Genome-editing technologies have ushered in a new era in gene therapy, providing novel
therapeutic strategies for a wide range of diseases, including both genetic and nongenetic …

Main conclusion This review provides a comprehensive overview of the CRISPR/Cas9
technique and the research areas of this gene editing tool in improving wheat quality …

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

Since its discovery as a genome editing tool, the clustered regularly interspaced short
palindromic repeats and CRISPR-associated protein 9 (CRISPR/Cas9) system has opened …

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 …

Since its discovery in 2012, the clustered regularly interspaced short palindromic repeats
(CRISPR) and CRISPR-associated protein 9 (Cas9) system has supposed a promising …

The past decade has witnessed an expansion of molecular approaches facilitating the
differential diagnosis of ectodermal dysplasias, a group of genetic diseases characterized by …

Retinitis pigmentosa (RP) is one of the most common forms of hereditary
neurodegeneration. It is caused by one or more of at least 3,100 mutations in over 80 genes …

Since the development of CRISPR/Cas9 gene editing in 2012, therapeutic editing research
has produced several phase 1-2a trials. Here we provide an overview of the mechanisms …

Results Magnetoliposome endocytosis‑mediated uptake is not cytotoxic and increases the
transfec‑tion ratio on primary MPS IVA fibroblasts. Magnetoliposomes used in this study are …