CRISPR-Based In Vivo Genome Editing and Base Editing

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CRISPR-Based In Vivo Genome Editing and Base Editing

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 correction in somatic tissue.

In this webinar from GEN and The CRISPR Journal, Dr. Wen Xue will present a modular delivery strategy for homology-directed repair by combining lipid nanoparticle delivery of Cas9 mRNA with adeno-associated viruses (AAV) encoding a sgRNA and a repair template. In a mouse model of human hereditary tyrosinemia, gene correction efficiency of >6% in hepatocytes was achieved after a single application, suggesting the potential of Cas9-based therapeutic genome editing.

Dr. Xue will also discuss advances in base editing, a highly promising tool to correct single-base pathogenic mutations. Efficient base editing requires cellular exposure to levels of base editors that can be difficult to attain in hard-to-transfect cells or in vivo. Dr. Xue will present new results on a chemically modified mRNA-encoded adenine base editor (ABE) that mediates robust base editing. This ABE shows the therapeutic potential in correcting pathogenic mutations in cell and animal models of diseases. Cas9 and ABE mRNA expands the applicability of CRISPR-associated gene-editing tools in vitro and in vivo.

Wen Xue, PhD
Assistant Professor
RNA Therapeutics Institute atUniversity of Massachusetts Medical School

Ubigene Biosciences is co-founded by biological academics and elites from China, the United States, and France. We are located in Guangzhou Science City, which serves as a global center for high technology and innovation. Ubigene Biosciences has 1000㎡ office areas and laboratories, involving genome editing, cell biology technology, and zebrafish research. We provide products and services for plasmids, viruses, cells, and zebrafish. We aim to provide customers with better gene-editing tools for cell or animal research.

We developed CRISPR-U™ and CRISPR-B™(based on CRISPR/Cas9 technology) which is more efficient than general CRISPR/Cas9 in double-strand breaking, CRISPR-U™ and CRISPR-B™ can greatly improve the efficiency of homologous recombination, easily achieve knockout (KO), point mutation (PM) and knockin (KI) in vitro and in vivo. 

Genome Editing Platform
——Focusing on the Application of CRISPR-U™ and CRISPR-B™ Gene Editing Technology
1. Provides various types of gene-editing vectors for different species.
2. Provides different virus packaging services, including lentiviruses, adenoviruses and adeno-associated viruses.3. Provides high-quality services for gene knockout, point mutation and knockin cell lines

Cell Biology Platform
——Focusing on primary cell
1. Provides over 400 types of primary cells.
2. Provides culture strategies and related products for different cell types.3. Provides cell biology-related services such as cell isolation, extraction and validation.