THERAPEUTICS OF PTD-SMAD7 FUSION PROTEINS
    145.
    发明申请

    公开(公告)号:US20200332302A1

    公开(公告)日:2020-10-22

    申请号:US16959252

    申请日:2018-12-30

    Inventor: Xiao-Jing WANG

    Abstract: The present technology provides methods and compositions for the treatment of inflammatory and/or tissue damage conditions. In particular, the use of truncated Smad7 compositions delivered locally or systemically to a site of inflammation and/or tissue damage is described. Other specific embodiments concern treatment or prevention of side effects caused by radiation and/or chemotherapy, including but not limited to oral and gastric mucositis. Also provided are codon-optimized nucleic acids encoding for Smad7 fusion proteins.

    HIGH-THROUGHPUT BLOCK OPTICAL DNA SEQUENCE IDENTIFICATION

    公开(公告)号:US20200299762A1

    公开(公告)日:2020-09-24

    申请号:US16211817

    申请日:2018-12-06

    Abstract: Disclosed herein is a multiplexed design with three-dimensional plasmonic nanofocusing and confinement of light, demonstration of reproducible and robust single-molecule optical fingerprints using two complementary vibrational spectroscopy techniques (infrared and Raman spectroscopy), identification of respective vibrational modes which uniquely fingerprint the biomolecular species, and facile differentiation of respective fingerprints in DNA mixtures, as well as epigenetic modifications. While the nanometer scale mode volumes still prevent single letter identification of DNA sequence, we show an alternative method for identifying A, T, G, C DNA nucleotides in “k-mers” using sequences of these blocks as a unique and high-throughput alternative to single letter sequences (similar to binary and hexadecimal systems). Furthermore, additivity shown in single-molecule DNA mixtures and robust optical signatures can also be used in a raster-type step scan to identify single letter sequences. These results can pave the way for the development of a novel, high-throughput block optical sequencing (BOS) method.

    METHODS OF TREATING DANON DISEASE
    150.
    发明申请

    公开(公告)号:US20200299727A1

    公开(公告)日:2020-09-24

    申请号:US16827454

    申请日:2020-03-23

    Abstract: The disclosed technology includes methods of treating Danon disease, for example correcting genetic mutations in the LAMP-2 gene and ameliorating at least one Danon disease phenotype, for example defective LAMP-2B-mediated autophagy. In some implementations, the disclosed methods include editing a mutated form of the LAMP-2 gene in a patient in need thereof. In some implementations, editing the mutated form of the LAMP-2 gene may include use of a CRISPR editing technique targeted to the mutated form of the LAMP-2 gene. As a result, mutated LAMP-2 proteins in mammalian subjects may be restored in at least some of the affected cells, for example cardiomyocytes.

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