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  • Gene Therapy
    The Gene Editing Technology Innovation Platform
    Hematopoietic Stem Cell Platform

    Gene Editing Technology Innovation Platform

    Gene Editing Technology Innovation Platform

    The Gene Editing Technology Innovation Platform (CRISTARS®) is dedicated to developing international leading gene editing tools, obtaining core technologies with independent intellectual property rights, and paving the important foundation for gene editing technology translation. The platform is based on the combination of different single-stranded DNA deaminase structural domains and Cas9 nickase to develop a new generation of DNA base editing tools for broader and more accurate gene editing. The platform has successfully created the world-leading dual base editor and the ultra-high activity cytidine single-base editor (hyper CBE, HyCBE), which can efficiently convert two bases, improve editing activity and broaden the target range. The research results have been published in high-impact peer-review journals such as Nature Biotechnology, Nature Cell Biology, and Cell Research.

    World's first successful application of CRISPR/Cas9 for mammalian gene editing

    Gene editing is a technology to modify genes with precise modifications, which can be achieved with high efficiency and specificity for specific genomic DNA (or RNA), just as freely and accurately as editing text. In August 2013, a team of Bioraylab scientists published in the prestigious international journal Nature Biotechnology (IF≈43.11) the description of The CRISPR/Cas9 system was successfully used to edit genes in mice and rats. In the years since, Bioraylab scientists have continued to improve the gene editing system, applying it to a variety of applications including gene therapy, cell therapy and humanized animals.

    The development of the era of gene therapy

    In 2021, China's first CAR-T product, Aquilensai injection, was approved for marketing by the Chinese NMPA for post-treatment relapsed or refractory diffuse large B-cell lymphoma (DLBCL)
    In 2020,CRISPR/Cas9 gene editing technology wins Nobel Prize in Chemistry
    In June 2019, zynteglo, the world's first gene therapy drug ,was approved for marketing by the EU EMA for the treatment of thalassemia
    In May 2019, Zolgensma, the world's first gene therapy drug,was approved for marketing by the FDA for the treatment of SMA
    In 2018, the first CRISPR/Cas9-based in vivo gene therapy clinical trial (EDIT-101) was approved by the FDA for the treatment of Leber Congenital Amaurosis Type 10 (LCA)
    In 2017, Kymriah, the world's first CAR-T product, received FDA approval to market for the treatment of relapsed or refractory acute lymphoblastic leukemia (ALL)
    In 2016, the first generation of single-base editors were developed
    In November 2013, the third generation gene editing technology CRISPR/Cas9 (clustered regularly spaced short palindromic repeats) was introduced
    In 2012, Glybera, the world's first AAV gene therapy drug, was approved for marketing by the EU EMA for the treatment of lipoprotein lipase deficiency (LPLD)
    In 2010, TALENs (transcription activation-like effector nucleases), a second-generation gene editing technology, was introduced
    In 2003, Gendicine, the world's first gene therapy drug, was approved for marketing by the NMPA in China for the treatment of head and neck squamous cell carcinoma (HNSCC)
    In 2001, the first generation of gene editing technology ZFNs (zinc finger nucleases) was introduced
    In 1990, the world's first clinical trial of gene therapy was approved by the FDA for the treatment of adenosine deaminase deficiency (ADA-SCID)
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