Abstract Presenter: Henna Butt, MD, hematology/oncology fellow, Children’s National Hospital

Abstract Title: Comparative Analysis of CRISPR-Cas9, Lentiviral Transduction, and Base Editing for Sickle Cell Disease Therapy in a Murine Model

Session: Welcome, Awards & Best Abstracts, 8:15 – 10 a.m. on Feb. 14, Ballroom B

Presentation Time: 8:45 – 9 a.m.

What is your presentation/abstract about?

“My abstract is looking at three types of gene therapies for sickle cell disease and comparing them in both ex vivo studies and in a murine model. The goal was to study how all three approaches — CRISPR-Cas9 editing, lentiviral transduction, and base editing — differ in terms of toxicity, engraftment, differentiation, and in a competitive transplantation setting in a murine model. It is the first study to look at all three approaches in a single murine model to really delve into the competitive advantage of each.”

How did you first become interested in this topic?

“I became interested in hematopoietic stem cell transplantation when I did an away rotation at Children’s National Hospital during my residency. From there, my interests have grown to include gene therapy and specifically for hemoglobinopathies.“

What are you working on next related to this topic?

“We are now working on a secondary transplantation model to study the long-term effects of all three approaches.”

How do you hope your work influences the field?

“With the two current therapies with U.S. Food and Drug Administration (FDA) approval and many more on the way, it will become harder for both patients and doctors to choose the best option for each individual. I am hoping this work will lead to further studies to really tease out which option is best for each individual patient.”

What excites you most about transplantation and cellular and gene therapy today?

“After so many years with so little research on sickle cell disease, I am most excited about how rapidly this field is now evolving. I am excited to see what will come in even the next five years.” 

Is there anything else you would like Tandem Meetings attendees to know about your abstract?

“This work is very exciting to me, and I am honored to be able to present it at the Tandem Meetings and very grateful to my mentors, John Tisdale, MD, and Selami Demirci, PhD, for making this work possible.”