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Non-Viral Vector-Mediated Gene Therapy for ALS: Challenges and Future Perspectives

19 May 2021

Abstract excerpt

Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease, for which no effective treatment is yet available to either slow or terminate it. Recent advances in gene therapy renew hope for developing an effective approach to control this disease. Non-viral vectors, such as lipid- and polymer-based nanoparticles, cationic polymers, and exosomes, can effectively transfer genes into primary...

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PMID: 34010004
DOI: 10.1021/acs.molpharmaceut.1c00297

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