Biotech Company Editas Medicine is intending to reverse blindness by genetically editing human #genes. If they succeed in the enterprise, this company would become the first laboratory in the world to genetically edit the genes of patients suffering from leber congenital amaurosis, which is a blinding disorder inherited and appearing during the first months of life. Generally, only one copy of the affected gene is necessary to cause the disorder.
Leber congenital amaurosis
This is a disorder of the eyes that affects the retina- a tissue situated in the back of the eye which detects light and color. The symptoms appear in the infancy. Although stable at first, it may worsen gradually over time; leading to complete blindness... this disorder is also connected to sensitiveness to light (photophobia), farsightedness (hyperopia) and involuntary movements of the eye (hyperopia), among others.
What causes Leber Congenital Amaurosis?
The congenital condition is produced by a defect in a gene which instructs the production of an essential protein for vision. The disorder usually stems from an autosomal recessive pattern of inheritance, meaning that copies of both genes in a cell mutate. Parents of a person with the disorder each carry one copy of the mutated gene. In autosomal dominant inheritance, one copy of the affected gene is sufficient to create the disorder. In some cases, the disorder may originate from new mutations and affect people who have no history of the condition in their family.
The scientists at this laboratory believe that the mutation of DNA in patients can be fixed with the editing technology of Crispr (Clustered regularly interpaced short palindromic repeats), which is a technology that enables geneticists to manipúlate parts of the genome by removing adding or altering parts of the DNA sequence. This technology is in the present the most effective method for the manipulation of DNA.
The company Editas Medicine is planning to star the test of the technology in blind patients in 2017. This would mark the first time that the technology is used in humans. Although gene editing is currently banned in the US, the company hopes to obtain the permission from the health administration.