In Vivo Administration of Splice Switching PNAs Using the mdx Mouse as a Model System

Publikation: Bidrag til bog/antologi/rapport › Bidrag til bog/antologi › Forskning › fagfællebedømt

Standard

In Vivo Administration of Splice Switching PNAs Using the mdx Mouse as a Model System. / Brolin, Camilla; Lim, Ernest Wee Kiat; Nielsen, Peter E.

Peptide Nucleic Acids. Humana Press, 2020. s. 241-250 (Methods in Molecular Biology, Bind 2105).

Publikation: Bidrag til bog/antologi/rapport › Bidrag til bog/antologi › Forskning › fagfællebedømt

Harvard

Brolin, C, Lim, EWK & Nielsen, PE 2020, In Vivo Administration of Splice Switching PNAs Using the mdx Mouse as a Model System. i Peptide Nucleic Acids. Humana Press, Methods in Molecular Biology, bind 2105, s. 241-250. https://doi.org/10.1007/978-1-0716-0243-0_15

APA

Brolin, C., Lim, E. W. K., & Nielsen, P. E. (2020). In Vivo Administration of Splice Switching PNAs Using the mdx Mouse as a Model System. I Peptide Nucleic Acids (s. 241-250). Humana Press. Methods in Molecular Biology Bind 2105 https://doi.org/10.1007/978-1-0716-0243-0_15

Vancouver

Brolin C, Lim EWK, Nielsen PE. In Vivo Administration of Splice Switching PNAs Using the mdx Mouse as a Model System. I Peptide Nucleic Acids. Humana Press. 2020. s. 241-250. (Methods in Molecular Biology, Bind 2105). https://doi.org/10.1007/978-1-0716-0243-0_15

Author

Brolin, Camilla ; Lim, Ernest Wee Kiat ; Nielsen, Peter E. / In Vivo Administration of Splice Switching PNAs Using the mdx Mouse as a Model System. Peptide Nucleic Acids. Humana Press, 2020. s. 241-250 (Methods in Molecular Biology, Bind 2105).

Bibtex

@inbook{33b2ac4cf88a42068bfb3f155b4202af,
title = "In Vivo Administration of Splice Switching PNAs Using the mdx Mouse as a Model System",
abstract = "Duchenne muscular dystrophy (DMD) is the most common and severe form of muscular dystrophy and is caused by gene mutations that abolish production of functional dystrophin muscle protein. A promising new treatment exploits specifically targeted RNA-acting drugs that are able to partially restore the dystrophin protein. The mdx mouse model (animal model of DMD) serves as a good in vivo model for testing these antisense drugs. The simplest in vivo test, which circumvents the systemic circulation, is intramuscular administration of the compound. After 7 days it is possible to detect exon skipping by reverse transcriptase PCR, and newly synthesized dystrophin-positive fibers by immunohistochemistry and western blotting. All muscles, including the heart, are affected by the disease and must be treated. Therefore the use of antisense therapy for treatment of DMD requires systemic administration, and the model is also useful for systemic administration.",
keywords = "Administration, Exon skipping, mdx, PNA",
author = "Camilla Brolin and Lim, {Ernest Wee Kiat} and Nielsen, {Peter E.}",
year = "2020",
doi = "10.1007/978-1-0716-0243-0_15",
language = "English",
isbn = "978-1-0716-0242-3",
series = "Methods in Molecular Biology",
publisher = "Humana Press",
pages = "241--250",
booktitle = "Peptide Nucleic Acids",
address = "United States",

}

RIS

TY - CHAP

T1 - In Vivo Administration of Splice Switching PNAs Using the mdx Mouse as a Model System

AU - Brolin, Camilla

AU - Lim, Ernest Wee Kiat

AU - Nielsen, Peter E.

PY - 2020

Y1 - 2020

N2 - Duchenne muscular dystrophy (DMD) is the most common and severe form of muscular dystrophy and is caused by gene mutations that abolish production of functional dystrophin muscle protein. A promising new treatment exploits specifically targeted RNA-acting drugs that are able to partially restore the dystrophin protein. The mdx mouse model (animal model of DMD) serves as a good in vivo model for testing these antisense drugs. The simplest in vivo test, which circumvents the systemic circulation, is intramuscular administration of the compound. After 7 days it is possible to detect exon skipping by reverse transcriptase PCR, and newly synthesized dystrophin-positive fibers by immunohistochemistry and western blotting. All muscles, including the heart, are affected by the disease and must be treated. Therefore the use of antisense therapy for treatment of DMD requires systemic administration, and the model is also useful for systemic administration.

AB - Duchenne muscular dystrophy (DMD) is the most common and severe form of muscular dystrophy and is caused by gene mutations that abolish production of functional dystrophin muscle protein. A promising new treatment exploits specifically targeted RNA-acting drugs that are able to partially restore the dystrophin protein. The mdx mouse model (animal model of DMD) serves as a good in vivo model for testing these antisense drugs. The simplest in vivo test, which circumvents the systemic circulation, is intramuscular administration of the compound. After 7 days it is possible to detect exon skipping by reverse transcriptase PCR, and newly synthesized dystrophin-positive fibers by immunohistochemistry and western blotting. All muscles, including the heart, are affected by the disease and must be treated. Therefore the use of antisense therapy for treatment of DMD requires systemic administration, and the model is also useful for systemic administration.

KW - Administration

KW - Exon skipping

KW - mdx

KW - PNA

U2 - 10.1007/978-1-0716-0243-0_15

DO - 10.1007/978-1-0716-0243-0_15

M3 - Book chapter

C2 - 32088875

AN - SCOPUS:85079917856

SN - 978-1-0716-0242-3

T3 - Methods in Molecular Biology

SP - 241

EP - 250

BT - Peptide Nucleic Acids

PB - Humana Press

ER -

ID: 239812123