Gene therapy is a promising approach in the Batten disease treatment market. Batten disease is caused by mutations in specific genes, which lead to the accumulation of toxic substances in the brain and ultimately, neurodegeneration. Gene therapy aims to replace or repair the faulty gene to address the root cause of the disease.
In Batten disease, gene therapy involves delivering a healthy copy of the affected gene to the patient's cells. This can be done using various methods, such as viral vectors or CRISPR/Cas9 genome editing. Once the healthy gene is delivered, it can produce the normal protein, which can help to prevent the accumulation of toxic substances and slow the progression of the disease.
Clinical trials of gene therapy for Batten Disease Treatment Market, and early results have been promising. In some cases, patients have shown significant improvements in their symptoms, such as improved vision and cognitive function.
While there are still challenges to be addressed in the development of gene therapy for Batten disease, such as the delivery of the therapy to the brain and long-term safety, the potential of this approach to treat this devastating disease is significant.
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