The ALS Center for Cell Therapy and Regeneration Research at Johns Hopkins is committed to iden...tifying the causes of the neurodegenerative disease, amyotrophic lateral sclerosis (ALS), and discovering new and effective treatment options. At the ALS Center, Johns Hopkins researchers work with other investigators, including those at the Robert Packard Center for ALS Research at Johns Hopkins and clinicians within the Johns Hopkins ALS Clinic to aggressively take groundbreaking scientific discoveries and turn them into clinical applications that will improve the quality of life of those diagnosed with ALS. view more

The Charles Wiener Lab primarily conducts research on pulmonary circulation and hypoxia as well... as respiratory muscle function in patients with neuromuscular diseases. Our recent studies have included investigating the treatment of pericardial effusions in patients with pulmonary arterial hypertension and examining the use of non-invasive ventilation in patients with amyotrophic lateral sclerosis (ALS). We also have an interest in medical education research. Our work in this area has included reviewing the role of academic medical centers in emerging health care markets. view more

The Bergles Laboratory studies synaptic physiology, with an emphasis on glutamate transporters ...and glial involvement in neuronal signaling. We are interested in understanding the mechanisms by which neurons and glial cells interact to support normal communication in the nervous system. The lab studies glutamate transport physiology and function. Because glutamate transporters play a critical role in glutamate homeostasis, understanding the transporters' function is relevant to numerous neurological ailments, including stroke, epilepsy, and neurodegenerative diseases like amyotrophic lateral sclerosis (ALS). Other research in the laboratory focuses on signaling between neurons and glial cells at synapses. Understanding how neurons and cells communicate, may lead to new approaches for stimulating re-myelination following injury or disease. Additional research in the lab examines how a unique form of glia-to-neuron signaling in the cochlea influences auditory system development, whether defects in cell communication lead to certain hereditary forms of hearing impairment, and if similar mechanisms are related to sound-induced tinnitus. view more

The Philip Wong Lab seeks to understand the molecular mechanisms and identification of new ther...apeutic targets of neurodegenerative diseases, particularly Alzheimer's disease (AD) and amyotrophic lateral sclerosis (ALS). Taking advantage of discoveries of genes linked to these diseases (mutant APP and PS in familial AD and mutant SOD1, dynactin p150glued ALS4and ALS2 in familial ALS), our laboratory is taking a molecular/cellular approach, including transgenic, gene targeting and RNAi strategies in mice, to develop models that facilitate our understanding of pathogenesis of disease and the identification and validation of novel targets for mechanism-based therapeutics. Significantly, these mouse models are instrumental for study of disease mechanisms, as well as for design and testing of therapeutic strategies for AD and ALS. view more

The nervous system has extremely complex RNA processing regulation. Dysfunction of RNA metaboli...sm has emerged to play crucial roles in multiple neurological diseases. Mutations and pathologies of several RNA-binding proteins are found to be associated with neurodegeneration in both amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). An alternative RNA-mediated toxicity arises from microsatellite repeat instability in the human genome. The expanded repeat-containing RNAs could potentially induce neuron toxicity by disrupting protein and RNA homeostasis through various mechanisms.

The Sun Lab is interested in deciphering the RNA processing pathways altered by the ALS-causative mutants to uncover the mechanisms of toxicity and molecular basis of cell type-selective vulnerability. Another major focus of the group is to identify small molecule and genetic inhibitors of neuron toxic factors using various high-throughput screening platforms. Finally, we are also highly interested in developing novel CRISPR technique-based therapeutic strategies. We seek to translate the mechanistic findings at molecular level to therapeutic target development to advance treatment options against neurodegenerative diseases. view more