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Therapy: Gene Therapy

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Total 5 results found since Jan 2013.

Pre‐existing interleukin 10 in cerebral arteries attenuates subsequent brain injury caused by ischemia/reperfusion
Abstract Recurrent stroke is difficult to treat and life threatening. Transfer of anti‐inflammatory gene is a potential gene therapy strategy for ischemic stroke. Using recombinant adeno‐associated viral vector 1 (rAAV1)‐mediated interleukin 10 (IL‐10), we investigated whether transfer of beneficial gene into the rat cerebral vessels during interventional treatment for initial stroke could attenuate brain injury caused by recurrent stroke. Male Wistar rats were administered rAAV1‐IL‐10, rAAV1‐YFP, or saline into the left cerebral artery. Three weeks after gene transfer, rats were subjected to occlusion of the...
Source: IUBMB Life - August 1, 2015 Category: Research Authors: Qiu‐Juan Liang, Mei Jiang, Xin‐Hong Wang, Li‐Li Le, Meng Xiang, Ning Sun, Dan Meng, Si‐Feng Chen Tags: Research Communication Source Type: research

Gene Addition Strategies for β-Thalassemia and Sickle Cell Anemia.
Authors: Dong AC, Rivella S Abstract Beta-thalassemia and sickle cell anemia are two of the most common diseases related to the hemoglobin protein. In these diseases, the beta-globin gene is mutated, causing severe anemia and ineffective erythropoiesis. Patients can additionally present with a number of life-threatening co-morbidities, such as stroke or spontaneous fractures. Current treatment involves transfusion and iron chelation; allogeneic bone marrow transplant is the only curative option, but is limited by the availability of matching donors and graft-versus-host disease. As these two diseases are monogenic ...
Source: Advances in Experimental Medicine and Biology - November 13, 2017 Category: Research Tags: Adv Exp Med Biol Source Type: research

Current and Novel Therapeutical Approaches of Classical Homocystinuria in Childhood With Special Focus on Enzyme Replacement Therapy, Liver-Directed Therapy and Gene Therapy
J Clin Med Res. 2023 Feb;15(2):76-83. doi: 10.14740/jocmr4843. Epub 2023 Feb 28.ABSTRACTClassical homocystinuria is a hereditary defect of the enzyme cystathionine beta synthase, which is produced in the liver. If this enzyme fails, the synthesis pathway of cysteine from methionine is interrupted, leading to the accumulation of homocysteine in the blood plasma and homocysteine in the urine. After birth, the children are unremarkable except for the characteristic laboratory findings. Symptoms rarely appear before the second year of life. The most common symptom is a prolapse of the crystalline lens. This finding is seen in ...
Source: Clin Med Res - March 10, 2023 Category: Research Authors: Stefan Bittmann Gloria Villalon Elena Moschuring-Alieva Elisabeth Luchter Lara Bittmann Source Type: research