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A new and effective gene therapy has been developed that can target a rare mutation causing Amyotrophic Lateral Sclerosis (ALS), a neurological disease that leads to the death of nerve cells and loss of motor functions, often resulting in death within 2 to 5 years of diagnosis. Preliminary study results have shown that this treatment, which relies on antisense nucleic acids, led to improvements in the symptoms of a patient with a rare form of the disease, including a reduction in the levels of proteins associated with the damaged nerve cells and improvements in motor and respiratory functions over the course of a year. Researchers hope that this therapy will pave the way for developing more effective treatment strategies for neurodegenerative diseases linked to rare genetic mutations, though longer-term follow-up on a larger number of patients is necessary to confirm these benefits.
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