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Optimizing AAV9 Therapy for SMARD1: Safety and Efficacy
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BIOENGINEER.ORGOptimizing AAV9 Therapy for SMARD1: Safety and Efficacy
In the realm of genetic disorders, SMARD1 (Spinal Muscular Atrophy with Respiratory Distress 1) and CMT2S (Charcot-Marie-Tooth Disease type 2S) stand out due to their profound impact on motor functions and respiratory health. Recent advancements in gene therapy present a transformative approach that might alter the therapeutic landscape for these debilitating conditions. Researchers are now focusing on AAV9 (Adeno-Associated Virus serotype 9) as…
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