GENE THERAPY

Zosia Wojciechowska, Aniela McGrath,

Izabella Kawycz
 WHAT IS GENE THERAPY?
● An experimental form of treatment that uses
gene transfer of genetic material into the cell of a
patient
● Replaces a faulty gene or adds a new gene (or
even disactivates a particular sequence)
● Holds promise for treating a wide range of
diseases

METHODS:

1. Microinjection of a gene into a target cell

2. Electroporation
3. Through a viral vector
 VECTOR TYPES

*engineered viruses that can deliver the material by infecting the cell

● Adeno-associated vectors AAVS

● Retroviruses Enters through
endocytosis
● Adenoviruses
● Liposomes

RETROVIRUS:
1. An RNA version of the wanted allele for the
faulty gene is synthesised and inserted into
the retrovirus.
2. Retrovirus makes a DNA transcript of the
RNA genome.
3. It is inserted into the host cell.
CRISPR CAS9 Genome Editing
Emmanuelle Charpentier and Jennifer Doudna
Feng Zhang

https://www.youtube.com/watch?v=
2pp17E4E-O8
 APPLICATIONS

1. SCID
● Bone marrow transplant

2. CYSTIC FIBROSIS
● Small molecule treatment, cloning CFTR

3. EYESIGHT
● Ophthalmology ex. Achromatopsia
POTENTIAL RISKS

This technique of viral vectors presents the following risks:

● Unwanted immune system reaction.

● The virus can target the wrong cells.
● It can lead to an infection caused by the virus.
● There’s a possibility of causing a tumor.
GERM/STEM CELLS APPLICATION
● Promise of more
effective therapy

● Still in progress
● Has drawbacks
● Germline alterations
controversy
ETHICAL DISCUSSION NEW DISCOVERIES
● Future promise, technology development ● heteroduplex oligonucleotide (HDO)
● viral vector manufacturing improvement
● FDA approval
BIBLIOGRAPHY
Gene therapy intro: www.mayoclinic.org https://www.sciencedirect.com/science/article/abs/pii/S0378111913004344

SCID: www.childrenshospital.org

Cystic Fibrosis:

https://www.liebertpub.com/doi/abs/10.1089/hum.2015.027 https://web.williams.edu/imput/synapse/pages/IIIB4.htm

Retrovirus: www.khanacademy.org

Gene therapy advances. discoveries: www.ncbi.nlm.nih.gov https://academic.oup.com/nsr/article/6/3/402/5304991

New discoveries: www.sciencedaily.com

Adenovirus: www.chop.edu

About the CRISPR Nobel controversy

https://www.science.org/content/article/crispr-revolutionary-genetic-scissors-honored-chemistry-nobel