TS-002095 — About 3 of every 100,000 births are affected by Batten disease, a disorder that prevents the body from removing cellular waste like lipids and proteins. The build up of cellular waste throughout the body can cause seizures, vision loss, delays in thinking, abnormal movements and death. Researchers at Nationwide Children’s Hospital created a new intrathecal gene therapy using Adeno-Associated viral vectors for Batten Disease caused by mutations in the CLN8 gene. They generated an Adeno-Associated viral vector serotype 9 (AAV9) that contains the human CLN8 cDNA driven by a promoter.

TS-000208 — Ninety percent of cases of amyotrophic lateral sclerosis (ALS) are sporadic and lack a familial association, but the etiology of sporadic ALS remains largely unknown. The laboratory of Dr. Brian Kaspar at Nationwide Children’s Hospital has discovered that overexpression of the HLA-F MHC class I molecule in motor neurons is protective against ALS. Further, they have identified a pharmaceutical composition that increases the expression of HLA-F in motor neurons and would serve as a treatment option for patients with both sporadic and familial ALS.

TS-000170 — Nuclear Factor-kappa B (NF-κB) is a master regulator of inflammation and is upregulated in the spinal cord of ALS patients and in ALS mice models. Researchers at Nationwide Children’s Hospital have demonstrated that NF-κB inhibition in ALS microglia rescued motor neurons (MNs) from microglia-mediated death in vitro and extended survival in ALS mice by impairing pro-inflammatory microglial activation. This work for the first time provides a cellular and molecular mechanism by which microglia induce motor neuron death in ALS and suggests a new therapeutic target to modulate microglial activation and slow the progression of ALS and other neurodegenerative diseases in which microglial activation plays a role. The USPTO has issued a patent for this application in May, 2016

TS-000149 — ALS, commonly referred to as Lou Gehrig’s disease, is characterized by selective, premature degeneration and death of motor neurons (MNs) in the motor cortex, brain stem and spinal cord. Studies have demonstrated that not only MNs but also non-neuronal cell types including microglia and astrocytes play a significant role in disease onset and progression. Researchers at Nationwide Children’s Hospital have identified a previously undescribed disease mechanism in which astrocytes use killing pathways typically ascribed to the innate immune system, and can use these mechanisms as targets for therapeutic intervention in ALS.