HIGHLIGHTS
- who: Jan Lejman and collaborators from the Student Scientific Society, Independent Laboratory of Genetic Diagnostics, Medical University of Lublin, Lublin, Poland have published the research work: Gene Therapy in ALS and SMA: Advances, Challenges and Perspectives, in the Journal: (JOURNAL)
- what: The aim of this review is to present current progress and prospects for the use of therapy in the treatment of both SMA and ALS. The study aims at evaluating efficacy, safety, pharmacodynamics and pharmacokinetics. The study showed a good distribution of ASO within the central nervous system, effective suppression of rat SOD1 mRNA . . .
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