Gene Therapy
Overview
Retinal gene therapy involves the use of adeno-associated virus (AAV) or other types of viral or nonviral vectors to deliver functional copies of genes to specific cell types within the retina. This approach has shown promise in treating a variety of inherited and non-inherited retinal diseases that cause vision loss or blindness.
AAV vectors are the most widely used delivery systems for retinal gene therapy due to their low immunogenicity, ability to transduce non-dividing cells, and long-term transgene expression. The retina's immune-privileged status further enhances the safety and efficacy of AAV-mediated gene delivery. Since AAVs have a limited capacity to carry small genes, other vectors such as lentivirus or nonviral vectors are considered for the delivery of larger genes.
Mechanism
Retinal gene therapy targets specific cell types, such as retinal pigment epithelium (RPE), photoreceptors, or retinal ganglion cells, depending on the disease being treated. The choice of vector, route of administration (intravitreal or subretinal), and promoter sequence can influence the tropism and expression of the therapeutic gene.
Successful gene therapy requires the delivered transgene to produce a functional protein that can compensate for the defective or missing protein caused by the disease. Modulation of transgene expression may be necessary to maintain long-term therapeutic benefits and avoid potential toxicity.
Challenges
Despite the progress made in retinal gene therapy, several challenges remain. These include optimizing vector design and delivery, improving the specificity and efficiency of transgene expression, and addressing potential immune responses. Researchers are also exploring novel AAV variants and targeting strategies to expand the applications of retinal gene therapy.
Overview
Retinal gene therapy involves the use of adeno-associated virus (AAV) or other types of viral or nonviral vectors to deliver functional copies of genes to specific cell types within the retina. This approach has shown promise in treating a variety of inherited and non-inherited retinal diseases that cause vision loss or blindness.
AAV vectors are the most widely used delivery systems for retinal gene therapy due to their low immunogenicity, ability to transduce non-dividing cells, and long-term transgene expression. The retina's immune-privileged status further enhances the safety and efficacy of AAV-mediated gene delivery. Since AAVs have a limited capacity to carry small genes, other vectors such as lentivirus or nonviral vectors are considered for the delivery of larger genes.
Mechanism
Retinal gene therapy targets specific cell types, such as retinal pigment epithelium (RPE), photoreceptors, or retinal ganglion cells, depending on the disease being treated. The choice of vector, route of administration (intravitreal or subretinal), and promoter sequence can influence the tropism and expression of the therapeutic gene.
Successful gene therapy requires the delivered transgene to produce a functional protein that can compensate for the defective or missing protein caused by the disease. Modulation of transgene expression may be necessary to maintain long-term therapeutic benefits and avoid potential toxicity.
Challenges
Despite the progress made in retinal gene therapy, several challenges remain. These include optimizing vector design and delivery, improving the specificity and efficiency of transgene expression, and addressing potential immune responses. Researchers are also exploring novel AAV variants and targeting strategies to expand the applications of retinal gene therapy.
Overview
Retinal gene therapy involves the use of adeno-associated virus (AAV) or other types of viral or nonviral vectors to deliver functional copies of genes to specific cell types within the retina. This approach has shown promise in treating a variety of inherited and non-inherited retinal diseases that cause vision loss or blindness.
AAV vectors are the most widely used delivery systems for retinal gene therapy due to their low immunogenicity, ability to transduce non-dividing cells, and long-term transgene expression. The retina's immune-privileged status further enhances the safety and efficacy of AAV-mediated gene delivery. Since AAVs have a limited capacity to carry small genes, other vectors such as lentivirus or nonviral vectors are considered for the delivery of larger genes.
Mechanism
Retinal gene therapy targets specific cell types, such as retinal pigment epithelium (RPE), photoreceptors, or retinal ganglion cells, depending on the disease being treated. The choice of vector, route of administration (intravitreal or subretinal), and promoter sequence can influence the tropism and expression of the therapeutic gene.
Successful gene therapy requires the delivered transgene to produce a functional protein that can compensate for the defective or missing protein caused by the disease. Modulation of transgene expression may be necessary to maintain long-term therapeutic benefits and avoid potential toxicity.
Challenges
Despite the progress made in retinal gene therapy, several challenges remain. These include optimizing vector design and delivery, improving the specificity and efficiency of transgene expression, and addressing potential immune responses. Researchers are also exploring novel AAV variants and targeting strategies to expand the applications of retinal gene therapy.