Here, we present a new Cpf1 ortholog, EeCpf1, as a genome-editing tool; this ortholog is derived from the gut bacterial species. EeCpf1 exhibits a higher cleavage activity with the Mnmetal cofactor and efficiently cuts the target DNA with an engineered, nucleotide extended crRNA at the 5′...
A method for CRISPR-based genome editing that harnesses cellular non-homologous end joining activity to achieve targeted DNA knock-in in non-dividing tissues. A current challenge in genome editing is achieving efficient targeted integration of transgenes
In vivo gene editing has the potential to permanently cure genetic diseases. Unlike some editing technologies that have been designed as research tools and subsequently engineered for therapeutic use, ARCUS is a precise and versatile genome editing technology with the distinct potential to insert, delet...
Overall, the development of therapeutic strategies leads currently into two directions: (i) ex vivo genome editing, and (ii) in vivo genome editing (Fig. 1). Ex vivo approaches are based on the idea to take a skin biopsy, de-differentiate the cells into induced pluripotent stem cells (iPS...
We have previously shown that in vivo genome editing can be applied successfully with therapeutic benefit in an engineered Rosa26 locus.7,10 Here, we demonstrate the therapeutic potential of targeting the endogenous albumin locus by insertion of a variety of transgenes in wild-type C57BL/6 mice...
In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy Duchenne muscular dystrophy (DMD) is a devastating disease affecting about 1 out of 5000 male births and caused by mutations in the dystrophin gene. Genome... CE Nelson,CH Hakim,DG Ousterout,... ...
CRISPR genome editing tools are driving innovations in precision genetic medicine. However, in vivo delivery of CRISPR components for treating diseases remains a challenge as researchers strive to eliminate or reduce off-target effects, provide transient editing systems, and increase the rates of gene ...
In vivo genome editing via CRISPR/Cas9 mediated homology- independent targeted integration. Nature 540, 144-149.Suzuki K, Tsunekawa Y, Hernandez-Benitez R, Wu J, Zhu J, Kim EJ, Hatanaka F, Yamamoto M, Araoka T, Li Z, Kurita ... X Meng,R Jia,X Zhao,... - 《Nature Communications》...
In vitro, ZFNs have been shown to promote efficient genome editing via homology-directed repair by inducing a site-specific double-strand break (DSB) at a target locus2,3,4, but it is unclear whether ZFNs can induce DSBs and stimulate genome editing at a clinically meaningful level in vivo...
A method for CRISPR-based genome editing that harnesses cellular non-homologous end joining activity to achieve targeted DNA knock-in in non-dividing tissues. A current challenge in genome editing is achieving efficient targeted integration of transgenes