Malekos Eric, Montano Christy, Carpenter Susan
Department of Biomolecular Engineering, University of California Santa Cruz, California, USA.
Department of Molecular, Cell, and Developmental Biology, University of California Santa Cruz, California, USA.
bioRxiv. 2024 Jun 22:2024.06.18.599405. doi: 10.1101/2024.06.18.599405.
We present CRISPRware, an efficient method for generating guide RNA (gRNA) libraries against transcribed, translated, and noncoding regions. CRISPRware leverages next-generation sequencing data to design context-specific gRNAs and accounts for genetic variation, which allows allele-specific guide design on a genome-wide scale. The latter ability holds promise for the development of gene therapy in the context of gene dosing and dominant negative mutations.
我们展示了CRISPRware，这是一种针对转录区域、翻译区域和非编码区域生成向导RNA（gRNA）文库的有效方法。CRISPRware利用下一代测序数据来设计特定背景的gRNA，并考虑到遗传变异，从而能够在全基因组范围内进行等位基因特异性向导设计。后一种能力为在基因剂量和显性负突变背景下开发基因治疗带来了希望。
