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Have you ever imagined to curing genetic diseases with a single injection ?
Currently, CAR-T therapy has made significant advancements in gene editing
However, it involves inefficient and costly purification and culture of monoclonal cells outside the body, making its exorbitant price unaffordable for many patients.
So is it possible to develop a more efficient and cost-effective gene editing therapy?
The realization of in vivo gene editing could simplify the treatment process, improve efficiency, and reduce costs, thereby expanding therapeutic potential to a wider range of patients.
How to realize this more efficient and cost-effective in-vivo gene editing therapy
Our team has recently developed a method for the in vivo self-assembly of Cas9 RNP gene editing
complexes using Escherichia coli as a culture cell. These complexes exhibit high activity and
stability, making them powerful agents for gene editing.
To date, this technology has been adopted by more than 200 laboratories worldwide through
Addgene, including esteemed researchers like Zhang Feng from MIT and Nobel laureate Jennifer
Doudna's group at UC Berkeley.
Build a safe and efficient targeted gene-editing delivery system
Building upon this foundation, our project aims to utilize probiotic Escherichia coli Nissle1917
for the production of safer Cas9 RNPs. Additionally, we plan to encapsulate RNPs within Outer
Membrane Vesicles (OMVs), which will be generated through physical extrusion and engineered to
display targeting elements on their surfaces.
Our ultimate objective is to engineer an efficient, stable, cost-effective, and minimally invasive in vivo gene editing therapy that advances the field while enhancing global public health.
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The repository used to create this website is available at gitlab.igem.org/2024/hubu-4-chn.
The repository used to create this website is available at gitlab.igem.org/2024/hubu-4-chn.