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2. Next-generation phenotyping integrated in a national framework for patients with ultrarare disorders improves genetic diagnostics and yields new molecular findings

5. Single-cell, whole-embryo phenotyping of mammalian developmental disorders

6. Single cell, whole embryo phenotyping of pleiotropic disorders of mammalian development

9. Biallelic loss-of-function variants of ZFTRAF1 cause neurodevelopmental disorder with microcephaly and hypotonia

10. Evaluating the association of biallelic OGDHL variants with significant phenotypic heterogeneity

12. Functional connectivity signatures of NMDAR dysfunction in schizophrenia—integrating findings from imaging genetics and pharmaco-fMRI

15. Aberrant phase separation and nucleolar dysfunction in rare genetic diseases

17. Adult human kidney organoids originate from CD24+ cells and represent an advanced model for adult polycystic kidney disease

18. ADGRL1 haploinsufficiency causes a variable spectrum of neurodevelopmental disorders in humans and alters synaptic activity and behavior in a mouse model

19. Sepsis induces long-lasting impairments in CD4+ T-cell responses despite rapid numerical recovery of T-lymphocyte populations

20. Genetic pain loss disorders

21. GestaltMatcher Database - A global reference for facial phenotypic variability in rare human diseases

23. Early IFN-α signatures and persistent dysfunction are distinguishing features of NK cells in severe COVID-19

27. Genome sequencing in families with congenital limb malformations

28. Balance between macrophage migration inhibitory factor and sCD74 predicts outcome in patients with acute decompensation of cirrhosis

30. Longitudinal Multi-omics Analyses Identify Responses of Megakaryocytes, Erythroid Cells, and Plasmablasts as Hallmarks of Severe COVID-19

31. Severe COVID-19 Is Marked by a Dysregulated Myeloid Cell Compartment

34. GestaltMatcher Database - A global reference for facial phenotypic variability in rare human diseases

36. The difficulty to model Huntington’s disease in vitro using striatal medium spiny neurons differentiated from human induced pluripotent stem cells

39. Noncoding copy-number variations are associated with congenital limb malformation

41. DEGSI-associated aberrant sphingolipid metabolism impairs nervous system function in humans

45. Genetic landscape of congenital insensitivity to pain and hereditary sensory and autonomic neuropathies

46. Closing the Gap - Detection of 5q-Spinal Muscular Atrophy by Short-Read Next-Generation Sequencing and Unexpected Results in a Diagnostic Patient Cohort

47. Hereditary Sensory and Autonomic Neuropathy: A Case Series of Six Children

50. The genetic landscape of axonal neuropathies in the middle-aged and elderly: Focus on MME

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