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Gene Therapy

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by

Riya Qasimi

on 9 October 2013

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Transcript of Gene Therapy

Gene Therapy
Process of replacing mutated gene that causes diseases with healthy copy of the gene to fix problem

Inactivating or "knocking out" a mutated gene that is functioning improperly
Detecting a disease
What is it, anyway?

Using
genetic screening
, scientists detect whether someone can develop disease in future and able to trace back to gene responsible
Done by cutting piece of DNA and analyzing gene

Once gene located and defined as cause of disease, scientists start gene therapy
A four-year old girl became the first gene therapy patient on September 14, 1990
ADA deficiency; white blood cells taken from her, and normal genes for making ADA were inserted
The corrected cells were reinjected into her
Receives injections of T-cells every two months
Dr Anderson helped this landmark clinical trial happen
Dr. Rosenberg

Leading cancer researcher and surgeon
Chief of Surgery
Professor of Surgery at George Washington University

Pioneered development of gene therapy and was first to successfully insert foreign genes into humans and to conduct clinical studies of gene therapy of cancer

Risks to Society
Many ethical issues surrounding gene therapy
Ex. Who will decide which traits are normal and which ones are disabilities
If gene therapy is expensive, will it only be available to the wealthy?
Gene therapy could potentially lead to discrimination of others
Where do we draw the line when using gene therapy?
Ex. to advance traits such as height, intellect and abilities?

Risks to Science
Chance of inducing a tumor (insertional mutagenesis) Ex. Some children developed leukemia
Germline gene therapy (targets sex cells which will be passed on to generations) is controversial
Ex. it would remove the chance of the genetic disorder, BUT may have unknown affect development of the fetus in the future, also since this affects the unborn, they have no choice in choosing the treatment

Benefits to Science
Gene-based treatments can attack existing cancer at the molecular level, eliminating the need for drugs, radiation or surgery
Injecting cancer cells with special genes that make the tumor more receptive to the effects of anti-cancer drugs

Who Benefits the Most?
Currently only available to public through participation in clinical trial
Only those willing to take part and take risks will benefit
Beneficial to those to have conditions stemming from genetic roots
Best candidates for gene therapy are the so-called "single-gene" disorders - caused by mutations in only one gene
Many disorders or medical conditions might be treated using gene therapy, but others may not be suitable for this approach

Jenny Tang
Zainab Qasimi

William French Anderson
Contribution & Use
used for treating medical conditions that have genetic origins
replaces a faulty gene or adds new gene in an attempt to cure disease or improve your body’s ability to fight diseases
holds promise for treating a wide range of diseases, including cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS
First Patient
American physician, geneticist, molecular biologist
Considered first person to succeed in gene therapy
"Father of Gene Therapy"
Sentenced 14 years in prison
"Great loss to science"
Human Genome Project
sequence of chemical base pairs which make up human DNA
Advances in human genome project opened new doors for exploration of gene therapy
Stem Cells
can differentiate into specialized cells (any type of cell)
used as vectors to deliver genes intended to correct hereditary disease
used in first procedure of gene therapy
Genetic Screening
among newest and most sophisticated techniques used to test for genetic disorders
direct examination of DNA itself
done before birth
Used in 1993, to show that Andrew Gobea has SCID
Benefit to Society
Identifying cancer susceptibility in individuals or families can have major role in preventing disease before it occurs
Pioneers of Gene Therapy
In 1989,
Dr. Anderson

and

Dr. Eli Gilboa

worked with

Dr. Steven Rosenberg
to test the safety and effectiveness of gene therapy process in cancer patients
Anderson and Dr. Eli Gilboa developed retroviral vectors that provided efficient gene transfer
This trial paved the way for dozens of gene-therapy efforts
Full transcript