Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors.

Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors. Research Abstract Details 

Research Abstract Table of Contents

Jump to the:

Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors. Abstract Text:

Mariacarmela Allocca,Claudio Mussolino,Maria Garcia-Hoyos,Daniela Sanges,Carolina Iodice,Marco Petrillo,Luk H Vandenberghe,James M Wilson,Valeria Marigo,Enrico M Surace,Alberto Auricchio,Mariacarmela Allocca,Claudio Mussolino,Maria Garcia-Hoyos,Daniela Sanges,Carolina Iodice,Marco Petrillo,Luk H Vandenberghe,James M Wilson,Valeria Marigo,Enrico M Surace,Alberto Auricchio,

Severe inherited retinal diseases, such as retinitis pigmentosa and Leber congenital amaurosis, are caused by mutations in genes preferentially expressed in photoreceptors. While adeno-associated virus (AAV)-mediated gene transfer can correct retinal pigment epithelium (RPE) defects in animal models, approaches for the correction of photoreceptor-specific diseases are less efficient. We evaluated the ability of novel AAV serotypes (AAV2/7, AAV2/8, AAV2/9, AAV2rh.43, AAV2rh.64R1, and AAV2hu.29R) in combination with constitutive or photoreceptor-specific promoters to improve photoreceptor transduction, a limiting step in photoreceptor rescue. Based on a qualitative analysis, all AAV serotypes tested efficiently transduce the RPE as well as rod and cone photoreceptors after subretinal administration in mice. Interestingly, AAV2/9 efficiently transduces Müller cells. To compare photoreceptor transduction from different AAVs and promoters in both a qualitative and quantitative manner, we designed a strategy based on the use of a bicistronic construct expressing both enhanced green fluorescent protein and luciferase. We found that AAV2/8 and AAV2/7 mediate six- to eightfold higher levels of in vivo photoreceptor transduction than AAV2/5, considered so far the most efficient AAV serotype for photoreceptor targeting. In addition, following subretinal administration of AAV, the rhodopsin promoter allows significantly higher levels of photoreceptor expression than the other ubiquitous or photoreceptor-specific promoters tested. Finally, we show that AAV2/7, AAV2/8, and AAV2/9 outperform AAV2/5 following ex vivo transduction of retinal progenitor cells differentiated into photoreceptors. We conclude that AAV2/7 or AAV2/8 and the rhodopsin promoter provide the highest levels of photoreceptor transduction both in and ex vivo and that this may overcome the limitation to therapeutic success observed so far in models of inherited severe photoreceptor diseases.

Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors. Publishing Authors By Initials

M Allocca,C Mussolino,M Garcia-Hoyos,D Sanges,C Iodice,M Petrillo,LH Vandenberghe,JM Wilson,V Marigo,EM Surace,A Auricchio,M Allocca,C Mussolino,M Garcia-Hoyos,D Sanges,C Iodice,M Petrillo,LH Vandenberghe,JM Wilson,V Marigo,EM Surace,A Auricchio,

For similar abstracts research abstracts see: abstracts research

PUBMED ID PMID:

MEDLINE DATE:

Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors. Journal Published:

PUBLICATION TYPE: Research Support, Non-U.S. Gov

Journal: Journal of virology

VOLUME: 81

Page Numbers: 11372-80

Journal Abbreviation: J. Virol.

ISSN: 0022-538X

DAY: 15

MONTH: 08

YEAR: 2007

Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors. Information

Number of References:

LANGUAGE: eng

NlmUniqueID: 113724

Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors. Keywords Mesh Terms:

KEYWORDS:

MESH TERMS:

Chemical & Substance for Abstract: Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors. Information

Substance Name:

Registry Number:

Grant and Affiliation Information for Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors.

AFFILIATION: Telethon Institute of Genetics and Medicine, Via P. Castellino 111, 80131 Napoli, Italy.

Country: United States

AGENCY: United States NEI

GRANT: 1R01EY015136-01

ACRONYM: EY

MEDLINETA: J Virol

REFSOURCE:

DATABASENAME:

ACCESSION NUMBER:

Number Hits: 0

Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors Related Publications

• AAV-mediated gene transfer for retinal diseases.
• Activity monitoring in heart failure patients with cardiac resynchronization therapy.
• Biochemical, Pathological, and Skeletal Improvement of Mucopolysaccharidosis VI After Gene Transfer to Liver but Not to Muscle.
• Comparison of Brain Natriuretic Peptide Plasma Levels Versus Logistic EuroSCORE in Predicting In-Hospital and Late Postoperative Mortality in Patients Undergoing Aortic Valve Replacement for Symptomatic Aortic Stenosis.
• Constitutive and AP20187-induced Ret activation in photoreceptors does not protect from light-induced damage.
• Coronary Sinus Visualization by 3-dimensional Real-time Echocardiography.
• Filamin A is mutated in X-linked chronic idiopathic intestinal pseudo-obstruction with central nervous system involvement.
• Hormone-dependent nuclear export of estradiol receptor and DNA synthesis in breast cancer cells.
• In vitro effect of alpha-tocopherol on lysophosphatidylcholine-induced lens damage.
• Influence of left atrial and ventricular volumes on the relation between mitral valve annulus and coronary sinus.
• Inhibition of Estradiol Receptor/Src Association and Cell Growth by an Estradiol Receptor {alpha} Tyrosine-Phosphorylated Peptide.
• Inhibition of the SH3 domain-mediated binding of Src to the androgen receptor and its effect on tumor growth.
• Integrating signals between cAMP and MAPK pathways in breast cancer.
• Latent and potential coeliac disease.
• Long-term outcome in diabetic heart failure patients treated with cardiac resynchronization therapy.
• Mankind adaptation and present human health.
• Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors.
• Preferential silencing of a common dominant rhodopsin mutation does not inhibit retinal degeneration in a transgenic model.
• Transglutaminase 1 deficiency and corneocyte collapse: an indication for targeted molecular screening in autosomal recessive congenital ichthyosis.
• Versatility of AAV vectors for retinal gene transfer.
• [Not Available.]
• [Not Available.]