Therapeutic targeting of noncoding RNAs (ncRNAs), such as microRNAs (miRNAs) and
long noncoding RNAs (lncRNAs), represents an attractive approach for the treatment of …

Huntington's disease is caused by an abnormally expanded CAG repeat expansion in the
HTT gene, which confers a predominant toxic gain of function in the mutant huntingtin …

The ability to elicit robust and long-term transgene expression in vivo together with minimal
immunogenicity and little to no toxicity are only a few features that make recombinant adeno …

Several attempts have been made to discover the ideal vector for gene therapy in central
nervous system (CNS). Adeno-associated viruses (AAVs) are currently the preferred vehicle …

Reducing expression of the fetal hemoglobin (HbF) repressor BCL11A leads to a
simultaneous increase in γ-globin expression and reduction in β-globin expression. Thus …

Abstract β-thalassemias (β-thal) are a group of blood disorders caused by mutations in the β-
globin gene (HBB) cluster. β-globin associates with α-globin to form adult hemoglobin (HbA …

The importance of various classes of regulatory non-protein-coding RNA molecules
(ncRNAs) in the normal functioning of the CNS is becoming increasingly evident. ncRNAs …

Rodents remain one of the most widespread and damaging invasive alien species on
islands globally. The current toolbox for insular rodent eradications is reliant on the …

Spinobulbar muscular atrophy (SBMA) is caused by CAG expansions in the androgen
receptor gene. Androgen binding to polyQ-expanded androgen receptor triggers SBMA …

RNA interference has become a ubiquitous biological tool, and is being harnessed for
therapeutic purposes as well. Therapeutic posttranscriptional gene silencing takes …