Gene therapy for eye disease UCL Institute of Ophthalmology Department of Genetics An experimental technique that uses the delivery of genetic material.

Slides:

Advertisements

Similar presentations

Advertisements

Presenter: Juan Mo. Learning objectives What is gene therapy? What is a Dox-regulatable system and how can gene expression be regulated by Dox? What is.

UCL Institute of Ophthalmology Department of Genetics

 Gene therapy is a technique used to correct defective genes responsible for disease development.  There are several techniques to do this:  Normal.

HISTORY AND DEVELOPMENT OF GENE THERAPY 1960: The concepts of Gene Therapy was introduced 1970: Friedmann and Roblin author of a paper in Science titled.

GENE THERAPY Presented at Paradoxes Sunday School Class, Sierra Madre Congregational Church, June 27, 2004.

FOLLOWING PATTERNS OF INHERITANCE. GENETIC TESTS Types of Genetic Tests a)Karyotype b)Fluorescence in situ hybridization c)Gene testing d)Biochemical.

GENE THERAPY. In humans Cancer 69% General concerns The Food and Drug Administration (FDA) has not yet approved any human gene therapy product for sale.

Cancer Gene Therapy …Using Tumor Suppressor Genes.

Charlie McLaughlin. What are Genetic Disorders?  Genetic disorders are illnesses stemming from errors in a person’s genes  Any mistake in a gene can.

KEY CONCEPT Genetics provides a basis for new medical treatments.

LO: Be able to describe what gene therapy is and how it could be used.

Viruses are used for gene therapy

LEQ: WHAT ARE THE BENEFITS OF DNA TECHNOLOGY & THE HUMAN GENOME PROJECT? to

Gene therapy- Methods, Status and Limitations. Methods of gene delivery (therapeutic constructs) It Includes two methods: Nonviral gene-delivery systems.

Gene Therapy and Genetic Counseling. Treating Genetic Diseases Protein-based therapiesProtein-based therapies Disease Therapeutic Agent Cystic Fibrosis.

Genes, which are carried on chromosomes, are the basic physical and functional units of heredity. Genes are specific sequences of bases that encode instructions.

Gene Therapy. What is Gene Therapy? Defective genes make non-functional proteins, creating genetic disorders Gene therapy corrects defective genes by.

VIRUSES Tobacco mosaic virus Influenza virus Adenovirus Bacteriophage.

Consider: WWWWe get all of our traits from all the generations before us. NNNNaturally, we each have our own probability of something in.

Color Blindness By: Emily Landers. What is color blindness??? Color blindness is condition where someone has a hard time distinguishing between certain.

An Overview of the curriculum module available on

Kortlynn Johnson. What is Gene Therapy? A technique for correcting defective genes responsible for disease development 1.

Experimental Gene Therapy Use On Humans. What is gene therapy? Gene therapy is a method of curing genetic disorders by introducing functioning genes into.

Chapter 8: Genetic Engineering by Mailee Phet by Mailee Phet My Hoa Nguyen.

An Ethical Debate.  Humans have been breeding farm animals for thousands of years  Selective breeding is done to get the traits a person wants.

Gene Therapy. Gene Therapy is a technique for correcting defective genes responsible for disease development Gene Therapy is a technique for correcting.

Genomics and Me The Study of Cancer Disease. Introduction What is Cancer Disease? A set of disease in which cells escape from the control mechanisms A.

Jordan Jones Lauren Johnson. What is it? Gene therapy, also known as genetic modification, is a technique for correcting faulty genes that are responsible.

Gene Therapy and Viral Vector

Gene Therapy By: Chris Smith and Darran Prewitt. What is gene therapy? Why is it used? Gene therapy = Introduction of normal genes into cells that contain.

GENE THERAPY -“molecular bandage” -use of DNA as a pharmaceutical, to treat disease.

What is gene therapy? Do now: In your own words,

GENETICS AND DNA HEREDITY. CELL Cells are the building blocks of human body. Different cells have many different functions. They all contain the same.

Control of Gene Expression. Ways to study protein function by manipulating gene expression Mutations –Naturally occurring, including human and animal.

Genetic Testing & Gene Therapy 5.3. Genetic Testing & Gene Therapy (5.3)  Genetic tests look for signs of a disease or disorder in DNA taken from an.

6.5 Gene Therapy Pages Gene therapy Defective genes are corrected using a normal copy of the gene.

What is... Gene Therapy?. Genes Specific sequence of bases that encode instructions on how to make genes. Genes are passed on from parent to child. When.

Gene Therapy By: Destiny Osborne & Amanda Owens. Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future,

Gene Therapy. What is Gene Therapy? Gene Therapy is the insertion of genes into an individual’s cells and tissues to treat a disease. Gene Therapy is.

In most gene therapy studies, a "normal" gene is inserted into the genome to replace an "abnormal," disease-causing gene. A carrier molecule called a.

Gene Therapy Molecular genetics presentation Made by / Mostafa Ahmed Supervisor Dr / Noha Khalifa.

Gene Therapy. antisense oligonucleotides(ODNs) antisense oligonucleotides(ODNs) Ribozymes Ribozymes DNAzymes DNAzymes RNA interference(RNAi). RNA interference(RNAi).

Gene Therapy Mostafa A. Askar NCRRT By M.Sc. In Molecular Biology

STEM CELL RESEARCH. What are stem cells? Cells that can generate more copies of themselves Have specialized function in body.

Gene Therapy Vasileios Antonopoulos (Teacher) Evangelos Tsouramanis (Teacher) Eleni Loukopoulou (Student) Vasileios Vagenas (Student)

Human Disorders and Gene Therapy

Gene Therapy: Molecular Biology

Full length dystrophin gene Microgene for dystrophin

What is ... Gene Therapy?.

Gene Therapy By: Ashley Hale & Cody Stevens.

Gene Therapy Presentation brought to you by: Therapeutic Genes Inc.

Gene Therapy Contemporary Issue – Genetic Disorders and Gene Therapy

KEY CONCEPT Genetics provides a basis for new medical treatments.

Genes The basic unit of heredity Encode how to make a protein

Gene Therapy Learning Goal: To explore gene therapy. Success Criteria:

KEY CONCEPT Genetics provides a basis for new medical treatments.

KEY CONCEPT Genetics provides a basis for new medical treatments.

Gene Therapy Section 6.5.

KEY CONCEPT Genetics provides a basis for new medical treatments.

KEY CONCEPT Genetics provides a basis for new medical treatments.

Section 4 Lesson 6 – Gene Therapy

KEY CONCEPT Genetics provides a basis for new medical treatments.

Presentation transcript:

Gene therapy for eye disease UCL Institute of Ophthalmology Department of Genetics An experimental technique that uses the delivery of genetic material to treat or prevent disease by: Delivering a working copy of a damaged gene that causes disease – gene supplementation therapy Inactivating, or “knocking out,” a mutated gene that is functioning improperly – gene silencing Introducing a new gene into the body to help fight a disease – neuroprotection or angiostatic therapy

What is gene therapy? A carrier called a vector delivers the therapeutic gene to the patient's target cells – the most common vector is a virus that has been genetically altered to carry the therapeutic gene. Which vector we choose to use depends on the cells we want to target and the size of the gene we wish to deliver to these cells. The three types of virus usually used to study gene therapy in the eye are lentivirus (such as HIV), adenovirus and adeno-associated virus (AAV).

Adeno-associated virus (AAV) AAV is the most commonly-used vector, as it is not harmful even in its "wild" form. AAV efficiently delivers genes to light-sensitive photoreceptor cells and the underlying retinal pigment epithelium (RPE), making it an ideal gene therapy vector for inherited retinal disorders When injected under the retina, AAV efficiently delivers a reporter gene, encoding Green Fluorescent Protein, to photoreceptor and RPE cells

Direct injection of viral vectors into the eye. For our gene therapy clinical trials, the surgical procedure involves injecting the virus under the retina, producing a temporary retinal detachment Recovery time is usually rapid and it is often possible to go home the same day Quality of sight is reduced temporarily until recovery is complete, which is usually expected to take a few weeks.

Targeting different cells in the eye Ideally, the therapeutic gene should only be delivered to the cells that would naturally produce this protein The vector may infect many cell types when it is injected, so by using a cell-specific promoter, we can restrict gene expression to certain cell types within a tissue: (a) photoreceptor cells and RPE; (b) RPE only, (c) cone photoreceptor cells only; (d), (e) corneal cells and (f) Muller cells in the inner retina