Scientists Jennifer Doudna from UC Berkeley recently was awarded the Nobel Prize together with her partner Emmanuelle Charpentier. CRISPR has been a buzz for a while now and it has had to go through a lot of controversy about whether or not it is ethical to use gene therapy on humans. One of many obstacles that had to be surpassed was when Chinese scientist He Jiankui used their technology to create what many scientists know as the “CRISPR babies”. He Jiankui used gene editing technology (CRISPR) in order to edit the genes of two twin girls, with the main goal being to make them resistant to HIV.
CRISPR has now been put into many other uses that are widely accepted by the scientific community. A man named Paddy Doherty who had a history with the disease ATTR (amyloidosis), his father had passed from that same disease. Once Doherty reached age 65 he started to suffer from symptoms of ATTR. This is when Doherty decided to volunteer for clinical trials which tested gene therapy in hope of limiting the damage of his disease. After Doherty underwent this treatment he claimed to feel back to normal after just 2 days with most symptoms disappearing.
CRISPR has also been undergoing clinical trials for other diseases like sickle cell disease. The way CRISPR works involves a RNA guide which guides Cas9 (Cas9 is a DNA binding protein complex which cuts off target sequences of DNA) to a target DNA sequence which is the part of the genome that it is meant to cut. Once it binds to the target sequence it cuts it which causes the targeted gene to be shut off (gene causing the symptoms for the disease). For example in sickle cell disease, the sequence targeted is a mutation in the B-globin, once the sequence of the mutation is found, it is targeted to be cut off.
Many more advancements need to be made before CRISPR is released to the public but these advancements are a great sign as to what is to come. There is definitely a buzz of excitement in the medical field surrounding CRISPR even after all the controversy it has been through.