Blood-Brain Barrier
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A rapidly developing technology with widespread treatment potential is adeno-associated virus gene therapy. IN stereotaxic brain surgery, AAV2 vectors have been successfully injected directly into the brain to treat aromatic L-amino acid decarboxylase deficiency. Additionally, as a treatment for spinal muscular atrophy, gene therapy with the AAV9 vector, which crosses the blood-brain barrier, has been administered to more than 2,000 patients worldwide. Gene therapies for a variety of paediatric diseases have been developed using AAV vectors.Hemophilia and ornithine transcarboxylase deficiency gene therapy trials are currently underway. For Niemann-Pick disease type C, glucose transporter I deficiency, and spinocerebellar ataxia type 1, clinical trials are planned. Because the genome of AAV vectors is in the episode and rarely integrates into chromosomes, the vectors are safe. However, serious side effects like thrombotic microangiopathy and hepatic failure have been reported, and ongoing research focuses on developing vectors that are more effective at lowering dosages. Since gene therapy was first used to treat diseases, there have been numerous ups and downs. However, as the number of gene therapy products on the market indicates, the journey toward gene therapy has reached a significant turning point. Gene therapy has a bright future based on the products that have been approved and those that are not yet approved, as well as the numerous clinical trials conducted in this area. Pharmaceutical companies, policymakers, and researchers could learn a lot from the trend in gene therapy strategies, vectors, and targets. After briefly describing the history of gene therapy, this paper looks at both current gene therapy products and potential gene therapies that might be approved soon.We also looked at how gene therapy clinical trials strategies have changed over the past ten years, including the use of vectors, target cells, transferred genes, and ex-vivo/in-vivo techniques. We also looked at the most important fields into which gene therapy has entered. Cancer currently has the greatest number of gene therapy clinical trials, despite the fact that gene therapy was initially used to treat genetic diseases. Future clinical products may be influenced by changes in gene therapy strategies, particularly in pioneering nations. Abdominal aortic aneurysms still require a treatment, according to the cardiovascular field. The rupture that occurs as a result of this inflammatory disease has a high mortality rate and is frequently not diagnosed until a later stage. There are no pharmaceutical options for treatment.
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With Regards,
Sara Giselle
Associate Managing Editor
Journal of Critical Care Obsestrics & Gynocology