OnPoint Special Issues

Spinal Muscular Atrophy (SMA)

Table of Content (DRAFT)

Summary.. 2

TECHNOLOGY TRANSFER.. 3

1.         Therapeutic potentiation of neurotransmission by a synaptic chaperone variant to treat neurodegeneration including spinal muscular atrophy, Umrao R. Monani, Columbia University Medical Center (CUMC), New York, United States. 3

2.         Therapeutic targets for Spinal Muscular Atrophy and Amyotrophic Lateral Sclerosis, Christopher E. Henderson, Columbia University Medical Center (CUMC), New York, United States  3

3.         Modulated protein for motor neuron disease treatment, Umrao R. Monani, Columbia University Medical Center (CUMC), New York, United States. 4

4.         Engineered neuron-specific protein inhibits uncontrolled cell death to combat neurodegeneration, Carol Troy, Columbia University Medical Center (CUMC), New York, United States  4

5.         Antisense Morpholino Treatment to Increase SMN Levels and Treat Spinal Muscular Atrophy, Arthur Burghes, Ohio, Ohio State University. 5

6.         Antibody for a Human Spinal Musculary Atrophy Protein, Chris Lorson, University of Missouri, Missouri, USA.. 6

7.         Predicting Treatment Response in Patients with Spinal Muscular Atrophy (SMA), William Hu, Sumit Verma, Emory University, Georgia, USA.. 6

8.         Aza-Peptide Epoxides for Treating Neurological Diseases, Jonathan Glass, James Powers , Emory University, Georgia, USA.. 7

9.         Rapid generation of human forebrain, midbrain, and hindbrain cells from human pluripotent stem cells, Kyle Loh, Standord University, California, USA.. 8

SPONSORED PROJECTS. 8

1.         SARM1 antagonism as adjunct therapy in SMA, Charlotte Sumner, Johns Hopkins University School of Medicine, Maryland, United States. 8

2.         Restoration of motor neuron function via potassium channels in SMA mice, Nandhini Sivakumar, Columbia University Medical Center, New York, United States  9

3.         Analysis of Brain-derived Neurotrophic Factor Signaling in Spinal Muscular Atrophy, Michael A. Sendtner, University Hospital Würzburg, Würzburg, Germany  9

4.         Biogenesis and degradation of miRNAs in spinal muscular atrophy, Brunhilde Wirth, University Hospital Cologne, Cologne, Germany. 10

5.         Therapeutic Gene Editing for Spinal Muscular Atrophy (Trailblazer), Mandana Arbab, Jackson Labs, Maine, United States. 10

6.         Precision Base Editing for the Treatment of Motor Neuron Diseases, Mandana Arbab, Boston Children’s Hospital, Massachusetts, United States. 11

7.         Search for SMN-independent treatments for spinal muscular atrophy focusing on BMP signaling, Hideki Yoshida, Kyoto University of Medicine, Kyoto, Japan. 11

8.         Elucidating the pathology and developing treatments for spinal muscular atrophy by applying the dynamics of skeletal muscle development in iPS cells, Shiori Ando, Gifu Pharmaceutical University, Gifu, Japan. 12

STARTUPS.. 13

VC Funding.. 14

News. 14

Study Unveils Early Developmental Defects in Spinal Muscular Atrophy Using Patient-Derived Organoids. 14

Novel GLP-1 receptor agonists (Coming)

TBA

Gaucher's Disease (Coming)

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