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Robert H Brown

Title: A Method to Quantify Net Axonal Transport in Motor Nerves. UMMS15-65; Patent Pending.

This invention provides methods of diagnosing motor neuron pathologies either before or after treatment by administering to the subject a radiolabeled agent comprising tetanus toxic C fragment and assessing both transport across the neuromuscular junction and retrograde transport. This novel biomarker system can greatly facilitate the study and treatment of diseases such as ALS.
- Technology: Diagnostics
- Applications: Neurology, Neurodegenerative disorders, Amyolateral Sclerosis (ALS), Nerve injury

Title: Modified Mullerian Inhibiting Substance (MIS) Proteins and Uses Thereof for the Treatment of Diseases. UMMS14-56; Patent Pending.

A new recombinant human MIS protein with improved effectivity may improve the treatment of cancer or neurodegenerative disease. This technology maximizes bioactivity using a leader sequence and a modified cleavage site that promotes increased product yield and MIS processing. This recombinant MIS protein also includes a tag to facilitate purification for research and therapeutic purposes.
- Technology: Gene Therapy
- Applications: Neurodegenerative disorders, AAV, Cancer

Title: Antisense Oligonucleotides to Restore Dysferlin mRNA and Protein Expression in Dysferlin-deficient Cells. UMMS14-41; Patent Pending.

This technology originates from identification of a deep intronic mutation in Dyferlin (DYSF) that alters mRNA splicing to include a mutant peptide fragment within a key DYSF domain. This new antisense oligo nucleotide(AON-)mediated exon-skipping to restore production of normal, full-length DYSF in patients' cells in vitro, offering therapeutic solution for patient with Dyferlin-associated maladies.
- Technology: Gene Therapy
- Applications: Dysferlin, Muscular dystrophy, Myopathy, Dysferlinopathy, Antisense oligonucleotide (AON)

Title: MicroRNA Mediated Knockdown of SOD1 Using Recombinant Adeno-associated Vectors. UMMS13-19; Patent Pending.

This invention discloses a novel miRNA delivered by rAAV for silencing SOD1 & C9orf72 SODl genes, which are associated with ALS. This method enables effective therapy at low doses with the persistence of rAAV episomes that continually expresses the nucleic acids, thus rendering re-treatment unnecessary. This method also minimizes rAAV exposure to non-CNS peripheral tissue.
- Technology: Gene Therapy, RNAi/micro-RNA Therapy
- Applications: AAV, Amyolateral Sclerosis (ALS), Neuronal SOD1

Title: METHOD OF DETECTING AMYOTROPHIC LATERAL SCLEROSIS (ALS). UMMS13-11; Patent 8,753,818

This invention is directed to a diagnostic test for identifying individuals with amyotrophic lateral sclerosis (ALS) or individuals who are at risk of developing ALS. This test is based on detecting one or more alterations in a profilin 1 (PFN1) sequence of an individual.

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