Literary investigation into gene therapy in the treatment of cancer by Evin Rothschild, a Pine Crest School sophomore

1. Literary Investigation on
Gene Therapy in the
Treatment of Cancer
By: Evin Rothschild

2. Background
 Radiation, chemotherapy, and surgery are widely used
treatment methods
 Severe side effects for radiation and chemotherapy
 Radiation and chemotherapy are not always effective
 Gene therapy can either work on its own or in addition
to radiation and chemotherapy
 Gene therapy can be more effective with less side
effects because it targets cancer cells rather than all
cells

3. Purpose
 To investigate safer, more effective ways in treating
cancer

4. Methods
 Many gene therapy methods used to fight cancer involve
a gene knockdown
 Gene knockdowns involve silencing the expression of the
specific targeted gene downstream in the process to
avoid upstream effects of the mutated gene

5. Knock down of Ubb gene to down
regulate Ubiquitin
 Ubitiquin has wide variety of uses
 Focus on it’s ability to help a cancer cell overcome
stresses that would normally kill the cancer cell
 Found in high amounts in a variety of cancers
 After knockdown, cell proliferation inhibited by 55% and
induced apoptotic death in many cells
 Shrinks tumors in mice so the tumors could
potentially be operated on

6. Knock down of WTAP in acute
myeloid leukemia (AML)
 WTAP is normally an oncogenic protein that stabilizes cyclin
A2
 Acts as a switch gene when partnered with Wilms Tumor 1
 Found to be overexpressed in cases of AML which causes
unregulated cell growth
 After knock down, proliferation and clonogenic survival
significantly decreased
 Increased cell apoptosis after knockdown in addition to
chemical treatment
 Tumor size was smaller by 9 times in mice with knocked down
WTAP

7. Knock down of EGFR in gastric
cancers
 Epidermal growth factor receptor- binds with EGF to
allow cell growth in normal cells
 When overactive, causes uncontrolled cell division
 Shown to be overexpressed in gastric cancer cells
 After knockdown, cell proliferation and invasion of the
cancer were reduced while cell apoptosis was induced
after the gene knockdown

8. Discussion
 Gene therapy could be a promising treatment method
for a variety of types of cancers
 Many methods are very gene specific, thus making them
cancer specific
 A form of personalized medicine- finding the cause of
the problem and stopping it there

9. Works cited
http://www.cancer.net/navigating-cancer-care/how-cancer-treated/chemotherapy/side-effects-chemotherapy)
http://www.cancer.gov/cancertopics/factsheet/Therapy/radiation
http://ghr.nlm.nih.gov/handbook/therapy/genetherapy
http://en.wikipedia.org/wiki/Ubiquitin#Functions_of_ubiquitin_modification)
Oh, Choongseob, Soonyong Park, Eun Kyung Lee, and Yung Joon Yoo. "Downregulation of Ubitiquin Level via Knockdown of
Polyubitiquin Gene Ubb as Potential Cancer Theraputic Intervention." Nature 11 Sept. 2013: 2623. Print.
"WTAP Is a Novel Oncogenic Protein in Acute Myeloid Leukemia." Leukemia 13 Jan. 2014: 1171-174. Print
http://www.ncbi.nlm.nih.gov/gene/1956
Zhen, Y., L. Guanghui, and Z. Xiefu. "Knockdown of EGFR Inhibits Growth and Invasion of Gastric Cancer Cells." Cancer Gene
Therapy 14 Nov. 2014: 491-97. Print.
http://en.wikipedia.org/wiki/Jesse_Gelsinger
http://drbcshah.com/chemotherapy/