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One-pot, degradable, silica nanocarriers with encapsulated oligonucleotides for mitochondrial specific delivery

2023-08-21

Abstract excerpt

Mutations in nuclear and mitochondrial genes are responsible for severe chronic disorders such as mitochondrial myopathies (MM). Gene therapy using antisense oligonucleotides is a promising strategy to treat mitochondrial DNA (mtDNA) diseases by blocking the replication of the mutated mtDNA. However, transport vehicles are needed for intracellular, mitochondria-specific transport of oligonucleotides. Nanoparticle...

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Identifiers and source

Literature Corpus work
9e137e0b-d85e-58bb-9de3-a1dcd7efda15
DOI
10.21203/rs.3.rs-3231179/v1
Open publication

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One-pot, degradable, silica nanocarriers with encapsulated oligonucleotides for mitochondrial specific deliveryDOI 10.21203/rs.3.rs-3231179/v1
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