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The invention relates to a plasmid that enables gene knowndown (via CRISPRi) in human induced pluripotent stem cells (iPSCs), including derived cell types such as neurons. The plasmid contains homology arms to direct insertion of a cassette into the CLYBL safe-harbor locus in the human genome. The cassette expresses CRISPRi machinery using a CAG promoter. The CRISPRi machinery consists of double degron-tagged dCas9-BFP-KRAB. Addition of the small molecule trimethoprim to cell culture media stabilizes the degron and thereby increases dCas9-BFPKRAB levels, enabling CRISPRi activity.

The technology relates to the identification and validation of eight biomarkers for active central nervous system (CNS) intrathecal inflammation. The management of neuroimmunological diseases is severely hindered by an inability to reliably measure intrathecal inflammation. Current laboratory tests, that were developed over 40 years ago, do not capture low to moderate levels of CNS inflammation and provide limited information about its phenotype.

This technology includes the creation and use of a polyclonal antibody for Neuroligin-4, NLGN4, that was created by injecting a peptide surrounding the pT707 phosphorylation site into rabbits and affinity purifying the resulting serum. Neuroligin-4 is a member of the neuroligin family of cell adhesion proteins. This family has been shown to play a role in the maturation and function of the neuronal synapse and has been implicated in patients with autism and intellectual disability.

This invention includes the identification of a new mutation in the collagen type VI (COL6A1) gene, including a method for diagnosing and treating patients with this mutation. Collagen type VI-related dystrophies (COL6-RD) are devastating neuromuscular disorders that manifest with progressive generalized muscle weakness, contractures, and respiratory failure. Currently, no cure exists for COL6-RD.

This technology includes an automated and non-invasive assessment system called Automated Identification of subjects at risks of Multiple Sclerosis (AIMS) to assist clinicians in their diagnostic evaluation. A focus of AIMS is the ‘central vein sign’ (and other radiological findings).

This technology includes the design and use of several nanobodies that bind to the SARS-CoV2 spike protein receptor binding domain and block spike protein interaction with the angiotensin converting enzyme 2 (ACE2) receptor. Nanobodies are 12-15 kDa single-domain antibody fragments that are more stable and easier to produce in large quantities compared to conventional antibodies. SARS-CoV2 is the virus responsible for the COVID19 pandemic. The SARS-CoV2 spike protein is responsible for viral entry into human cells via interaction with ACE2 on the cell surface.

This technology includes the design and implementation for 1H-nuclear magnetic resonance imaging (MRI) that allows single transmit hardware to be "transformed" for another nucleus excitation to perform multi-nuclear MR. A radiofrequency (RF) optically controlled switch-mode amplifier prototype is tuned for excitation of two nuclei. The amplifier received the nuclei carrier signals optically through a single fiber.

This technology includes Cyclopentane-modified Peptide Nucleic Acids (cp-PNAs) which can be combined with (forced-intercalation) FIT-PNAs to create highly sensitive probes that detect the presence of complementary RNA sequences. We have studied the beneficial effects of incorporating cyclopentane groups into the backbone of PNAs, which leads to proper preorganization of the PNA backbone into the conformations needed to bind complementary RNA sequences. The cp-PNAs typically have improved thermodynamic stability for binding to complementary nucleic acids compared to unmodified PNAs.

This technology includes a sensitive and specific method to rapidly detect antibodies in biofluids. This assay has been used for the detection of antibodies in blood, urine, and saliva. Until now, no one has used LIPS to detect clinically relevant antibodies to SARS-CoV-2 Nucleocapsid (N) or Spike (S) in saliva. Briefly, LIPS employs recombinantly synthesized target proteins or peptides (e.g., S and N proteins) tagged with light-emitting proteins as targets to be captured by host produced immunoglobulins. These immunoglobulins can be captured by protein A/G beads and immobilized.

This technology includes an alternative synthetic biotin-streptavidin replacement system for use in the development of clinical diagnostics. Peptide nucleic acids (PNA) when functionalized onto the surface of microspheres are capable of targeting short RNA targets from solutions. However, when the target nucleic acid becomes longer and complicated in structure, the PNA no longer efficiently binds due to steric hindrance from the microspheres and/or slow hybridization kinetics of larger nucleic acid targets.