Nucleic acid therapeutics in neurodevelopmental disease.

Nucleic acid therapeutics in neurodevelopmental disease. Curr Opin Genet Dev. 2020 Jul 02;65:112-116 Authors: Winkelsas AM, Fischbeck KH Abstract Nucleic acid therapeutics allow sequence-based targeting of mutation-harboring genes. They can be used to increase the expression and function of disease genes or to decrease the expression of toxic gene products. Antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), and gene-replacement therapies have received FDA approval, and in vivo gene editing applications are currently under development. Special consideration should be given to target engagement in neurons and amelioration of neurological phenotypes. Here we discuss the uses and limitations of different nucleic acid therapeutics, highlighting examples in the clinical and pre-clinical application of these modalities for the treatment of neurodevelopmental diseases. PMID: 32623324 [PubMed - as supplied by publisher]
Source: Current Opinion in Genetics and Development - Category: Genetics & Stem Cells Authors: Tags: Curr Opin Genet Dev Source Type: research