CRISPR TECHNOLOGY used to treat cancer cells. CAR T-cell therapy.

1. CRISPR TECHNOLOGY
TREATING IN CANCER
CELLS
NITHIYA NANTHINI V
3rd B.Sc.BIOTECHNOLOGY

2. • CRISPR ( Clustered regularly interspaced short palindromic
repeats) is a technology that research scientists use to
selectively modify the DNA of living microorganisms.
• CRISPR was adapted for use in the laboratory from naturally
occuring genome editing systems f0und in bacteria
WHAT IS CRISPR
TECHNOLOGY

3. CAR T CELL
THERAPY
• CRISPR Therapeutics’ gene-editing approach to cancer uses
allogeneic CAR T cells.
• Chimeric antigen receptors (CAR) are genetically encoded artificial
fusion molecules that can reprogram the specificity of peripheral
blood polyclonal T-cells against a selected cell surface target
• CARs are genetically engineered receptors that combine the
specific binding domains from a tumor targeting antibody with T cell
signaling domains to allow specifically targeted antibody redirected
T cell activation.

4. HISTORY
• In 1987, Israeli immunologist Zelig Eshhar created the first chimeric antigen
receptor (CAR), an engineered receptor for T cells to combat cancer.
• In 2010, the first successful CAR-T cell therapy for advanced follicular
lymphoma was reported by Dr. Steven Rosenberg's lab at the NCI.
• On August 30, 2017, tisagenlecleucel (Kymriah) became the first FDA-
approved CAR T-cell immunotherapy for children and young adults under 25
with acute lymphoblastic leukemia (ALL).
• This therapy represents a significant milestone in cancer treatment.

5. CANCER TREATMENT
• CAR T cell therapy involves modifying a patient's own T cells (a type of
immune cell) to target and destroy cancer cells.
• A patient's T cells are extracted and genetically engineered to express a
CAR, which is a synthetic receptor that recognizes specific proteins on cancer
cells.
• Once modified, these CAR-T cells are multiplied in the lab and then infused
back into the patient's body.
• The CAR-T cells are designed to identify and attack cancer cells bearing the
targeted protein, effectively destroying them.

6. • CAR-T cell therapy has shown remarkable success in treating certain blood cancers,
such as acute lymphoblastic leukemia (ALL) and non-Hodgkin lymphoma.
• It has been particularly effective in cases where conventional treatments have failed or
when the cancer has relapsed.
• While CAR-T cell therapy has shown great promise, it can have significant side effects,
and its use is currently limited to specific cancer types and patients.

8. CAR T CELL DESIGN
• CAR T cell therapy uses a special protein called CAR to help T-Cells
recognize and destroy cancer cells.
• The CAR is not naturally in T-cells ,so its added using a gene, often
delivered by engineered viruses.
• The gene makes the CAR protein , which stays on the T cells surface.
• Patients T cells are collected , the CAR is added and the T cells are
activated and multiplied in the lab.

9. • T-cells can be modified to become better at killing tumors.
• CAR T-cells are frozen, transported back, and injected into the
patient.
• An alternative idea is to create "off-the-shelf" CAR T-cells from
donor cells.
• These donor T-cells are modified to reduce side effects.
• This approach could speed up treatment and lower costs in
fighting certain cancers.

10. ADVANTAGE
• CAR T-cell therapy is a quick treatment, often just two weeks, without harsh
chemotherapy, approved for patients unlikely to be cured by transplants or
those who relapse after.
• Clinical trials demonstrate CAR T-cells achieve long remissions, benefiting
patients even after multiple prior treatments.
• These living CAR T-cells can persist for years, recognizing and attacking cancer
cells during relapses.
• For example, 42% of adult lymphoma patients on CD19 CAR T-cell therapy
remained in remission after 15 months.
• Childhood acute lymphoblastic leukemia patients, including aggressive cases,
saw two-thirds in remission after six months.

11. SIDE EFFECTS
Cytokine release syndrome (CRS):
As CAR T cells multiply, they can release large amounts of chemicals called
cytokines into the blood, which can ramp up the immune system. Serious
side effects from this release can include:
• High fever and chills
• Trouble breathing
• Severe nausea, vomiting, and/or diarrhea
• Feeling dizzy or lightheaded
• Headaches
• Fast heartbeat
• Feeling very tired
• Muscle and/or joint pain

12. Nervous system problems:
This treatment can sometimes have serious effects on the nervous system, which
can result in symptoms such as:
• Headaches
• Changes in consciousness
• Confusion or agitation
• Seizures
• Shaking or twitching (tremors)
• Trouble speaking and understanding
• Loss of balance

13. THANK YOU