CRISPR/Cas9 technology in the modeling of and treatment of mucopolysaccharidosis

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 are a number of applied or prospective treatment modalities for MPS, including bone marrow transplantation, enzyme repla...

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Bibliographic Details
Published in:Biochemistry and Biophysics Reports
Main Authors: Mehran Reyhani-Ardabili, Soudeh Ghafouri-Fard
Format: Article
Language:English
Published: Elsevier 2024-09-01
Subjects:
Mucopolysaccharidosis
MPS
CRISPR/Cas9
Online Access:http://www.sciencedirect.com/science/article/pii/S2405580824001353
Description
Summary: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 are a number of applied or prospective treatment modalities for MPS, including bone marrow transplantation, enzyme replacement therapy, targeted gene therapy and substrate reduction therapy. Recently, CRISPR/Cas9 technology has emerged as a novel tool for several metabolic disorders, such as MPS. This review concentrates on the application of this technique in the treatment of MPS, particularly MPS I, and modeling of disease-causing mutations.
ISSN:2405-5808

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