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Functional Analysis of Complex Structural and Splice‐Altering Variants in the ARSB Gene Towards the Personalized Antisense‐Based Therapy for Mucopolysaccharidosis Type VI Patients...
by
Bychkov, Igor
,
Filatova, Alexandra
,
Baydakova, Galina
,
Sikora, Nataliya
,
Garifullina, Emiliya
,
Bykova, Anna
,
Tabakov, Vyacheslav
,
Skretnev, Alexandr
,
Skoblov, Mikhail
,
Zakharova, Ekaterina
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Human mutation
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Human Mutation
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Antisense Therapy
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Enzymes
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Genes
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Genetics & Heredity
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Life Sciences & Biomedicine
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Maroteaux-Lamy Syndrome
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Mucopolysaccharidosis
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Patients
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Polymerase Chain Reaction
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Science & Technology
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Structure-Function Relationships
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