journal cover

Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology

Nature Medicine

Jan 31, 2006

Julia AlterQi Long Lu

Abstract

For the majority of Duchenne muscular dystrophy (DMD) mutations, antisense oligonucleotide (AON)-mediated exon skipping has the potential to restore a functional protein. Here we show that weekly intravenous injections of morpholino phosphorodiamidate (morpholino) AONs induce expression...read more

Mentioned in this Paper

DMD gene
Mice, Inbred mdx
Intravenous Injections
Muscle Function
Skeletal Muscle Structure
Soleus Muscle Structure
Mice, Inbred C57BL
Morpholinos
Muscular Dystrophy, Animal
Oligonucleotides
170
1
Paper Details
References
  • References15
  • Citations261
  • References15
  • Citations261
12345...

Similar Papers Found In These Feeds

Antisense Oligonucleotide - Therapies For ALS

This feed focuses on antisense oligonucleotide therapies such as Inotersen, Nusinursen, and Patisiran, in neurodegenerative diseases including amyotrophic lateral sclerosis.

Antisense Oligonucleotides: ND

This feed focuses on antisense oligonucleotide therapies such as Inotersen, Nusinursen, and Patisiran, in neurodegenerative diseases including amyotrophic lateral sclerosis.

Myopathies

Myopathies are genetic or acquired disorders of skeletal muscle that lead to varying degrees of weakness, atrophy, and exercise intolerance. Here are the latest discoveries pertaining to this disease.

Muscular Dystrophy

Muscular dystrophy refers to a range of muscle diseases caused by defects in muscle proteins, leading to death of the muscle cells, with loss of muscle tissue, and weakness. Here are the latest discoveries pertaining to this disease.

CREs: Gene & Cell Therapy

Gene and cell therapy advances have shown promising outcomes for several diseases. The role of cis-regulatory elements (CREs) is crucial in the design of gene therapy vectors. Here is the latest research on CREs in gene and cell therapy.

© 2020 Meta ULC. All rights reserved

Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology

Nature Medicine

Jan 31, 2006

Julia AlterQi Long Lu

PMID: 16444267

DOI: 10.1038/nm1345

Abstract

For the majority of Duchenne muscular dystrophy (DMD) mutations, antisense oligonucleotide (AON)-mediated exon skipping has the potential to restore a functional protein. Here we show that weekly intravenous injections of morpholino phosphorodiamidate (morpholino) AONs induce expression...read more

Mentioned in this Paper

DMD gene
Mice, Inbred mdx
Intravenous Injections
Muscle Function
Skeletal Muscle Structure
Soleus Muscle Structure
Mice, Inbred C57BL
Morpholinos
Muscular Dystrophy, Animal
Oligonucleotides
170
1

Related Papers

Paper Details
References
  • References15
  • Citations261
  • References15
  • Citations261
12345...
/papers/systemic-delivery-of-morpholino-oligonucleotide/16444267