• 17097 Citations
1993 …2022
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Personal profile

Research Interests

The overarching goal of our scientific work has been to define key molecular pathways in the pathogenesis of neurodegeneration and to uncover novel targets for therapeutic development. We have focused on pathogenic mechanisms that are commonly altered in neurodegenerative disorders such as deficient degradation of aggregation-prone proteins and mitochondrial dysfunction. As a general strategy, we study rare genetic diseases with mutations in genes that play a role in these key mechanisms and pathways. Models of Huntington’s, Parkinson’s and Gaucher’s disease have been utilized to examine if activation of cellular degradation pathways can lead to neuroprotection. To validate and study these findings in human neurons, we utilize induced pluripotent stem cells (iPS) generated by reprogramming of patient-specific skin fibroblasts. These iPS cells are differentiated into specific neuronal subtypes in order to characterize the interplay of genetic, epigenetic and environmental factors in disease pathogenesis. The ultimate goal of these studies is to develop targeted therapies for Parkinson’s and related neurodegenerative disorders.

Certifications and Licenses

Neurology

Training Experience

2000Residency, Massachusetts General Hospital and Brigham and Women’s Hospital, Harvard Medical School
2002Fellowship, Massachusetts General Hospital, Harvard Medical School

Education/Academic qualification

MD, University of Zagreb

… → 1992

Keywords

  • Aging
  • Alzheimer's Disease
  • Gene Regulation
  • Movement Disorders
  • Neurogenetics
  • Neurology
  • Neuroscience
  • Parkinson’s Disease

Fingerprint Fingerprint is based on mining the text of the experts' scientific documents to create an index of weighted terms, which defines the key subjects of each individual researcher.

  • 23 Similar Profiles
Synucleins Medicine & Life Sciences
Parkinson Disease Medicine & Life Sciences
Huntington Disease Medicine & Life Sciences
Glucosylceramidase Medicine & Life Sciences
Neurodegenerative Diseases Medicine & Life Sciences
Neurons Medicine & Life Sciences
Induced Pluripotent Stem Cells Medicine & Life Sciences
Mutation Medicine & Life Sciences

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Grants 2013 2022

Synucleins
Parkinson Disease
Genotype
Phenotype
Glucosylceramidase
Parkinson Disease
Neurons
Mitochondrial DNA
Clustered Regularly Interspaced Short Palindromic Repeats
Cell Aging
Frontotemporal Lobar Degeneration
Cathepsin D
Mutation
Neurons
Neuroglia
Glucosylceramidase
Parkinson Disease
Lewy Body Disease
Gaucher Disease
Mesencephalon

Research Output 1993 2019

Conversion of Quinazoline Modulators from Inhibitors to Activators of β-Glucocerebrosidase

Zheng, J., Jeon, S., Jiang, W., Burbulla, L., Ysselstein, D., Oevel, K., Krainc, D. & Silverman, R. B., Feb 14 2019, In : Journal of Medicinal Chemistry. 62, 3, p. 1218-1230 13 p.

Research output: Contribution to journalArticle

Glucosylceramidase
Quinazolines
Parkinson Disease
Mutant Proteins
Structure-Activity Relationship
Glucosylceramidase
Quinazolines

Corrigendum: Iron overload is accompanied by mitochondrial and lysosomal dysfunction in WDR45 mutant cells (Brain (2018) 141 (3052-3064) DOI: 10.1093/brain/awy230)

Seibler, P., Burbulla, L., Dulovic, M., Zittel, S., Heine, J., Schmidt, T., Rudolph, F., Westenberger, A., Rakovic, A., Munchau, A., Krainc, D. & Klein, C., Mar 1 2019, In : Brain. 142, 3, p. E10

Research output: Contribution to journalComment/debate

Open Access
Iron Overload
Osteogenesis Imperfecta
Ferritins
Brain
Data Display
1 Citation (Scopus)

Emerging links between pediatric lysosomal storage diseases and adult parkinsonism

Ysselstein, D., Shulman, J. M. & Krainc, D., Jan 1 2019, In : Movement Disorders.

Research output: Contribution to journalReview article

Lysosomal Storage Diseases
Movement Disorders
Parkinsonian Disorders
Glucosylceramidase
Pediatrics

Fluctuations in cell density alter protein markers of multiple cellular compartments, confounding experimental outcomes

Trajkovic, K., Valdez, C., Ysselstein, D. & Krainc, D., Feb 1 2019, In : PloS one. 14, 2, e0211727.

Research output: Contribution to journalArticle

Cell Count
Cells
Focal Adhesion Protein-Tyrosine Kinases
Proteins
Cell culture