Research that could change lives

For her ARP, Kaelan Patel jumped into medical research that could be used to treat sickle cell anemia, and shared with us what she learned.

Your topic is currently being researched at a pretty high level: a gene therapy called CRISPR/Cas9.
Tell us about it.

It’s an acronym for Clustered Regularly Interspaced Short Palindromic Repeats, which were originally found in bacterial DNA. Bacteria used these repeats as a way to fight off infection, and doctors experimented to see if they could manipulate human DNA to fight off invading illnesses as a model after the bacterial CRISPRs. They discovered that they could create CRISPRs that could be guided to exact locations of genes by R

NA molecules to attack mutated sections of the DNA that cause life altering ailments such as sickle cell anemia (SCA). Then, the Cas9 protein in human DNA cuts the mutated section of DNA so a donor DNA template that has the corrected sequence to heal is inserted. I have decided to focus my research on SCA because it is a devastating and painful disease. Currently, patients suffering from SCA only have three options for treatment, which include a drug called hydroxyurea, blood transfusion, and bone marrow transplant.

You worked with mentors locally at Children’s Hospital of Michigan, and at UC Berkeley. What were these experiences like?
I worked both with Dr. [Michael] Callaghan from the Pediatric Hematology Clinic at Children’s Hospital of Michigan, and Dr. [Jeffrey] Corn, from UC Berkeley. Dr. Callaghan offers the perspective of using current treatments, and I had the opportunity to interview all of his patients over the summer to talk about their treatments, as well as how SCA affects their daily lives. I learned none of the patients had heard of CRISPR/Cas9. At Dr. Corn’s lab, I experienced the equipment and processes used to collect data that will prove CRISPR is safe enough to start human clinical trials. They are getting close to the numbers they need to earn approval.

What are some of the other topics you considered?
Stem cell therapy to treat disease, and also CRISPR/Cas9’s use in treating cardiovascular disease, which has environmental factors, and high efficacy rates for current treatments. SCA is a monogenic disease with few environmental factors, and no effective treatment for patients who suffer intense pain and shortened life spans.

What is the most challenging thing about presenting this topic?
There is so much to say and only one poster board and three minutes to talk! For people to comprehend the topic, I need to define a lot of material to make it understandable to someone who is learning about it for the first time.