Controversial 3D. Probably none of you knows about Leber congenital amaurosis type 10, congenital blindness that affects 3 out of 100,000 children, condemning them to blindness for life. The only possible treatment is through gene therapy. 1/n
Thanks to Crispr-CAS9 technology we MODIFY the defective gene inside the patient eye ( they directly inject the treatment into the eye). Before CRISPR the treatment was based on Adenovirus carrying the correct form of the gene
In both cases, we MODIFY the patient DNA and that's the only way (today) to treat this disease. LCA10 is not the only genetic disease affecting kids unfortunately the list is long and the variety of diseases is terrifying.
Now think about a parent with a kid with a genetic disease, he/she would try and do anything to save him/her. ANYTHING and they would take some RISKS, gene therapy was a risk at the beginning, and any new experimental treatment shares a percentage of risk. 4/n
Now we come to #COVID19 and #vaccines. The new RNA vaccines are a HUGE scientific breakthrough, that may open new amazing therapeutic paths. And like ANY other human activity share a risk.