Gene Therapy

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Gene Therapy

China RP Integrative Treatment Center has started the collaboration with China Gene Technology to provide access to gene therapy for our RP patients worldwide. Through cooperation with gene therapy authorities and top RP specialists, China Gene Technology has nearly 20 years of research experience on gene therapy for inherited retinal disorders. We are focusing on gene replacement therapy and gene editing therapy, as these two methods complement each other.

In 2013, as CRISPR technology became more and more mature, our team began to establish different gene mutations animal models and develop corresponding gene therapies.

The reasons why we put RP as the top research priority are:

1, The incidence of RP in China is 1/1000, which is much higher than the incidence of 1/4000 in other countries in the world. There are about 1.4 million RP patients in China.

2, Because the eyeballs are relatively independent organs with immune pardoning properties, they can circumvent some of the problems that arise in gene therapy, which is relatively easy to breakthrough in gene therapy.

At present, our team are focusing on two gene mutations: RPGR and CYP4V2. These two genes have a high mutation rate among Chinese RP patients. RPGR gene mutation accounts for 10% to 15% of the total RP population in China. Because it’s an x-linked genetic disorder, it’s easy to find more patients in the same family. Patients with CYP4V2 gene mutation are mainly concentrated in China, Japan, South Korea, Singapore, etc. There are no related research and treatment options for these genotypes in western countries.

For these two types of mutations, we take the complementary treatment strategies, that is virus vector replacement therapy and gene editing. The two programs have different characteristics and different specific patients. They can play a complementary role.

Generally speaking, gene replacement therapy can only be applied to genetic diseases with missing functional genes. And it also involves importing foreign genes, which is difficult to completely match the expression in the body, this will affect the treatment efficacy. Gene-editing therapy only modifies the mutated gene, it does not affect the regulatory sequence. It has a more permanent effect. But if it is regarding a large fragment deletion that exceeds the scope of gene editing, gene replacement therapy is required. Anyway, the two strategies have their own advantages and disadvantages.

Currently, we have carried out phase II clinical trials of the following genotypes: USH2A, RHO, ABCA4, CYP4V2, RPGR. There will be more gene therapies for other gene mutations available in the near future.

If you already know your gene mutation type and want to keep abreast of the latest gene therapy trends, please submit the online evaluation form and attach your gene test report. We will inform you of the appropriate treatment options as the first priority.


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