In early 2023, a 13-year-old girl named Alyssa was cured of her previously ‘incurable’ T-cell acute lymphoblastic leukemia through the use of base editing. Read more: https://stevepapermaster.com/base-editing-revolutionizing-precision-genome-editing

1. BASE
EDITING
Steve G
Papermaster
Nano
in Action

2. STEVE G PAPERMASTER NANO
2
BASE EDITING IN ACTION
In early 2023, a 13-year-old girl
named Alyssa was cured of her
previously ‘incurable’ T-cell acute
lymphoblastic leukemia through the
use of base editing. A bone marrow
transplant and chemotherapy had
failed to treat the cancer, so Alyssa
took part in a clinical trial at Great
Ormond Street Hospital in the UK.
HOW THE TREATMENT
WORKED
The treatment was a type of CAR-T
cell therapy but differed in that it
used donor stem cells rather than
Alyssa’s own.
Doctors took healthy T-cells from a
donor and set about editing them.
They did this in three stages:
First Base Edit: This disabled the
targeting mechanism of the T-cells
so they couldn’t attack Alyssa’s body
Second Base Edit: This removed
CD7, a chemical marking present on
all T-cells
Third Base Edit: This altered the
DNA so that the cells could resist
being killed by chemotherapy
The last stage was to instruct the
T-cells to hunt down and destroy any
cells with the CD7 marking on them,
obliterating all of Alyssa’s T-cells, the
cancerous ones included.
This case highlights the incredible
promise base editing holds for the
future of genetics.

3. STEVE G PAPERMASTER NANO 3
THELASTSTAGEWASTO
INSTRUCTTHET-CELLS
TOHUNTDOWNAND
DESTROYANYCELLSWITH
THECD7MARKINGON
THEM,OBLITERATING
ALLOFALYSSA’ST-CELLS,
THECANCEROUSONES
INCLUDED.

4. To find out more
about this topic, visit
the blog of Steve G
Papermaster, Nano
chairman and CEO.