Department of Genetics
School of Medicine     .      Case Western Reserve University

Research.

Our lab focus on the investigations of cell autonomous and non-cell autonomous molecular events involved in spinal bulbar muscular atrophy and amyotrophic lateral sclerosis using induced pluripotent stem cell (iPSC) models.

A substantial number of drugs found to alleviate symptoms of motor neuron diseases (MNDs) in animal models have failed in clinical trials. While the reasons for this are not clear, modeling with disease cell types from affected patients may yield novel targets and pathways, and setting the stage for the discovery of drugs targeting MNDs.

New advents in stem cell research currently include the possibility of exploring co-cultures of multiple cell types to study their interactions. Therefore, our lab uses iPSCs developed for SBMA and ALS8 to dissect the contributions of motor neurons, astrocytes and skeletal muscles for motor neuron diseases.

Helen Miranda

graduated with a BS in Biomedicine and an MS in Immunopathology from the State University of Londrina In Brazil. She then went to earn her PhD in Cell and Molecular Biology in the University of Sao Paulo, also in Brazil, under the mentorship of Dr. Lewis Joel Greene. In her graduate work, Helen performed proteomics comparisons of Adult Stem Cells from two different sources (bone marrow and umbilical cord vein). As a graduate student, Helen was one of only 10 Brazilians selected by the renowned stem cell biology experts in the UK for the “Embryonic Stem (ES) Cells as a Model System for Embryonic Development” course.

During this experience, she became fascinated by the use of induced pluripotent stem cells (iPSCs) for modeling human diseases.

For that reason, Helen chose to come to the USA as a joint postdoctoral fellow in the La Spada lab and in the Muotri lab at the University of California San Diego (UCSD). During her postdoctoral training, Helen developed stem cell models for two different motor neuron disorders, spinal bulbar muscular atrophy and amyotrophic lateral sclerosis. She joined the Department of Genetics and Genomes Sciences at Case Western Reserve University as an Assistant Professor in May 2018 to continue her studies on the pathophysiology of motor neuron diseases.

Publications

01

Selective modulation of the androgen receptor AF2 domain rescues degeneration in spinal bulbar muscular atrophy.

02

Adenylyl cyclase activating polypeptide reduces phosphorylation and toxicity of the polyglutamine-expanded androgen receptor in spinobulbar muscular atrophy.

03

PPAR-δ is repressed in Huntington's disease, is required for normal neuronal function and can be targeted therapeutically.

04

Nemo-like kinase is a novel regulator of spinal and bulbar muscular atrophy.

People

  • Helen C. Miranda

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  • Helen C. Miranda

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Open Positions

Nemo-like kinase is a novel regulator of spinal and bulbar muscular atrophy.

Elife (2015);4:e08493

Nemo-like kinase is a novel regulator of spinal and bulbar muscular atrophy.

Elife (2015);4:e08493

Department of Genetics School of Medicine Case Western Reserve University

Biomedical Research Building 719
2109 Adelbert Road
Cleveland, Ohio 44106-4955
Tel: (216) 368-8811
Fax: (216) 368-3432
E-mail: helen.miranda@case.edu