AAV vectors for gene editing

Provided herein are polynucleotides configured for incorporation into recombinant adeno-associated virus (AAV) vectors. These polynucleotides encode auxiliary components of CRISPR proteins, gRNAs, and AAV vectors for modifying target nucleic acids. The system can also be used to introduce cells, such as eukaryotic cells having mutations in the target nucleic acid of a gene. Also provided are methods of modifying cells having such mutations using such AAV vectors. 本文提供了被配置用于掺入重组腺相关病毒(AAV)载体中的多核苷酸。这些多核苷酸编码用于修饰靶核酸的CRISPR蛋白、gRNA和AAV载体的辅助组分。该系统还可用于导入细胞，例如在基因的该靶核酸中具有突变的真核细胞。还提供了使用此类AAV载体修饰具有此类突变的细胞的方法。