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Current Therapeutic Approaches in Leukodystrophies: A Review.

ABSTRACT: Leukodystrophies are a heterogeneous class of genetic diseases affecting the white matter in the central nervous system with a broad range of clinical manifestations and a frequently progressive course. An interest in precision medicine has emerged over the last several decades, and biomedical research in leukodystrophies has made exciting advances along this front through therapeutic target discovery and novel disease model systems. In this review, we discuss current and emerging therapeutic approaches in leukodystrophies, including gene therapy, antisense oligonucleotide therapy, CRISPR/CAS-based gene editing, and cell and stem cell based therapies.

SUBMITTER: Gordon-Lipkin E

PROVIDER: S-EPMC6698898 | biostudies-literature | 2018 Nov

REPOSITORIES: biostudies-literature

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Current Therapeutic Approaches in Leukodystrophies: A Review.

Gordon-Lipkin Eliza E Fatemi Ali A

Journal of child neurology 20180816 13

Leukodystrophies are a heterogeneous class of genetic diseases affecting the white matter in the central nervous system with a broad range of clinical manifestations and a frequently progressive course. An interest in precision medicine has emerged over the last several decades, and biomedical research in leukodystrophies has made exciting advances along this front through therapeutic target discovery and novel disease model systems. In this review, we discuss current and emerging therapeutic ap ...[more]

PMID: 30112967

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