Oligonucleotides can be used to modulate gene expression via a range of processes
including RNAi, target degradation by RNase H-mediated cleavage, splicing modulation …

B-type lamins are fundamental components of the nuclear lamina, a complex structure that
acts as a scaffold for organization and function of the nucleus. Lamin B1 and B2, the most …

Lamin B1 is an essential protein of the nuclear lamina that plays a crucial role in nuclear
function and organization. It has been demonstrated that lamin B1 is essential for …

The CRISPR-based genome editing holds immense potential to fix disease-causing
mutations, however, must also handle substantial natural genetic variations between …

Nanoparticles with oligonucleotides bound to the outside or incorporated into the matrix can
be used for gene editing or to modulate gene expression in the CNS. These nanocarriers …

Gene therapy is emerging as a powerful tool to modulate abnormal gene expression, a
hallmark of most CNS disorders. The transformative potentials of recently approved gene …

Introduction Ribonucleic acid (RNA) therapeutics are a new class of drugs whose
importance is highlighted by the growing number of molecules in the clinic. Sources of data …

Autosomal-dominant leukodystrophy (ADLD) is a rare fatal neurodegenerative disorder with
overexpression of the nuclear lamina component, Lamin B1 due to LMNB1 gene duplication …

Human genome wide association studies confirm the association of the rs738409 single
nucleotide polymorphism (SNP) in the gene encoding protein patatin like phospholipase …

Classical osteogenesis imperfecta (OI) is an inherited rare brittle bone disease caused by
dominant mutations in the COL1A1 or COL1A2 genes, encoding for the α chains of collagen …