18255951. TREATMENT OF CONGENITAL STATIONARY NIGHT BLINDNESS USING GENE THERAPY simplified abstract (CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE)
Contents
- 1 TREATMENT OF CONGENITAL STATIONARY NIGHT BLINDNESS USING GENE THERAPY
- 1.1 Organization Name
- 1.2 Inventor(s)
- 1.3 TREATMENT OF CONGENITAL STATIONARY NIGHT BLINDNESS USING GENE THERAPY - 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 Unanswered Questions
- 1.11 Original Abstract Submitted
TREATMENT OF CONGENITAL STATIONARY NIGHT BLINDNESS USING GENE THERAPY
Organization Name
CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE
Inventor(s)
José-Alain Sahel of Paris (FR)
TREATMENT OF CONGENITAL STATIONARY NIGHT BLINDNESS USING GENE THERAPY - A simplified explanation of the abstract
This abstract first appeared for US patent application 18255951 titled 'TREATMENT OF CONGENITAL STATIONARY NIGHT BLINDNESS USING GENE THERAPY
Simplified Explanation
The present invention involves an expression cassette that allows for the expression of a functional LRIT3 protein in mammal eyes. This cassette is inserted into an expression vector, preferably an adeno-associated virus (AAV). Additionally, the invention includes a recombinant AAV vector carrying a nucleic acid sequence encoding a normal LRIT3 gene or fragment under the control of regulatory sequences that express the gene product in ocular cells. A pharmaceutical composition comprising this recombinant AAV vector can be used for the treatment of congenital stationary night blindness.
- Expression cassette for LRIT3 protein expression in mammal eyes
- Inserted in an AAV vector
- Recombinant AAV vector carrying LRIT3 gene for ocular cell expression
- Treatment of congenital stationary night blindness
Potential Applications
The technology can be applied in gene therapy for the treatment of congenital stationary night blindness.
Problems Solved
This technology addresses the issue of impaired night vision in individuals with congenital stationary night blindness.
Benefits
- Improved vision in low light conditions - Potential for enhanced quality of life for affected individuals
Potential Commercial Applications
- Gene therapy for ocular disorders - Development of targeted treatments for genetic eye conditions
Possible Prior Art
Prior research may exist on gene therapy for ocular disorders or the use of AAV vectors for gene delivery in the eye.
Unanswered Questions
How effective is the treatment in the long term?
The article does not provide information on the long-term effectiveness of the gene therapy treatment for congenital stationary night blindness.
Are there any potential side effects of using AAV vectors for ocular gene therapy?
The potential risks or side effects associated with the use of AAV vectors for gene delivery in the eye are not discussed in the article.
Original Abstract Submitted
The present invention relates to an expression cassette allowing expression of a functional LRIT3 protein in mammal eyes; said expression cassette is inserted in an expression vector, preferably an adeno-associated virus (AAV); accordingly, the present invention further relates to a recombinant adeno-associated virus (AAV) vector carrying a nucleic acid sequence encoding a normal LRIT3 gene, or fragment thereof, under the control of regulatory sequences which express the product of the gene in the ocular cells, a pharmaceutically acceptable composition comprising such a recombinant AAV vector and to its use for the treatment of congenital stationary night blindness
