Gene Therapy

Characters:

Student 1(pau)

Doctor (penille)

Nurse (erica)

Patient(rale)

Script

Intro:

Student 1:

Have you ever wonder how you possess all of your unique physical features? Would you believe if I say
that it is all because of a double strand inside your body cells? Yes! You heard it right! It is all because of
the double strand inside your body cells called DNA! This stucture is very important as it carries the
genes or the blueprint of organisms such as plants and animals, including us! And do you know that if
something wrong happens in your DNA, it would cause illnesses and diseases? But, do not worry! Gene
therapy is to the rescue! For today's video, we are going to see how gene therapy works, and also
determine the process of gene cloning! So, stay tune and be with us till the end!

Gene therapy:

Patient: Hi! I'm mathew and let me share you my experience with gene therapy. At a very young age, I
was diagnosed for having

Leber Congenital Amaurosis). Leber congenital amaurosis is a genetic disorder where it causes blindness
that begins from infancy. And with the help of the doctors from Ladrex Hospital, I was able to see all
things around me!

Doctor: That's right! Mathew was our first patient who regain vision through gene therapy. Gene
therapy is a medical treatment where we correct a mutated gene or a gene that has unsual or wrong
pattern causing disorders and illnesses. And one example of this was the Leber congenital amaurosis a
genetic disorder where there is a deffective RPE65 gene causing retina to malfunction and making us
unable to see. Let my assistant , show you the process we've followed to cure the genetic disorder of
Mathew.

Assistant: To cure Leber congenital amaurosis, first, we obtain a normal RPE65 gene and place it on a
vector. A vector is a like a messenger delivering a normal functioning gene into the patient's cell
genome. A vector is usually a virus due to its great function in infecting and sneaking up in cells. Next,
the gene containing vector will be injected into the patient's eyeball under retina between retinal
pigment ephithellium and choroid entering the cells of the retina. Lastly, the vector will release the gene
into the retina cells and will produce the RPE65 protein needed by the eyes for vision.

Doctor: The Gene therapy was a success and restored the vision of Matthew.

Patient: Yes! I was able to perceive all the colors from our surroundings and it was the best! Thanks to
gene Therapy!

Doctor: Gene therapy can also use or treat some conditions like Bubble Boy Disease, Sickle cell disease,
Cancer, Cystic fibrosis, heart disease, Diabetes, Hemophilia and AIDS.

Patient: Oh wow! That's great! There's a lot of gene therapy that will help a lot sick patients like me!