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Have you ever thought about curing genetic diseases with just one shot ?
Currently, gene therapies only enable in vitro gene editing
Car-T is a currently popular gene editing therapy. Although it has made major breakthroughs in in vitro gene editing therapy, the in vitro monoclonal cell purification and culture involved in this therapy has low efficiency and high cost, and its high price excludes many patients.
How to achieve safe, accurate and
convenient in vivo gene editing
If there is a way to achieve in vivo gene editing, it can not only avoid the tedious in vitro single-cell cloning and purification process, shorten the treatment cycle and improve the efficiency of treatment, but also save costs and bring hope of cure to more patients.
We got it !
Our laboratory has innovatively developed the in vivo self-assembly synthesis of Cas9RNP
With it, the gene editing complex developed has the following advantages:
1. High assembly success rate and high activity;
2. Long storage time, which is conducive to its direct injection into the body.
This gene expression pathway has been purchased and used by world-renowned research groups such as Zhang Feng's research group and Jennifer Doudna's research group.
Next, we selected human intestinal probiotic Escherichia coli EcN as the carrier, whose secreted outer membrane vesicles have excellent human compatibility
Compared with the previous adenovirus vector, the advantages of this delivery vector are:
1. It has lower toxicity and less immune response
2. It has better targeting.
Combining the two, we get this in vivo self-assembly system
This is the five.
Therefore, our team aims to develop an in vivo gene editing therapy with high gene editing efficiency, stability, low cost and few side effects
this is six
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The repository used to create this website is available at gitlab.igem.org/2024/hubu-4-chn.