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The technology presented is a breakthrough in the diagnosis of lymphatic filariasis, specifically targeting the B. malayi pathogen. It encompasses a novel soluble antigen extract used in both IgG and IgG4-based ELISA tests, aimed at detecting the presence of the filarial infection. This innovation serves as a cornerstone for a CLIA-certified reference test, established and utilized in Dr. Nutman's laboratory since the late 1980s.

Summary:

Lung metastases represent a major clinical challenge in advanced cancer, with poor survival rates and no effective therapies to prevent their development. Researchers at the National Cancer Institute (NCI) have developed C24:2, a first-in-class synthetic 3-O-sulfo-galactosylceramide analog. After lysosomal processing by dendritic cells, C24:2 switches immune specificity to activate type I NKT cells, triggering a potent IFN-γ–mediated Th1 response.

Mutations in the cone rod homeobox (CRX) transcription factor lead to distinct retinopathy phenotypes, including early-onset vision impairment in dominant Leber congenital amaurosis (LCA). Adeno-Associated virus (AAV) vector-mediated delivery of a CRX cDNA under the control of a CRX promoter region partially restored photoreceptor phenotype and expression of phototransduction genes in an in vitro model of CRX-LCA.

Glaucoma is one of the world’s leading causes of irreversible blindness. There is no cure and vision lost from glaucoma cannot be restored. Glaucoma is associated with fluid build-up in the eye resulting in an increased intraocular pressure (IOP). The pressure may cause damage to the optic nerve and lead to progressive degeneration of retinal ganglion cells (RGC) and vision loss. Currently, available treatments for glaucoma delay progression by reducing IOP, but no therapies exist to directly protect RGC from degradation and loss. 

X-linked retinoschisis (XLRS) is an inherited, monogenetic ocular disease caused by mutations in the retinoschisin (RS1) gene, resulting in the development of cystic cavities throughout the retina and leading to juvenile macular degeneration. Approximately 1:15,000 males in the US are affected, classifying the condition as an orphan indication. 

X-linked forms of retinitis pigmentosa (XLRP) are relatively severe blinding disorders, resulting from progressive photoreceptor dysfunction primarily caused by mutations in RPGR or RP2 gene.

Degeneration of retinal tissues occurs in many ocular disorders resulting in the loss of vision. Dysfunction and/or loss of Retinal Pigment Epithelium Cells (RPE) and disruption of the associated blood retinal barrier (BRB) tissue structures are linked with many ocular diseases and conditions including: age-related macular degeneration (AMD), Best disease, and retinitis pigmentosa. Engineered tissue structures that are able to replicate the function of lost BRB structures may restore lost vision and provide insight into new treatments and mechanisms of the underlying conditions. 

The retinal pigment epithelium (RPE) is a cell monolayer with specialized functions crucial to maintaining the metabolic environment and chemistry of the sub-retinal and choroidal layers in the eye. Damage or disease causing RPE cell loss leads to progressive photoreceptor damage and impaired vision. Loss of RPE is observed in many of the most prevalent cases of vision loss, including age related macular degeneration (AMD) and Best disease.

Retinal Degenerations (RD) are the leading cause of blindness in the United States. The degeneration of the Retinal Pigment Epithelium (RPE) is associated with various types of RD such as Stargardt’s disease, retinitis pigmentosa, choroideremia, Late-Onset Retinal Degeneration (L-ORD), and Age-related Macular Degeneration (AMD). The RPE as a layer of cells in the back of the eye. Therefore, it is essential to maintain the health and integrity of retinal photoreceptors.