Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

DG9-conjugated morpholino rescues phenotype in SMA mice by reaching the CNS via a subcutaneous administration.

JCI insight - 8 Mar 2023

Aslesh Tejal, Erkut Esra, Ren Jun, Lim Kenji Rowel Q, Woo Stanley, Hatlevig Susan, Moulton Hong M, Gosgnach Simon, Greer John, Maruyama Rika, Yokota Toshifumi

Abstract excerpt

Antisense oligonucleotide-mediated (AO-mediated) therapy is a promising strategy to treat several neurological diseases, including spinal muscular atrophy (SMA). However, limited delivery to the CNS with AOs administered intravenously or subcutaneously is a major challenge. Here, we demonstrate a single subcutaneous administration of cell-penetrating peptide DG9 conjugated to an AO called phosphorodiamidate...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Mice
Share to Morpholinos

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Muscular Atrophy, Spinal

Share to Oligonucleotides, Antisense

Share to Phenotype

Share to RNA Splicing

Source

Open in PubMed Open full text

Identifiers

PMID: 36719755
DOI: 10.1172/jci.insight.160516
PMCID: PMC10077475

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 3 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

DG9-conjugated morpholino rescues phenotype in SMA mice by reaching the CNS via a subcutaneous administration.PMID 36719755Read ↗

DG9-Conjugated Morpholino Rescues Phenotype in Spinal Muscular Atrophy Mice.Semantic proximity Read ↗

Cell-penetrating peptide-conjugated Morpholino rescues SMA in a symptomatic preclinical model.Semantic proximity Read ↗

Challenges and future perspective of antisense therapy for spinal muscular atrophy: A reviewSemantic proximity Read ↗

Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy.Semantic proximity Read ↗

Comparison of the efficacy of MOE and PMO modifications of systemic antisense oligonucleotides in a severe SMA mouse model.Semantic proximity Read ↗

Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy.Semantic proximity Read ↗

Effect of combined systemic and local morpholino treatment on the spinal muscular atrophy Δ7 mouse model phenotype.Semantic proximity Read ↗

Systemic and ICV Injections of Antisense Oligos into SMA Mice and Evaluation.Semantic proximity Read ↗

Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseasesSemantic proximity Read ↗

Systemic delivery of a splice-switching oligonucleotide heteroduplex corrects splicing in central nervous system and muscle in spinal muscular atrophy miceSemantic proximity Read ↗

Gene-Targeting Therapeutics for Neurological Disease: Lessons Learned from Spinal Muscular AtrophySemantic proximity Read ↗

A Short Antisense Oligonucleotide Ameliorates Symptoms of Severe Mouse Models of Spinal Muscular AtrophySemantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.