Abstract: Artificial expression constructs for selectively modulating gene expression in selected central nervous system cell types are described. The artificial expression constructs can be used to selectively express synthetic genes or modify gene expression in GABAergic interneurons.

Abstract: Newborn screening for primary immunodeficiencies, cystinosis, and Wilson disease is described. The newborn screening can detect these disorders from dried blood spots already routinely collected at the time of birth. Early detection of these disorders will greatly improve patient outcome as each of them can be fatal once symptoms emerge.

Abstract: The invention is directed to a bispecific chimeric antigen receptor, comprising: (a) at least two antigen-specific targeting regions; (b) an extracellular spacer domain; (c) a transmembrane domain; (d) at least one co-stimulatory domain; and (e) an intracellular signaling domain, wherein each antigen-specific targeting region comprises an antigen-specific single chain Fv (scFv) fragment, and binds a different antigen, and wherein the bispecific chimeric antigen receptor is co-expressed with a therapeutic control. The invention also provides methods and uses of the bispecific chimeric antigen receptors.

Abstract: Early detection of lysosomal storage diseases (LSDs) including Mucopolysaccharidosis Type I (MPS I) and Pompe Disease can greatly improve patient outcome as each disease can be fatal once symptoms emerge. Screening for MPS I and Pompe Disease using biological samples including dried blood spots (DBS), buccal swab, peripheral blood mononuclear cells (PBMCs), or white blood cells (WBCs) is described. The disclosed methods and assays provide a robust way to screen newborns for LSDs. The disclosed methods and assays can also allow rapid prediction of whether a patient with LSD will develop an immune response to enzyme replacement therapy (ERT), thus improving treatment for patients with LSDs. The disclosed methods and assays can also further reduce the number of false positives caused by pseudo deficiency cases of LSD, such as MPS I and Pompe Disease.

Abstract: Clinical diagnosis and newborn screening for primary immunodeficiencies (PIDDs) is described. The PIDDs include X-linked chronic granulomatous disease (X-CGD), X-linked lymphoproliferative syndrome (XLP1; SH2D1A deficiency), familial hemophagocytic lymphohistiocytosis 2 (FHL2), ataxia telangiectasia (AT), common variable immunodeficiency (CVID; B-cell dysfunctions), severe combined immunodeficiency (SCID), adenosine deaminase (ADA) deficiency, and dedicator of cytokinesis 8 (DOCKS) deficiency. Additionally, cell specific markers for platelets (CD42), natural killer (NK) cells (CD56), and T cells (CD3epsilon and CD3delta) can be used as secondary markers to provide support for diagnosis of disease.

Abstract: A liposomal drug formulation for treating a disease in a patient characterized by overexpression of HIF-1? and/or HIF-2? includes a plurality of liposomes in a pharmaceutically acceptable carrier. The liposomes encapsulate echinomycin and are made from a peglyated phospholipid, a neutral phosphoglyceride, and a sterol. The PEGylated liposomes may be used to treat proliferative diseases, leukemia, cancer, autoimmune diseases and graft-versus-host disease.

Abstract: Described herein is a reversible aptamer selection technology for production-scale isolation of label-free cells (e.g., CD8+ T cells). Provided herein are methods for isolating a cell of interest from a biological sample by contacting the biological sample with an aptamer that specifically binds a cell surface marker specific for the cell of interest; separating the aptamer-bound cells from cells not bound to the aptamer; and recovering a cell of interest by disrupting binding of the aptamer to the cell surface marker.

Abstract: The present disclosure relates to the co-expression of an endonuclease with an end-processing enzyme for the purpose of enhanced processing of the polynucleotide ends generated by endonuclease cleavage.

Abstract: The present disclosure relates to the co-expression of an endonuclease with an end-processing enzyme for the purpose of enhanced processing of the polynucleotide ends generated by endonuclease cleavage.

Abstract: Methods, compositions, and systems for treating subject(s) in need of plasma Factor VIII, particularly a subject having preexisting anti-FVIII inhibitory antibodies, are provided. The methods involve administering to the subject a therapeutically effective amount of an inflammation suppressor, a therapeutically effective amount of a CD8+ T cell depleting agent, and a therapeutically effective amount of a composition comprising a lentiviral vector (LV) comprising an optimized FVIII expression cassette expressibly linked to a megakaryocyte-specific promoter. Such methods, compositions, and systems are useful to treat subjects with blood clotting disorder(s), such as hemophilia A.

Abstract: Provided herein are anti-CD24 antibodies that selectively bind human CD24 expressed in cancer cells, but not human CD24 expressed in non-cancerous cells, and the use of such antibodies in cancer therapy.

Abstract: The present technology provides systems and methods for oligonucleotide ligation assays (“OLA”). In some embodiments, OLA devices are provided that incorporate pathogen detection testing and drug resistance testing into a single device using a lateral flow membrane. The OLA devices can be used at point of car settings to aid a clinician in selecting an appropriate therapeutic regimen for an infected patient.

Abstract: Examples of systems and methods described herein may estimate a state of the ear canal of a patient utilizing a smart phone by characterizing acoustic waveforms reflected off the patient's eardrum. Examples may include an acoustic focusing apparatus that may be connected to a smart phone to provide acoustic signals to and receive reflected signals from an ear canal.

Abstract: The invention relates to the treatment and prevention of problem behaviour by the administration of a composition that includes an effective amount of Prevotella.

Abstract: The present disclosure relates to the co-expression of an endonuclease with an end-processing enzyme for the purpose of enhanced processing of the polynucleotide ends generated by endonuclease cleavage.

Abstract: The present disclosure relates to the co-expression of an endonuclease with an end-processing enzyme for the purpose of enhanced processing of the polynucleotide ends generated by endonuclease cleavage.

Abstract: The present disclosure relates to the co-expression of an endonuclease with an end-processing enzyme for the purpose of enhanced processing of the polynucleotide ends generated by endonuclease cleavage.

Abstract: The present invention relates to compositions of anti-CTLA-4 antibodies that bind to the human CTLA4 molecule and their use in cancer immunotherapy and for the reduction of autoimmune side effects compared to other immunotherapeutic agents.

Abstract: A method of preventing the development of GvHD or reducing the severity of GvHD in a mammalian subject receiving an allogeneic hematopoietic stem cell (HSC) transplant includes administering to the subject a pharmaceutical composition comprising an active agent that inhibits the biological activity or expression of hypoxia-inducible factor-1a (HIF-1a) or hypoxia-inducible factor-2a (HIF-2a), wherein the active agent is administered in an amount effective for preventing or reducing the severity GvHD in the subject.

Abstract: Selectively providing voltage-gated sodium channel function sufficient to rescue impaired Nav1.1 function to inhibitory neurons is described. Provided voltage-gated sodium channel function sufficient to rescue impaired Nav1.1 function in inhibitory neurons can be used to treat disorders such as epilepsy, and more particularly, Dravet Syndrome.