The gangliosidoses GM2 are a group of pathologies mainly affecting the central nervous
system due to the impaired GM2 ganglioside degradation inside the lysosome. Under …

Ganglioside-monosialic acid (GM1) gangliosidosis, a rare autosomal recessive disorder, is
frequently caused by deleterious single nucleotide variants (SNVs) in GLB1 gene. These …

The GM2-gangliosidoses are neurological diseases causing premature death, thus
developing effective treatment protocols is urgent. GM2-gangliosidoses result from …

Abstract Mucopolysaccharidosis IV A (MPS IVA) is a lysosomal disorder caused by
mutations in the GALNS gene. Consequently, the glycosaminoglycans (GAGs) keratan …

The CRISPR-Cas9 system has significantly advanced regenerative medicine research by
enabling genome editing in stem cells. Due to their desirable properties, mesenchymal stem …

Sphingolipidoses are inherited genetic diseases characterized by the accumulation of
glycosphingolipids. Sphingolipidoses (SP), which usually involve the loss of sphingolipid …

Messenger RNA (mRNA) is being used as part of an emerging class of biotherapeutics with
great promise for preventing and treating a wide range of diseases, as well as encoding …

The CRISPR/Cas9 gene editing technology holds promise for the treatment of multiple
diseases. However, the inability to perform specific gene editing in targeted tissues and …

Abstract CRISPR/Cas9-based genome-editing therapies are poised to change the clinical
outcome for many diseases with validated therapeutic targets awaiting an appropriate …

Nonviral delivery of the CRISPR/Cas9 system provides great benefits for in vivo gene
therapy due to the low risk of side effects. However, in vivo gene editing by delivering the …