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Nucleic Acid-Based Therapeutic Approach for Spinal and Bulbar Muscular Atrophy and Related Neurological Disorders
Personal take on this article: This paper focuses on spinal and bulbar muscular atrophy (SBMA), an adult-onset neurodegenerative disorder linked to motor neuron and skeletal muscle damage, caused by trinucleotide expansions in the androgen receptor gene. SBMA shares features with polyglutamine diseases and motor neuron disorders, affecting both the central nervous system and muscle function. The paper emphasizes nucleic acid-based therapies like antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs) …
