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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

基本信息

批准号：
105691
负责人：
金额：
$ 25.11万
依托单位：
PENCIL BIOSCIENCES LIMITED
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
起止时间：
2019 至无数据
项目状态：
已结题

来源：
https://gtr.ukri.org/projects?ref=105691
关键词：
Developing Novel Precise Programmable Genome

项目摘要

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.

铅笔生物科学公司的目标是开发一种新型的人工基因组编辑工具，该工具保留了CRISPR-CAS系统的可编程性，但克服了与当前可用的基因组编辑技术相关的挑战。它打算通过聚合各个科学领域的进步，特别是基因工程和合成化学的进步，并将所需的特征注入人造和合成分子中来实现这一目标。CRISPR-CAS9系统是一个重新定位的、强大的RNA引导的DNA靶向平台，用于基因组编辑。遗憾的是，它有许多缺点，如非靶点编辑、大尺寸限制了高效传递、对PAM序列的要求和免疫原性等，一个最优的基因组编辑工具应该能够以高度的特异性编辑基因组中的任何靶基因，而不会产生任何不良影响。然而，在现实世界中，这样的工具并不存在，因为大自然开发它是为了(部分)不同的目的。因此，科学家们认识到有必要改进现有工具的范围，特别是在真核基因组调制和人类治疗应用的背景下。他们的一些努力已经开始在实现这一目标方面取得惊人的进展。然而，这些进展的一大缺点是它们的关注点很窄。通常，基因组编辑工具的一个领域的改进并不能为另一个领域的现有问题提供任何解决方案。铅笔生物科学旨在通过其解决问题的整体方法和汇集现代科学不同领域取得的进展来克服这些挑战。