Patent classifications
C12N15/1136
Treatment of ophthalmic conditions with angiopoietin-like 7 (ANGPTL7) inhibitors
The present disclosure provides methods of treating patients having an ophthalmic condition, methods of identifying subjects having an increased risk of developing an ophthalmic condition, methods of detecting human angiopoietin like 7 (ANGPTL7) variant nucleic acid molecules and variant polypeptides, and ANGPTL7 variant nucleic acid molecules and variant polypeptides.
COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE LECT2 GENE
The invention relates to antisense polynucleotide agents targeting the LECT2 gene, and methods of using such antisense polynucleotide agents to inhibit expression of LECT2 and to treat subjects having a LECT2-associated disease, e.g., amyloidosis.
Treatment Of Inflammation With Glucocorticoids And Angiopoietin-Like 7 (ANGPTL7) Inhibitors
The present disclosure provides methods of treating subjects having inflammation with an Angiopoietin-Like 7 (ANGPTL7) inhibitor and a glucocorticoid, methods of decreasing glucocorticoid-induced ophthalmic conditions in subjects, and methods of identifying subjects having an increased risk of developing glucocorticoid-induced ophthalmic conditions.
COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF MUTANT EGFR GENE
The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a mutant Epidermal Growth Factor Receptor (EGFR), and methods of using the dsRNA to inhibit expression of mutant EGFR.
COMPOSITIONS AND METHODS FOR EDITING THE ANGIOPOIETIN-LIKE 4 (ANGPTL4) GENE
The present application provides materials and methods for treating a patient with one or more conditions associated with ANGPTL4 whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of ANGPTL4 gene in a cell by genome editing.
GENE EDITING SYSTEMS COMPRISING A NUCLEASE AND USES THEREOF
The present invention relates to gene editing systems comprising nucleases or nucleic acids encoding the nucleases and RNA guides or nucleic acids encoding the RNA guides, processes for characterizing the gene editing systems, and methods or preparing and/or using the gene editing systems.
RNA interference in ocular indications
The present invention relates to ocular administration of sd-rxRNA and rxRNAori molecules.
TREATMENT OF MAJOR DEPRESSIVE DISORDER BY LOW DOSE INTERLEUKIN-2
Disclosed are methods, compositions of matter, and protocols useful for treatment of major depressive disorder through administration of low dose interleukin-2 at a concentration and/or frequency sufficient to increase expansion of T regulatory cell numbers and/or enhancement of T regulatory cell activity. In some embodiments administration of interleukin-2 is provided as means of enhancing efficacy of standard antidepressant therapies. Furthermore, administration of interleukin-2 receptor agonists is also described in the current invention as a treatment of major depressive disorder.
IL-15 HUMANIZED MOUSE MODEL AND USE THEREOF
Provided are a mouse and a functional activity part thereof, comprising a humanized IL-15 gene; the humanized IL-15 gene comprises a human IL-15 gene segment and a mouse IL-15 gene segment, the human IL-15 gene segment comprises at least a part of exon 4, exon 5, exon 6, exon 7 and exon 8 of the human IL-15 gene, and the mouse IL-15 gene segment comprises exon 1, exon 2 and exon 3 of the mouse IL-15 gene. Also provided are a preparation method and use of the mouse.
ENGINEERED IMMUNE CELLS WITH REDUCED TOXICITY AND USES THEREOF
In the disclosure provided herein are engineered immune cells (e.g., T cells) deficient in INFy expression and have reduced toxicity. Methods of producing the engineered immune cells (e.g., T cells) and methods of using the engineered immune cells (e.g., T cells) to treat cancer or autoimmune disease are also provided.