OPTIDICER CONSTRUCT FOR AGE-RELATED MACULAR DEGENERATION

    公开(公告)号:US20220401580A1

    公开(公告)日:2022-12-22

    申请号:US17776563

    申请日:2020-11-12

    摘要: Provided are nucleotide sequences encoding polypeptides with ribonuclease III activity, wherein the nucleotide sequences have been modified to reduce their regulation by miRNAs. In some embodiments, the nucleotide sequences are at least 50% and as much as 100% identical to SEQ ID NO: 20 or SEQ ID NO: 22, and/or encode polypeptides that are at least 90% percent identical to SEQ ID NO: 23. Also provided are vectors and host cells that include the nucleotide sequences, methods for expressing the nucleotide sequences in cells, tissues, and organs, which in some embodiments can be in the eye of a subject in need thereof, methods for preventing and/or treating development of diseases or disorders and/or for restoring undesirably low DICER1 expression using the nucleotide sequences, and pharmaceutical compositions that have the presently disclosed nucleotide sequences.

    MORPHOLINOS, MORPHOLINO UPREGULATING, AND ASSOCIATED METHODS
    9.
    发明申请
    MORPHOLINOS, MORPHOLINO UPREGULATING, AND ASSOCIATED METHODS 有权
    莫比诺霉素,吗啉升华和相关方法

    公开(公告)号:US20160040169A1

    公开(公告)日:2016-02-11

    申请号:US14358572

    申请日:2012-11-15

    IPC分类号: C12N15/113

    摘要: Various methods and compositions relating to vascular endothelial growth factor receptor 2 (VEGFR2) are provided. In one aspect, a method of increasing expression of the soluble form of VEGFR2 (sVEGFR2) in a subject can include binding an antisense morphoiino to an exon13-intron13 splicing site of VEGFR2 mRNA such that the VEGFR2 niRNA is spliced into a sVEGFR2 isoform,

    摘要翻译: 提供了与血管内皮生长因子受体2(VEGFR2)相关的各种方法和组合物。 一方面,增加受试者中可溶形式的VEGFR2(sVEGFR2)的表达的方法可包括将反义形态结合到VEGFR2 mRNA的外显子13-内含子13剪接位点,使得VEGFR2 niRNA被剪接成sVEGFR2同种型,