THE CORE

1. In Vivo Unit

1.1. Developing better assays for testing phenotypes in ALS animal models

1.2. Testing Prosetin uptake in animal model spinal cord

1.3. Taking longitudinal plasma samples Prosetin-treated ALS animal models

1.4. Testing viral method for introducing microRNA to prevent protein aggregation in ALS animal models

2. Lipidomics Unit

2.1. Characterizing lipid profiles of plasma from patients presenting distinct clinical progressions

2.2. Evaluating lipid profiles of plasma from Project ALS Families Project in presymptomatic ALS mutation carriers

2.3. Evaluating lipid distributions and levels in spinal cords of ALS animal models

3. Antibody Unit

3.1. Developing mutation-specific antibodies against commonly defective proteins in ALS

3.2. Checking localization of healthy version versus mutated version of protein in ALS animal models

3.3. Creating antibodies that can target and block the function of proteins known to aggregate in ALS motor neurons or cause neuroinflammation in ALS

4. In Vitro Unit

4.1. Performing screen on stem cell-derived motor neurons to determine which proteins contribute to motor neuron degeneration

4.2. Testing specific protein inhibitors that act on proteins known to be involved in ALS in motor neuron culture

4.3. Developing "aged" motor neuron cultures to better recapitulate the state of motor neuron in ALS patients

4.4. Testing Prosetin in "aged" motor neuron cultures

4.5. Developing cultures with more cell diversity to more accurately predict neuroimmune interactions that occur in patients

4.6. Applying cerebrospinal fluid samples from ALS patients to test if circulating molecules promote neurodegeneration

4.7. Identify human chaperone proteins that prevent protein aggregation and neurodegeneration in yeast model

5. Viral Unit

5.1. Generating novel viral vectors for delivery of microRNA against frequently mutated human gene in ALS

5.2. Testing transcriptional reprogramming approach for preventing ALS mechanism in spinal motor neurons

5.3. Developing novel methods for testing necessary dose and efficacy of viral delivery in in vitro systems to expedite tests

6. Electrophysiology Unit

6.1. Characterizing nerve-to-muscle output in ALS animal models

6.2. Evaluating motor neuron loss and motor neuron-to-muscle denervation in ALS in relationship to muscle function

6.3. Developing EMG-based fatigue test to determine what muscle function looks like in presymptomatic ALS animal models

7. Clinical Unit

7.1. Conducting multiple clinical trials of distinct therapy types, including Jacifusen and Prosetin, among others

7.2. Collecting samples for better diagnostics across distinct ALS presentations