GENERATION AND CORRECTION OF A HUMANIZED MOUSE MODEL WITH A DELETION OF DYSTROPHIN EXON 44

    公开(公告)号:US20180271069A1

    公开(公告)日:2018-09-27

    申请号:US15914728

    申请日:2018-03-07

    Abstract: Duchenne muscular dystrophy (DMD), which affects 1 in 5,000 male births, is one of the most common genetic disorders of children. This disease is caused by an absence or deficiency of dystrophin protein in striated muscle. The major DMD deletion “hot spots” are found between exon 6 to 8, and exons 45 to 53. Here, a “humanized” mouse model is provided that can be used to test a variety of DMD exon skipping strategies. Among these are, CRISPR/Cas9 oligonucleotides, small molecules or other therapeutic modalities that promote exon skipping or micro dystrophin mini genes or cell based therapies. Methods for restoring the reading frame of exon 44 deletion via CRISPR-mediated exon skipping in the humanized mouse model, in patient-derived iPS cells and ultimately, in patients using various delivery systems are also contemplated. The impact of CRISPR technology on DMD is that gene editing can permanently correct mutations.

    Methods for treating plague
    174.
    发明授权

    公开(公告)号:US10076562B2

    公开(公告)日:2018-09-18

    申请号:US15490261

    申请日:2017-04-18

    Abstract: Provided herein are methods for using compositions that include a fusion protein having a YscF protein domain, a mature F1 protein domain, and a LcrV protein domain. In one embodiment the composition is used to confer immunity to plague, such as pneumonic plague, caused by Yersinia pestis. In one embodiment, the composition is administered to a mucosal surface, such as by an intranasal route. In one embodiment, the administration to a mucosal surface includes a vector that has a polynucleotide encoding a fusion protein, where the fusion protein includes a YscF protein domain, a mature F1 protein domain, and a LcrV protein domain. The administration is followed by a second administration by a different route, such as an intramuscular route. The second administration includes a fusion protein having the same three domains, and in one embodiment the fusion protein is the same one administered to a mucosal surface.

    Diagnosis of benign and cancerous growths by measuring circulating tumor stem cells and serum AnnexinA2

    公开(公告)号:US10067134B2

    公开(公告)日:2018-09-04

    申请号:US15408886

    申请日:2017-01-18

    Inventor: Pomila Singh

    Abstract: Provided herein is a method for diagnosing/prognosing a metastatic cancer in a subject by measuring and detecting one or more of CS-ANXA2, DCAMKL, Lgr5 or CS-ANAX2 and DCAMKL or CS-ANXA2 and Lgr5 positive circulating tumor stem cells in the subject's blood or plasma. Also provided is a method for distinguishing the presence of early stage primary cancer from advanced stage metastatic cancer in the subject by measuring and detecting AnnexinA2, CS-ANXA2 and DCAMKL-1 or Lgr5 in the blood or plasma. In addition, there is provided a method for distinguishing the presence of benign, pre-cancerous tumorous growths or cancerous tumors in the subject by measuring and detecting AnnexinA2 and circulating tumor stem cells positive for CS-ANXA2 and DCAMKL or CS-ANXA2 and Lgr5 in the blood or plasma.

    Methods and systems for therapy of multiple sclerosis

    公开(公告)号:US10029094B2

    公开(公告)日:2018-07-24

    申请号:US14947797

    申请日:2015-11-20

    Abstract: A method for treating neural deficits resulting from multiple sclerosis, involving assessing a subject's motor, sensory, cognitive, or emotional deficits caused by multiple sclerosis; selecting a rehabilitative task based said deficits; determining the subject's acceptable performance threshold for the rehabilitative task; and providing a paired training therapy. The paired training therapy comprises having the subject to perform a trial of the rehabilitative task; classifying the subject's performance on the trial as either acceptable or unacceptable, wherein an acceptable performance is performance at or above the subject's acceptable performance threshold, and an unacceptable performance is performance below the subject's acceptable performance threshold; and reinforcing acceptable performance by selectively stimulating the subject's vagus nerve after completion of the acceptable performance.

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