Delivery of Antisense Oligonucleotides to the Mouse Brain by Intracerebroventricular Injections

Metz, Tom; Kuijper, Elsa; Roon‐Mom, Willeke M. C. van

doi:10.1007/978-1-0716-2010-6_23

Cited by 4 publications

(2 citation statements)

References 6 publications

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“…However, the transport of ASOs to the brain is challenging since ASOs cannot directly cross the BBB. [35] Loading ASOs into INs can solve the problem of weak targeted delivery to the brain. Calero et al integrated cell-permeable DNA-ASO into LNPs to interfere with mRNA expression.…”

Section: Antisense Oligonucleotides (Asos)mentioning

confidence: 99%

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Nucleic acid delivery by ionizable nanocarriers for brain disease treatment

Xiong

Ling

Zhang

et al. 2023

Brain-X

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The successful application of messenger RNA vaccines in the market has demonstrated the potential of gene therapy in treating various diseases, including infectious diseases, autoimmune disorders, brain diseases, and other cancers. However, gene therapy faces great challenges in treating brain diseases such as brain tumors, infections, and strokes because the limitations of the blood-brain barrier make it difficult for nucleic acid drugs to be delivered safely and effectively into the brain. Therefore, there is a high demand for carriers delivering nucleic acid drugs to the brain. Ionizable nanocarriers (INs) have great advantages in gene therapy due to their pH-responsive properties, which facilitate the safe and efficient delivery of targets, responsive release in the disease microenvironment, and the protection of nucleic acids from degradation. To better understand INs and their potential as therapeutic vectors for brain diseases, the present review describes their biological properties, recent progress in the field, and promising applications. In particular, the related prospects and challenges are discussed to promote the further development of INs. K E Y W O R D S blood-brain barrier (BBB), brain diseases, ionizable nanocarriers (INs), nucleic acidsThis is an open access article under the terms of the Creative Commons Attribution License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited.

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Section: Antisense Oligonucleotides (Asos)mentioning

confidence: 99%

“…The use of ASOs is a potential therapeutic strategy for the treating brain diseases. However, the transport of ASOs to the brain is challenging since ASOs cannot directly cross the BBB [35] . Loading ASOs into INs can solve the problem of weak targeted delivery to the brain.…”

Section: The Application Of Ins In Treating Brain Diseasesmentioning

confidence: 99%

Nucleic acid delivery by ionizable nanocarriers for brain disease treatment

Xiong

Ling

Zhang

et al. 2023

Brain-X

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Antisense oligonucleotide-mediated disruption of HTT caspase-6 cleavage site ameliorates the phenotype of YAC128 Huntington disease mice

Kuijper,

Overzier,

Suidgeest

et al. 2024

Neurobiology of Disease

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Experimental Model Systems Used in the Preclinical Development of Nucleic Acid Therapeutics

Zhou

Arechavala‐Gomeza

Garanto

2023

Nucleic Acid Therapeutics

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Preclinical evaluation of nucleic acid therapeutics (NATs) in relevant experimental model systems is essential for NAT drug development. As part of COST Action “DARTER” (Delivery of Antisense RNA ThERapeutics), a network of researchers in the field of RNA therapeutics, we have conducted a survey on the experimental model systems routinely used by our members in preclinical NAT development. The questionnaire focused on both cellular and animal models. Our survey results suggest that skin fibroblast cultures derived from patients is the most commonly used cellular model, while induced pluripotent stem cell-derived models are also highly reported, highlighting the increasing potential of this technology. Splice-switching antisense oligonucleotide is the most frequently investigated RNA molecule, followed by small interfering RNA. Animal models are less prevalent but also widely used among groups in the network, with transgenic mouse models ranking the top. Concerning the research fields represented in our survey, the mostly studied disease area is neuromuscular disorders, followed by neurometabolic diseases and cancers. Brain, skeletal muscle, heart, and liver are the top four tissues of interest reported. We expect that this snapshot of the current preclinical models will facilitate decision making and the share of resources between academics and industry worldwide to facilitate the development of NATs.

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Delivery of Antisense Oligonucleotides to the Mouse Brain by Intracerebroventricular Injections

Cited by 4 publications

References 6 publications

Nucleic acid delivery by ionizable nanocarriers for brain disease treatment

Nucleic acid delivery by ionizable nanocarriers for brain disease treatment

Antisense oligonucleotide-mediated disruption of HTT caspase-6 cleavage site ameliorates the phenotype of YAC128 Huntington disease mice

Experimental Model Systems Used in the Preclinical Development of Nucleic Acid Therapeutics

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