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Developing a Novel, Precise and Programmable Genome Editing Technology

Developing a Novel, Precise and Programmable Genome Editing Technology
开发新颖、精确、可编程的基因组编辑技术
批准号:
105691
负责人:
金额:
$25.11万
依托单位:
依托单位国家:
英国
项目类别:
Collaborative R&D
财政年份:
2019
资助国家:
英国
项目状态:
已结题
起止时间:
2019 至 --

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中文摘要
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英文摘要
Pencil Biosciences aims to develop a novel and artificial genome-editing tool, that retains the programmability of the CRISPR-Cas system but overcomes the challenges associated with the currently available genome-editing technologies. It intends to achieve this by aggregating the advances in various fields of science, especially genetic engineering and synthetic chemistry and infusing desired characteristics in an artificial and synthetic molecule.The CRISPR-Cas9 system is a re-purposed, powerful RNA-guided DNA targeting platform for genome editing. Unfortunately, it has many disadvantages such as off-target editing, a large size that restricts efficient delivery, a requirement for PAM sequences and immunogenicity etc.An optimal genome-editing tool should be able to edit any targeted locus in a genome with a high degree of specificity and without any undesired effects. However, in a real world, such a tool doesn't exist as nature developed it for a (partially) different purpose. Scientists have therefore recognized the necessity to improve the existing tools' scope, especially in the context of eukaryotic genome modulation and human therapeutic applications. Some of their efforts have already started yielding fantastic advances towards this objective. Nevertheless, one big drawback of these advances is their narrow focus. Oftentimes improvement in one area of the genome-editing tool does not offer any solutions to existing issues in another area.Pencil Biosciences aim to overcome these challenges with its holistic approach in solving the problems and by pooling the advances made in the different areas of modern sciences.
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