Wednesday, July 10, 2024

 The article “Gene Therapy Improves Vision in People With Inherited Blindness” discusses the significant breakthrough in the field of genetics in which gene therapy has shown positive results in improving visitation for people who have suffered from inherited blindness in the early stages of the clinical trial. About 14 people with Leber Congenital Amaurosis (LCA) which is a rare genetic disease which both parents must carry the defective gene for the condition in order to pass it onto their children will lead to babies to lose some or even all of their sight from birth. In the clinical trial a lot of people had improvement in their vision with a signal injection. Leber Congenital Amaurosis affects 2 to 3 out of 100,00 babies each year; mostly all babies are born blind or some can begin to lose their eyesight at 6 months of age from being born. n. Leber Congenital Amaurosis is primarily caused by a mutation in the centrosomal protein 290 gene which causes a malfunction in the rods and cones of the retina leading to defect incoming visual signals and processing of signals that are received by the brain which this gene is the leading cause of inherited blindness in the first decade of life. This led to come up with a genetic solution which the CRISPR-Cas9, which is a gene editing tool which acts as a pair of gene level scission which can cut away the portion of the mutated gene leading the CRISPR-based gene medicine directly injected inside the body.The first injection CRISPR-Case for treatment of LCA was given in early 2020 at Casey Eye Institute at Oregon Health & Science University in Portland which the results showed 11 participants had improvement in the one measure vision, 6 participants stated they had improved vision-related quality of life and 4 participants stated that improvement in visual activity by stating they could see certain letters or shapes in a particular chart. 

The CRISPR-based gene has demonstrated clinical meaningful outcomes that have primarily improved vision outcomes in people's life.The treatment had involved 12 adults and two children which were monitored every three months for one year and then less often in two years.As of now, future research is needed in order to determine the amount of dosage required to see how much vision can be improved with people Leber Congenital Amaurosis as well as if it could benefit others with treatment and people who have similar genetic disorders with vision and see if there vision can be improved or with retinal disease. As of November 2022 the clinical trial has been in November 2022 to the needs of commercial partners in order to collaborated to development more experiential therapy for the treatment of Leber Congenital Amaurosis.As of now researchers are primarily trying to find to work with commercial partners and to collaborate with others and try to conduct large scale trials.Overall, future research is needed in order to primarily advance the treatment overall. 

 Howard, Jacqueline. “Experimental Gene Therapy Restores Some Vision in Patients with Inherited Blindness.” CNN, Cable News Network, 6 May 2024,

Illustration of eye and DNA

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