Developing a Novel, Precise and Programmable Genome Editing Technology

Biosciences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，但克服了与目前可用的基因组编辑技术相关的挑战。CRISPR-Cas9系统是一个重新设计的、功能强大的RNA引导的DNA靶向平台，用于基因组编辑。CRISPR-Cas9系统是一个基于RNA的基因组编辑系统，它是一个基于RNA的基因组编辑系统。不幸的是，它具有许多缺点，例如脱靶编辑、限制有效递送的大尺寸、对PAM序列和免疫原性的要求等。最佳的基因组编辑工具应该能够以高度特异性编辑基因组中的任何靶向基因座，并且没有任何不期望的效果。然而，在一个真实的世界里，这样的工具并不存在，因为大自然出于（部分）不同的目的开发了它。因此，科学家们认识到有必要改善现有工具的范围，特别是在真核基因组调控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，这些进步的一个大缺点是它们的焦点狭窄。基因组编辑工具的一个领域的改进通常不会为另一个领域的现有问题提供任何解决方案。生物科学的目标是通过其解决问题的整体方法并通过汇集现代科学不同领域的进步来克服这些挑战。

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.