GENE THERAPY. What is gene therapy? Gene therapy is the introduction of normal genes into cells that...

GENE THERAPY

What is gene therapy?

• Gene therapy is the introduction of normal genes into cells that contain defective genes

Why gene therapy?

• To supply the cells with the correct directions to make a missing protein product

How?2 Delivery Systems

1. In vivo occurs within the organisms

2. Ex vivo occurs outside the organism

Who?The First Case

• September 14th, 1990– Ashanti DeSilva was treated for SCID

• Sever combined immunodeficiency • Gene is located on chromosome #22

– Doctors removed her white blood cells, inserted the missing gene into the WBC, and then put them back into her blood stream.

– This strengthened her immune system– Only worked for a few months

Would this be considered in vivo or ex vivo?

Vectors

Vectors are carriers. (ex. insects are considered vectors for pollen)

Vectors transport the DNA into the body or into the cells.

What was the vector in the transformation lab?

Viruses are frequently used as vectors because:

• They insert their viral DNA

into cells as a normal part of their lifecycle.

So, why not let the virus inject the good gene into a cell?

Imagine this lifecycle takingplace in the human body witha good gene. Explain whatwould happen?

General concernsThe Food and Drug Administration (FDA) has not yet approved any human gene

therapy product for sale.

Major problems with gene therapy:

2) Immune response. It reduces gene therapy effectiveness and makes repetitive rounds of gene therapy useless

3) Viral vectors . Immune and inflammatory responses, also fears that viral vector may recover disease-causing ability

5) Target. One does not have control over where the gene will be inserted into the genome

1) Short-lived. Very hard to achieve any long-term benefits

4) Multigene disorders. Disorders such as heart disease, Alzheimer's disease, arthritis, and diabetes, are caused by the combined effects of many genes.

More than 5000 patients have been treated in last ~12 years worldwide

Progress Slowed

• In 1999, 18-year-old Jesse Gelsinger died from multiple organ failure 4 days after treatment for an enzyme deficiency.– Death was triggered by severe

immune response to adenovirus vector

• January 2003, halt to using retrovirus vectors in blood stem cells because children developed leukemia-like condition after successful treatment for X-linked severe combined immunodeficiency disease (bubble boy disease)

Progress Continues

• Cystic Fibrosis

• Alzheimers

• Parkinsons

• Sickle Cell

You will read more…….

GENE THERAPY. What is gene therapy? Gene therapy is the introduction of normal genes into cells that...

Documents

University of Groningen Ovarian cancer gene …Table 1. Genes Targeted/Employed For Ovarian Cancer Gene Therapy Mutation compensation Molecular chemotherapy Tumor suppressor genes

Gene Therapy

Biotechnology. The manipulation of organisms or their genes for – Basic biological research – Medical diagnostics – Medical treatment (gene therapy) –

GENE THERAPY. What Genes can do Genes, which are carried on chromosomes, are the basic physical and functional units of heredity. Genes are specific sequences

The Wild World of Biotechnology!!. Applications Genetic Transformation Cloning - Genes and entire organisms Gene Therapy Environmental Clean-Up

GENE THERAPY By Sam Lai and Meng Lee. Gene Therapy Introduction : What is it? Introduction : What is it? Gene Therapy : A Closer Look Gene Therapy : A

Genes as Medicine Film Guide Educator MaterialsFilm Guide Educator Materials Genes as Medicine OVERVIEW Gene therapy—the delivery of corrective genes into cells to treat a genetic

“Gene Therapy: An Updated Review” · 1.1 Considerations Gene therapy is ‘the use of genes as medicine’ involving the transfer of a therapeutic or working copy of a gene into

MODULE 8- LECTURE 1 GENE THERAPY: INTRODUCTION AND … · GENE THERAPY: INTRODUCTION AND METHODS . 8-1.1 Introduction . Gene therapy is a novel treatment method which utilizes genes

Tissue-Engineering Advanced Therapies - European Commission · Tissue Cell Therapy Gene Therapy Biotech Chemicals Engineering Advanced Therapies Medicinal products based on • Genes

NOTE RAPIDE - L'Institut Paris Region · GENE THERAPY, CELL THERAPY Gene therapies treat genetic diseases by repairing or replacing defective genes: functional genes are delivered

Gene Therapy and Gene Editing

Gene Therapy: The Basics - ISCTMDefinition of gene therapy Gene therapy is the introduction of nucleic acids (e.g. DNA/genes) into somatic cells of the body to correct or prevent a

Gene therapy and immunology Gene Therapy – an approach designed to treat disease by replacing, altering or supplementing genes that are defective or missing

Gene polymorphisms and gene therapy potential applications ...semmelweis.hu/oralbiologia/files/2016/02/16-gene-polymorphism-and-gene-therapy-in... · Gene polymorphisms and gene therapy

Human Gene Therapy - Heavenly Fuel · A Guide to Human Gene Therapy use produced. Many genes have been cloned and are available. The main obstacle to successful gene therapy is the

All about genes oncogenes mutations-cloning-gene therapy

16.5 Gene therapy 10.1 Coordination.. Learning outcomes By the end of this lesson I will know – The use of gene therapy is to supplement defective genes

Genes And Gene Technology

Gene Therapy: Tiny Target, Colossal Costs · Gene Therapy: Tiny Target, Colossal Costs. HM STOP LOSS. CELLS. ARE THE BUILDING BLOCKS . OF. ALL LIVING THINGS. Genes provide information