MARC details
| 000 -LEADER |
|---|
| fixed length control field |
03785nam a22006017a 4500 |
| 008 - FIXED-LENGTH DATA ELEMENTS--GENERAL INFORMATION |
|---|
| fixed length control field |
140422s20132013 xxu||||| |||| 00| 0 eng d |
| 022 ## - INTERNATIONAL STANDARD SERIAL NUMBER |
|---|
| International Standard Serial Number |
0969-7128 |
| 040 ## - CATALOGING SOURCE |
|---|
| Original cataloging agency |
Ovid MEDLINE(R) |
| 099 ## - LOCAL FREE-TEXT CALL NUMBER (OCLC) |
|---|
| PMID |
22551778 |
| 245 ## - TITLE STATEMENT |
|---|
| Title |
MRI roadmap-guided transendocardial delivery of exon-skipping recombinant adeno-associated virus restores dystrophin expression in a canine model of Duchenne muscular dystrophy. |
| 251 ## - Source |
|---|
| Source |
Gene Therapy. 20(3):274-82, 2013 Mar. |
| 252 ## - Abbreviated Source |
|---|
| Abbreviated source |
Gene Ther. 20(3):274-82, 2013 Mar. |
| 253 ## - Journal Name |
|---|
| Journal name |
Gene therapy |
| 260 ## - PUBLICATION, DISTRIBUTION, ETC. |
|---|
| Year |
2013 |
| 260 ## - PUBLICATION, DISTRIBUTION, ETC. |
|---|
| Manufacturer |
FY2013 |
| 266 ## - Date added to catalog |
|---|
| Date added to catalog |
2014-04-22 |
| 520 ## - SUMMARY, ETC. |
|---|
| Abstract |
Duchenne muscular dystrophy (DMD) cardiomyopathy patients currently have no therapeutic options. We evaluated catheter-based transendocardial delivery of a recombinant adeno-associated virus (rAAV) expressing a small nuclear U7 RNA (U7smOPT) complementary to specific cis-acting splicing signals. Eliminating specific exons restores the open reading frame resulting in translation of truncated dystrophin protein. To test this approach in a clinically relevant DMD model, golden retriever muscular dystrophy (GRMD) dogs received serotype 6 rAAV-U7smOPT via the intracoronary or transendocardial route. Transendocardial injections were administered with an injection-tipped catheter and fluoroscopic guidance using X-ray fused with magnetic resonance imaging (XFM) roadmaps. Three months after treatment, tissues were analyzed for DNA, RNA, dystrophin protein, and histology. Whereas intracoronary delivery did not result in effective transduction, transendocardial injections, XFM guidance, enabled 30+10 non-overlapping injections per animal. Vector DNA was detectable in all samples tested and ranged from <1 to >3000 vector genome copies per cell. RNA analysis, western blot analysis, and immunohistology demonstrated extensive expression of skipped RNA and dystrophin protein in the treated myocardium. Left ventricular function remained unchanged over a 3-month follow-up. These results demonstrated that effective transendocardial delivery of rAAV-U7smOPT was achieved using XFM. This approach restores an open reading frame for dystrophin in affected dogs and has potential clinical utility. |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Topical term or geographic name entry element |
*Dependovirus/ge [Genetics] |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Topical term or geographic name entry element |
*Dystrophin/ge [Genetics] |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Topical term or geographic name entry element |
*Magnetic Resonance Imaging/mt [Methods] |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Topical term or geographic name entry element |
*Muscular Dystrophy, Duchenne/th [Therapy] |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Topical term or geographic name entry element |
*RNA, Small Nuclear/ge [Genetics] |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Topical term or geographic name entry element |
*Transduction, Genetic/mt [Methods] |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Topical term or geographic name entry element |
Animals |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Topical term or geographic name entry element |
Base Sequence |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Topical term or geographic name entry element |
Blotting, Western |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Topical term or geographic name entry element |
Disease Models, Animal |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Topical term or geographic name entry element |
Dogs |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Topical term or geographic name entry element |
Dystrophin/me [Metabolism] |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Topical term or geographic name entry element |
Exons/ge [Genetics] |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Topical term or geographic name entry element |
Female |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Topical term or geographic name entry element |
Gene Expression |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Topical term or geographic name entry element |
Genetic Therapy/mt [Methods] |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Topical term or geographic name entry element |
Genetic Vectors/ge [Genetics] |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Topical term or geographic name entry element |
Humans |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Topical term or geographic name entry element |
Immunohistochemistry |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Topical term or geographic name entry element |
Male |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Topical term or geographic name entry element |
Molecular Sequence Data |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Topical term or geographic name entry element |
Muscular Dystrophy, Duchenne/ge [Genetics] |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Topical term or geographic name entry element |
Muscular Dystrophy, Duchenne/me [Metabolism] |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Topical term or geographic name entry element |
Myocardium/me [Metabolism] |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Topical term or geographic name entry element |
Reverse Transcriptase Polymerase Chain Reaction |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Topical term or geographic name entry element |
RNA, Small Nuclear/me [Metabolism] |
| 650 ## - SUBJECT ADDED ENTRY--TOPICAL TERM |
|---|
| Topical term or geographic name entry element |
Sf9 Cells |
| 651 ## - SUBJECT ADDED ENTRY--GEOGRAPHIC NAME |
|---|
| Institution |
MedStar Heart & Vascular Institute |
| 657 ## - INDEX TERM--FUNCTION |
|---|
| Medline publication type |
Journal Article |
| 657 ## - INDEX TERM--FUNCTION |
|---|
| Medline publication type |
Research Support, N.I.H., Intramural |
| 657 ## - INDEX TERM--FUNCTION |
|---|
| Medline publication type |
Research Support, Non-U.S. Gov't |
| 700 ## - ADDED ENTRY--PERSONAL NAME |
|---|
| Local Authors |
Barbash, Israel M |
| 790 ## - Authors |
|---|
| All authors |
Barbash IM, Bogan JR, Cecchini S, Faranesh AZ, Garcia L, Hoyt RF, Kornegay JN, Kotin RM, Lederman RJ, Li L, Virag T, Yang Y |
| 856 ## - ELECTRONIC LOCATION AND ACCESS |
|---|
| DOI |
<a href="http://dx.doi.org/10.1038/gt.2012.38">http://dx.doi.org/10.1038/gt.2012.38</a> |
| Public note |
http://dx.doi.org/10.1038/gt.2012.38 |
| 942 ## - ADDED ENTRY ELEMENTS (KOHA) |
|---|
| Koha item type |
Journal Article |
| Item type description |
Article |
