METHOD
    2.
    发明申请
    METHOD 有权

    公开(公告)号:US20240424023A1

    公开(公告)日:2024-12-26

    申请号:US18690333

    申请日:2022-09-09

    Abstract: The present invention provides a method of engineering a cell, comprising the steps of introducing into the cell: i) a site-directed nuclease which is capable of cleaving a target nucleotide sequence at the 3′-end of Intron 1 of a Cytotoxic T-Lymphocyte Associated Protein 4 gene (CTLA4); and ii) a nucleic acid construct which comprises a nucleic acid sequence comprising one or more of exon 2, exon 3 and exon 4 of CTLA4, or a sequence with at least 70% identity to exon 2, exon 3 and/or exon 4 of the CTLA4 gene, and 5′- and 3′-homology arms, wherein each of the 5′ and '3 homology arms is essentially complementary to a sequence flanking the target nucleotide sequence at the 3′-end of Intron 1 of CTLA4.

    CASRX/CAS13D SYSTEMS TARGETING C9ORF72

    公开(公告)号:US20240374757A1

    公开(公告)日:2024-11-14

    申请号:US18554454

    申请日:2022-04-19

    Abstract: Provided herein is a composition comprising (i) a nucleic acid sequence encoding a CasRx/Cas13d polypeptide; and (ii) a guide RNA that binds specifically to a target sequence in C9orf72 RNA. Also provided are associated pharmaceutical compositions, guide RNAs, complexes, vectors and cells, and uses of the compositions to neurodegenerative disorders.

    Capsid
    6.
    发明授权
    Capsid 有权

    公开(公告)号:US12138318B2

    公开(公告)日:2024-11-12

    申请号:US16541516

    申请日:2019-08-15

    Abstract: There is described an AAV capsid protein having an amino acid sequence which has at least 98% identity to the sequence of SEQ ID NO: 3 or at least 94% identity to the sequence of SEQ ID NO: 4. Also described is a pharmaceutical composition, an AAV capsid and a viral particle comprising the capsid protein, a recombinant AAV vector comprising a nucleotide sequence which encodes for the capsid protein, and a host cell and a transgenic animal comprising the capsid protein or the vector. In addition, there is described a method of transferring a nucleic acid of interest into a mammal comprising introducing a recombinant AAV vector into the mammal, wherein the recombinant AAV vector comprises a gene of interest which is encapsidated into a capsid comprising the capsid protein.

    METHODS AND REAGENTS FOR THE DETECTION OF ANALYTES

    公开(公告)号:US20240345086A1

    公开(公告)日:2024-10-17

    申请号:US18579635

    申请日:2022-07-20

    CPC classification number: G01N33/56983 G01N33/6848 G01N2333/165 G01N2469/20

    Abstract: The present invention relates to a high throughput targeted proteomics-based method for identifying and quantifying one or more binding molecules in a biological fluid of an individual. The present invention involves the use of a plurality of bait molecules to capture the binding molecules. The invention uses proteomics techniques, such as mass spectrometry, for the identification and quantification of the binding molecules. The methods of the invention can be used to identify and quantify antibodies produced by an individual, and proteins expressed by an individual, following exposure to antigens, such as viral antigens, particularly coronavirus antigens and more particularly SARS-CoV-2. Exposure may be via vaccination or following natural infection. The methods of the invention can be used to identify and quantify molecules which bind to a range of proteins of interest, such as autoantigens and neoantigens, as well as to viral vectors.

    NANOMECHANICALLY ACTUATED NUCLEIC ACID NANOPORE

    公开(公告)号:US20240280530A1

    公开(公告)日:2024-08-22

    申请号:US18570995

    申请日:2022-06-17

    CPC classification number: G01N27/3278

    Abstract: A membrane-spanning actuatable nucleic acid nanopore is provided. The nanopore comprises: one or more polynucleotide strands that provide a scaffold component and a plurality of polynucleotide strands that provide a plurality of staple components wherein each of the plurality of staple components hybridise to the scaffold component; and a trigger that is actuatable in response to a stimulus. Actuation of the trigger results in a detectable conformational change of the nanopore from a first conformation to at least a second conformation. Detection may be via a signal change, such as via fluorescence (e.g. FRET) or a change in an electrical signal readout. Semi-fluid membranes, and sensor devices comprising the actuatable nucleic acid nanopores are also provided.

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