In cystic fibrosis, researchers have used CRISPR HDR-mediated knock-in to correct mutations in the CFTR gene in airway stem cells. For sickle cell disease, the FDA has approved a CRISPR/Cas9 therapy ...

In contrast, the CRISPR/Cas9 size range is 4000-5000 base pairs, sometimes requiring more than one AAV for its delivery (see image below). Although the ARCUS platform is completely applicable to ...

This process is dramatically more efficient for insertion of foreign genetic material at sites when Cas9 is used to introduce targeted DSBs. While HDR has been achieved for years with conventional ...

The CRISPR/Cas9-mediated genome editing approach has been applied to ... Precise corrections can be made in the DNA or directed sequences can be inserted through HDR repair system Created with ...

We will also consider the generation of zebrafish disease models from a practical standpoint, by discussing how genome editing technologies, particularly the recently developed clustered regularly ...

Human extracellular matrices can be edited in their composition using the CRISPR/Cas9 system, leading to materials exhibiting tailored regenerative capacities.

Advances in the gene-editing technology known as CRISPR-Cas9 over the past 15 years have yielded important new insights into ...