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1.
Current Strategies for Increasing Knock-In Efficiency in CRISPR/Cas9-Based Approaches.
Int J Mol Sci
; 25(5)2024 Feb 20.
Article
in English
| MEDLINE | ID: mdl-38473704
2.
The landscape of CRISPR/Cas9 for inborn errors of metabolism.
Mol Genet Metab
; 138(1): 106968, 2023 01.
Article
in English
| MEDLINE | ID: mdl-36525790
3.
Bone Growth Induction in Mucopolysaccharidosis IVA Mouse.
Int J Mol Sci
; 24(12)2023 Jun 08.
Article
in English
| MEDLINE | ID: mdl-37373036
4.
Mucopolysaccharidoses: Cellular Consequences of Glycosaminoglycans Accumulation and Potential Targets.
Int J Mol Sci
; 24(1)2022 Dec 28.
Article
in English
| MEDLINE | ID: mdl-36613919
5.
CRISPR/nCas9-Based Genome Editing on GM2 Gangliosidoses Fibroblasts via Non-Viral Vectors.
Int J Mol Sci
; 23(18)2022 Sep 14.
Article
in English
| MEDLINE | ID: mdl-36142595
6.
GM2 Gangliosidoses: Clinical Features, Pathophysiological Aspects, and Current Therapies.
Int J Mol Sci
; 21(17)2020 Aug 27.
Article
in English
| MEDLINE | ID: mdl-32867370
7.
Iduronate-2-sulfatase interactome: validation by yeast two-hybrid assay.
Heliyon
; 8(3): e09031, 2022 Mar.
Article
in English
| MEDLINE | ID: mdl-35284671
8.
Evaluation of HIV-1 derived lentiviral vectors as transductors of Mucopolysaccharidosis type IV a fibroblasts.
Gene
; 780: 145527, 2021 May 15.
Article
in English
| MEDLINE | ID: mdl-33636292
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