特集 神経疾患治療の進歩2023
脊髄小脳変性症治療開発 update
キーワード:
multiple system atrophy,
α–synuclein,
spinocerebellar degeneration,
spinocerebellar ataxia,
disease–modifying therapy
2024 年 41 巻 5 号 p. 740-746
詳細
2024 年 41 巻 5 号 p. 740-746
Spinocerebellar ataxia (SCA) and multiple system atrophy (MSA) are two neurodegenerative disorders with limited treatment options. Recent advancements in therapeutic strategies for these conditions have shown promising results. In the case of MSA, therapies targeting α–synuclein aggregation, neuroinflammation, and energy metabolism, along with cell–based therapies like mesenchymal stem cells, are under investigation. For SCA, novel molecular genetic approaches such as CRISPR/Cas9 gene editing, RNA interference (RNAi), and antisense oligonucleotides (ASO) are being developed to target the underlying genetic mutations. This paper reviews the current state of therapeutic development for SCA and MSA, including the latest clinical trial data and potential future directions. The ongoing research aims to slow disease progression and improve the quality of life for patients affected by these debilitating conditions.
