18167598. Compositions and Methods of Treating Ocular Diseases simplified abstract (THE REGENTS OF THE UNIVERSITY OF CALIFORNIA)
Contents
- 1 Compositions and Methods of Treating Ocular Diseases
- 1.1 Organization Name
- 1.2 Inventor(s)
- 1.3 Compositions and Methods of Treating Ocular Diseases - A simplified explanation of the abstract
- 1.4 Simplified Explanation
- 1.5 Potential Applications
- 1.6 Problems Solved
- 1.7 Benefits
- 1.8 Potential Commercial Applications
- 1.9 Possible Prior Art
- 1.10 How does this technology compare to other methods of gene delivery to retinal cells?
- 1.11 What are the potential long-term effects of using altered AAV virions for gene therapy in retinal cells?
- 1.12 Original Abstract Submitted
Compositions and Methods of Treating Ocular Diseases
Organization Name
THE REGENTS OF THE UNIVERSITY OF CALIFORNIA
Inventor(s)
David V. Schaffer of Danville CA (US)
Leah C. Byrne of San Francisco CA (US)
Timothy P. Day of Berkeley CA (US)
John G. Flannery of Berkeley CA (US)
Compositions and Methods of Treating Ocular Diseases - A simplified explanation of the abstract
This abstract first appeared for US patent application 18167598 titled 'Compositions and Methods of Treating Ocular Diseases
Simplified Explanation
The present disclosure provides recombinant adeno-associated virus (AAV) virions with altered capsid protein, where the recombinant AAV (rAAV) virions exhibit greater ability to cross barriers between intravitreal fluid and retinal cells, and thus greater infectivity of a retinal cell compared to wild-type AAV, and where the rAAV virions comprise a heterologous nucleic acid. The present disclosure provides methods of delivering a gene product to a retinal cell in an individual.
- Recombinant AAV virions with altered capsid protein
- Greater ability to cross barriers between intravitreal fluid and retinal cells
- Increased infectivity of a retinal cell compared to wild-type AAV
- Virions comprise a heterologous nucleic acid
- Methods for delivering a gene product to a retinal cell in an individual
Potential Applications
This technology could be applied in gene therapy treatments for retinal diseases, such as retinitis pigmentosa or age-related macular degeneration.
Problems Solved
This technology addresses the challenge of efficiently delivering gene products to retinal cells, which can be hindered by barriers between the intravitreal fluid and the cells.
Benefits
The altered AAV virions offer increased infectivity, potentially leading to more effective gene therapy treatments for retinal diseases.
Potential Commercial Applications
This technology could be valuable in the development of gene therapy products for retinal diseases, potentially improving treatment outcomes for patients.
Possible Prior Art
Previous research has focused on optimizing AAV vectors for gene therapy applications, but this specific approach of altering capsid proteins for enhanced infectivity may be novel.
Unanswered Questions
How does this technology compare to other methods of gene delivery to retinal cells?
This article does not provide a direct comparison to other gene delivery methods, leaving it unclear how this technology stands out in the field.
What are the potential long-term effects of using altered AAV virions for gene therapy in retinal cells?
The article does not address the potential long-term effects of using these altered AAV virions, leaving a gap in understanding the safety and efficacy of this approach over time.
Original Abstract Submitted
The present disclosure provides recombinant adeno-associated virus (AAV) virions with altered capsid protein, where the recombinant AAV (rAAV) virions exhibit greater ability to cross barriers between intravitreal fluid and retinal cells, and thus greater infectivity of a retinal cell compared to wild-type AAV, and where the rAAV virions comprise a heterologous nucleic acid. The present disclosure provides methods of delivering a gene product to a retinal cell in an individual.
