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We discuss the challenges for the molecular therapy in the brain and we highlight the renaissance of nucleic acid-based drugs to treat neurodegenerative diseases. We describe the potential therapeutic effects that can be obtained by inhibiting Yin and increasing Yang gene expression.

The Yin side of nucleic acid-based therapy: we summarize the current technologies and we provide examples of their applications to treat to neurodegenerative diseases in mouse models and in clinical trials. We provide examples of activatory RNA therapy in neurodegenerative disorders in mouse models and in clinical trials. Open to the public ; None of your libraries hold this item.

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Be the first to add this to a list. Comments and reviews What are comments? CrossRef Google Scholar. Benson, M. Inotersen treatment for patients with hereditary transthyretin amyloidosis. N Engl J Med, , 22— Adams, D. Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis. N Engl J Med , , 11— Chiriboga, C. Neurology, 86, — Aartsma-Rus, A.

Conformation of Nucleic acids, Drug-DNA interactions; Thermal stability of Nucleic Acids

FDA Approval of nusinersen for spinal muscular atrophy makes the year of splice modulating oligonucleotides. Nucleic Acid Ther, 27, 67— Corey, D.

Development of nucleic acid vaccines: use of self-amplifying RNA in lipid nanoparticles

Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy. Nat Neurosci, 20, — Seguin, R. Seguin pp. Targeted oligonucleotides for treating neurodegenerative tandem repeat diseases.

[Full text] Development of nucleic acid vaccines: use of self-amplifying RNA in li | IJN

Neurotherapeutics ; this issue. Google Scholar. Genetics, mechanisms and therapeutic progress in polyglutamine spinocerebellar ataxias. Henninger N, Mayasi Y. Nucleic acid therapies for ischemic stroke. Nucleic acid-based therapeutics relevant to neuroimmune conditions. Krichevsky A, Uhlmann EJ.

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Oligonucleotide therapeutics as a new class of drugs for malignant brain tumors: targeting mRNAs, regulatory RNAs, mutations, combinations, and beyond. Cohen, J. Informational drugs: a new concept in pharmacology.

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Antisense Res Dev, 1, — Khvorova, A. The chemical evolution of oligonucleotide therapies of clinical utility. Nat Biotechnol, 35, — Antisense oligonucleotides for the treatment of inner ear dysfunction.

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