact on phenotypical characteristics, this review provides an overview of the current knowledge of GBA-associated PD ... [more ▼] Given the clear role of GBA in the pathogenesis of Parkinson's disease (PD) and its impact on phenotypical characteristics, this review provides an overview of the current knowledge of GBA-associated PD with a special focus on clinical trajectories and the underlying pathological mechanisms. Importantly, differences and characteristics based on mutation severity are recognized, and current as well as potential future treatment options are discussed. These findings will inform future strategies for patient stratification and cohort enrichment as well as suitable outcome measures when designing clinical trials. [less ▲] Detailed reference viewed: 135 (5 UL) Grundlagen und Stellenwert der COMT- und MAO-B-Inhibitoren in der Therapie des idiopathischen Parkinson-Syndroms.; Krüger, Rejko ; et alin Fortschritte der Neurologie-Psychiatrie (2020) Inhibitors of COMT and MAO-B are well established in the pharmacotherapy of Parkinson's disease (PD). MAO-B inhibitors are used as monotherapy as well as in combination with levodopa, whereas COMT ... [more ▼] Inhibitors of COMT and MAO-B are well established in the pharmacotherapy of Parkinson's disease (PD). MAO-B inhibitors are used as monotherapy as well as in combination with levodopa, whereas COMT inhibitors exert their effects only in conjungtion with levodopa. Both classes of compounds prolong the response duration of levodopa and optimise its clinical benefit. As a result, the ON-times are prolonged significantly. In the past, MAO-B inhibitors were also adminstered for neuroprotection; however, despite convincing scientific reasoning in support of neuroprotective effects, these could not be substantiated in clinical studies performed so far. [less ▲] Detailed reference viewed: 213 (5 UL) Direct reprogramming of fibroblasts into neural stem cells by defined factors.; ; et al in Cell Stem Cell (2012), 10(4), 465-72 Recent studies have shown that defined sets of transcription factors can directly reprogram differentiated somatic cells to a different differentiated cell type without passing through a pluripotent state ... [more ▼] Recent studies have shown that defined sets of transcription factors can directly reprogram differentiated somatic cells to a different differentiated cell type without passing through a pluripotent state, but the restricted proliferative and lineage potential of the resulting cells limits the scope of their potential applications. Here we show that a combination of transcription factors (Brn4/Pou3f4, Sox2, Klf4, c-Myc, plus E47/Tcf3) induces mouse fibroblasts to directly acquire a neural stem cell identity-which we term as induced neural stem cells (iNSCs). Direct reprogramming of fibroblasts into iNSCs is a gradual process in which the donor transcriptional program is silenced over time. iNSCs exhibit cell morphology, gene expression, epigenetic features, differentiation potential, and self-renewing capacity, as well as in vitro and in vivo functionality similar to those of wild-type NSCs. We conclude that differentiated cells can be reprogrammed directly into specific somatic stem cell types by defined sets of specific transcription factors. [less ▲] Detailed reference viewed: 359 (19 UL)![]() New developments in diagnosis and treatment of Parkinson's disease--from basic science to clinical applications.; ; Krüger, Rejko et alin Journal of neurology (2004), 251 Suppl 6 In this review we summarize new developments in early diagnosis, establishing surrogate markers, genetics, neuroprotection and cell replacement in Parkinson's disease. Furthermore, we discuss the major ... [more ▼] In this review we summarize new developments in early diagnosis, establishing surrogate markers, genetics, neuroprotection and cell replacement in Parkinson's disease. Furthermore, we discuss the major problems in the translation of results from preclinical research into successful clinical trials. [less ▲] Detailed reference viewed: 187 (0 UL) |
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