Evolution of CRISPR/cas systems for precise genome editing
M Hryhorowicz, D Lipiński, J Zeyland - International Journal of Molecular …, 2023 - mdpi.com
The bacteria-derived CRISPR/Cas (an acronym for regularly interspaced short palindromic
repeats/CRISPR-associated protein) system is currently the most widely used, versatile, and …
repeats/CRISPR-associated protein) system is currently the most widely used, versatile, and …
The landscape of CRISPR/Cas9 for inborn errors of metabolism
AF Leal, N Fnu, E Benincore-Flórez… - Molecular genetics and …, 2023 - Elsevier
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 …
palindromic repeats and CRISPR-associated protein 9 (CRISPR/Cas9) system has opened …
Iron oxide-coupled CRISPR-nCas9-based genome editing assessment in mucopolysaccharidosis IVA mice
Mucopolysaccharidosis (MPS) IVA is a lysosomal storage disorder caused by mutations in
the GALNS gene that leads to the lysosomal accumulation of keratan sulfate (KS) and …
the GALNS gene that leads to the lysosomal accumulation of keratan sulfate (KS) and …
CRISPR/nCas9-Based genome editing on GM2 gangliosidoses fibroblasts via non-viral vectors
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 …
system due to the impaired GM2 ganglioside degradation inside the lysosome. Under …
Molecular Trojan Horses for treating lysosomal storage diseases
Lysosomal storage diseases (LSDs) are caused by monogenic mutations in genes encoding
for proteins related to the lysosomal function. Lysosome plays critical roles in molecule …
for proteins related to the lysosomal function. Lysosome plays critical roles in molecule …
Evidence of epigenetic landscape shifts in mucopolysaccharidosis IIIB and IVA
Lysosomal storage diseases (LSDs) are a group of monogenic diseases characterized by
mutations in genes coding for proteins associated with the lysosomal function. Despite the …
mutations in genes coding for proteins associated with the lysosomal function. Despite the …
Mucopolysaccharidosis IVA: Current Disease Models and Drawbacks
Mucopolysaccharidosis IVA (MPS IVA) is a rare disorder caused by mutations in the N-
acetylgalactosamine-6-sulfate-sulfatase (GALNS) encoding gene. GALNS leads to the …
acetylgalactosamine-6-sulfate-sulfatase (GALNS) encoding gene. GALNS leads to the …
[HTML][HTML] CRISPR/Cas9 technology in the modeling of and treatment of mucopolysaccharidosis
M Reyhani-Ardabili, S Ghafouri-Fard - Biochemistry and Biophysics …, 2024 - Elsevier
Mucopolysaccharidosis (MPS) syndromes are a group of heterogeneous genetic disorders
in terms of genetic basis and clinical manifestations, ranging from mild to fatal forms. There …
in terms of genetic basis and clinical manifestations, ranging from mild to fatal forms. There …
Diminished GALNS activity in induced pluripotent stem cells of mucopolysaccharidosis IVA caused by compound p. S162Y and p. C165F mutation
X Jiang, S Chen, Z Jiang, D Chen… - … Journal of Medicine, 2024 - academic.oup.com
Abstract Background Mucopolysaccharidosis (MPS) IVA is a lysosomal storage disorder
caused by mutations in the gene encoding the galactosamine (N-acetyl)-6-sulfatase …
caused by mutations in the gene encoding the galactosamine (N-acetyl)-6-sulfatase …
Lipi nski, D.; Zeyland, J. Evolution of CRISPR/Cas Systems for Precise Genome Editing
M Hryhorowicz - Int. J. Mol. Sci, 2023 - europepmc.org
The bacteria-derived CRISPR/Cas (an acronym for regularly interspaced short palindromic
repeats/CRISPR-associated protein) system is currently the most widely used, versatile, and …
repeats/CRISPR-associated protein) system is currently the most widely used, versatile, and …