Retinal gene delivery by adeno-associated virus (AAV) vectors: Strategies and applications.
- BiologyEuropean journal of pharmaceutics and biopharmaceutics : official journal of Arbeitsgemeinschaft fur Pharmazeutische Verfahrenstechnik e.V
Optimized Subretinal Injection Technique for Gene Therapy Approaches.
- BiologyMethods in molecular biology
The method described here aims to provide the details relevant to perform a transscleral pars plana virus-mediated gene transfer to achieve an optimized therapeutic effect in the small mouse eye.
Barriers for retinal gene therapy: Separating fact from fiction
- Biology, MedicineVision Research
Adeno-associated virus mediated gene therapy for retinal degenerative diseases.
- Biology, MedicineMethods in molecular biology
This chapter focuses on AAV-mediated specific gene therapy for inherited retinal degenerative diseases, describing the disease targets, the preclinical studies in animal models and the recent success of the LCA-RPE65 clinical trials.
Vector platforms for gene therapy of inherited retinopathies
- BiologyProgress in Retinal and Eye Research
Intraocular route of AAV2 vector administration defines humoral immune response and therapeutic potential
- BiologyMolecular vision
Findings have important clinical implications for the design of AAV-mediated ocular gene transfer for retinal diseases, particularly if both eyes require sequential treatment.
Expanding AAV cargo capacity for gene therapy of Stargardt disease
Dual AAV vectors are more efficient than oversize vectors, both in vitro and in the retina, and are an attractive strategy for gene therapy of STGD as well as other retinal diseases that require the delivery of large genes.
Ocular gene therapy: current progress and future prospects.
- Biology, MedicineTrends in molecular medicine
AAV and compacted DNA nanoparticles for the treatment of retinal disorders: challenges and future prospects.
- BiologyInvestigative ophthalmology & visual science
Of particular interest are various requirements for successful clinical application of these technologies including vector trafficking, delivery, specific gene expression, and treatment safety, and tolerance.
Delivery of Genetic Information: Viral Vector and Nonviral Vector Gene Therapies
- BiologyInternational ophthalmology clinics
This chapter will review current and prospective methodologies of gene delivery vehicles, along with retinal applications and the current challenges faced.
SHOWING 1-10 OF 115 REFERENCES
Adeno-associated viral vectors for retinal gene transfer
- BiologyProgress in Retinal and Eye Research
Pseudotyped AAV vectors for constitutive and regulated gene expression in the eye
- BiologyVision Research
Adeno-associated virus-vectored gene therapy for retinal disease.
- BiologyHuman gene therapy
Adeno-associated viral vectors for retinal gene transfer and treatment of retinal diseases.
- BiologyCurrent gene therapy
Recombinant AAV (rAAV) vectors are versatile gene transfer tools in that capsid proteins from dozens of AAV serotypes can be easily interchanged, resulting in the creation of recombinant vectors with unique transduction properties.
Gene delivery to the eye using adeno-associated viral vectors.
AAV-mediated gene transfer for retinal diseases
- Biology, MedicineExpert opinion on biological therapy
Years of successful rAAV-mediated gene transfer to the retina have resulted in restoration of vision in dogs affected with congenital blindness, paving the way to the first attempts at treating inherited retinal diseases in humans with rAAVs.
Delivery of Adeno-Associated Virus Vectors to the Fetal Retina: Impact of Viral Capsid Proteins on Retinal Neuronal Progenitor Transduction
- BiologyJournal of Virology
The neural progenitor transduction patterns of adeno-associated virus vectors following delivery to the developing retina resulted in efficient and stable transduction of retinal progenitors and provided a safe delivery vehicle for potential therapeutic applications and a means of assessing the mechanisms of retina development and disease.
Gene therapy progress and prospects: the eye
- Biology, MedicineGene Therapy
Gene therapy also offers a potentially powerful approach to the treatment of complex acquired disorders such as those involving angiogenesis, inflammation and degeneration, by the targeted sustained intraocular delivery of therapeutic proteins.
Gene transfer into the mouse retina mediated by an adeno-associated viral vector.
- BiologyHuman molecular genetics
This is the first report showing that AAV is capable of transducing photoreceptor cells and supports the use of this vector system for gene therapy of retinal diseases such as RP.
Pharmacologically regulated gene expression in the retina following transduction with viral vectors
- BiologyGene Therapy
Upon administration of doxycycline, reversible expression of green fluorescent protein in the retinal pigment epithelium as well as modulation of human growth hormone produced in the retina and secreted in the blood stream are demonstrated.