At present, there are more than 6,000 known genetic defect diseases. As far as China is concerned, the total number of people with serious genetic defect diseases is estimated to be 16.8 million. However, only 1% of genetic defect diseases have effective therapeutic drugs to control for a long time. At present, expensive drugs rely on private donations.
The movie "Lorenzo's Oil" tells of a cute and lively boy, Lorenzo, who suddenly screamed like madness in his mid-4s, and was sent to hospital to learn about the rare genetic disease ALD, namely "adrenal leukodystrophy". At that time, it was incurable, and the children could live for at most one year. The loving parents invented a kind of edible oil for them, which could delay the progress of the disease, so that the children could grow up healthily. The film was adapted from a real story. The oil invented by parents helped many families in real life, but it was very expensive and could not be cured.
At present, our hospital has the technical ability to treat many kinds of genetic defects including ALD. We hope that with the support of Shenzhen government and the people, Shenzhen Institute of Immunogene Therapy can create more "legends of gene therapy"! The CART immunocytotherapy technology project in our hospital is the only fourth generation technology with unique safety mechanism. Medical staff can induce cell self-apoptosis in patients at any time and withdraw completely to the body. Currently, CART immune cell therapy projects are being carried out by the Institute, including hematological tumors (leukemia and lymphoma), brain cancer (neuroblastoma and glioma), bladder cancer, which will be extended to other solid tumors in the future, and large-scale treatment projects for rare diseases with single gene defects, including MLD, ALD, thalassemia, aplastic hematopoietic disorders, etc. 。 Our hospital's CART immunocytotherapy technology and rare disease gene therapy technology platform have reduced the unit course cost to 2% of the cost of European and American counterparts, but the mitigation efficiency is the same. In the future, it is planned to use pre-packaged lentiviral vectors with specific genes as the main products to market all over the country, so that technicians in tertiary hospitals can simplify the process of cell GM modification in accordance with standard procedures, so that the technology can be popularized for the benefit of patients.
In order to improve the high-tech quality of cooperative treatment with nearby hospitals, the Shenzhen Institute of Immunogene Therapy will hold doctor training seminars from time to time. Relevant experts will be invited to attend to the latest treatment technologies and new developments in neighbouring areas, so as to pay attention to the quality of life and economic capacity of patients, provide the most advanced and effective treatment programs, and enable each patient to obtain the best treatment response.