GENE THERAPY FOR ADENOSINE DEAMINASE DEFICIENCY

Slides:

Advertisements

Similar presentations

Advertisements

Option F: Microbes and Biotechnology F.3 Microbes and Biotechnology.

Therapy of enzyme defects: general considerations ● How many organs are affected by the enzyme defect: One organ, a few, or all organs? ● How severe is.

F3 Microbes and biotechnology

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

10 Genomics, Proteomics and Genetic Engineering. 2 Genomics and Proteomics The field of genomics deals with the DNA sequence, organization, function,

KEY CONCEPT Genetics provides a basis for new medical treatments.

Gene Therapy Correcting defective genes. Definition & history  Normal gene inserted into the genome to replace non-functional gene  Trials began in.

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

Genetic Engineering: Transcription, Translation, and Genetically Modified Organisms.

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.

Ethics of Biotechnology. CLONING What is CLONING? Creating new and identical organisms using biotechnology.

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.

Gene Therapy AP Biology Unit 2 + What is Gene Therapy? A way to treat or cure diseases by inserting the “correct” DNA into the cell. Most promising for.

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

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

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

Copyright © The McGraw-Hill Companies, Inc. Permission required for reproduction or display Chapter 20 Genetic Testing, Genetic Counseling, and Gene.

Slide 1 of 24 Copyright Pearson Prentice Hall Biology.

$Human Genome$. Human Genome Project & Goals It is an international effort to completely map and sequence the human genome, their are approximately 80,000.

Molecular Basis for Relationship between Genotype and Phenotype DNA RNA protein genotype function organism phenotype DNA sequence amino acid sequence transcription.

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.

Genetic Engineering Application of techniques of molecular cloning and transformation.

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,

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.

GENE THERAPY D of PG Studies.

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.

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.

DNA Technology. TO DO HUMAN GENOME PROJECT Started in map the 3 billion nucleotide sequencesThe project’s purpose was to discover all the estimated.

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

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 definition: is the insertion of genes into an individual's cell and biological tissues to treat disease, such as cancer where deleterious.

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

DIAGNOSIS OF DISEASES AND GENE THERAPY

What’s wrong with the abnormal cells?

Relationship between Genotype and Phenotype

Enzyme and gene therapy of enzyme defects

Gene therapy is defined as a set of strategies that modify the expression of an individual’s genes or that correct abnormal genes. Each strategy involves.

Therapy of enzyme defects: general considerations

Option F Biotechnology and Microbes

What is ... Gene Therapy?.

Gene Therapy By: Ashley Hale & Cody Stevens.

Correcting defective genes

Dr. Peter John M.Phil, PhD Atta-ur-Rahman School of Applied Biosciences (ASAB) National University of Sciences & Technology (NUST)

Relationship between Genotype and Phenotype

Gene Therapy Contemporary Issue – Genetic Disorders and Gene Therapy

Immunodeficiencies.

27 November 2018 Today’s Title: CW: Gene therapy

KEY CONCEPT Genetics provides a basis for new medical treatments.

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

KEY CONCEPT Genetics provides a basis for new medical treatments.

KEY CONCEPT Genetics provides a basis for new medical treatments.

Enzyme and gene therapy of enzyme defects

A promising future to disease treatment

KEY CONCEPT Genetics provides a basis for new medical treatments.

KEY CONCEPT Genetics provides a basis for new medical treatments.

Gene Therapy (Zinc-finger nuclease, Crisper-CAS 9)

Learning Intentions What causes cystic fibrosis?

Section 4 Lesson 6 – Gene Therapy

KEY CONCEPT Genetics provides a basis for new medical treatments.

Presentation transcript:

GENE THERAPY FOR ADENOSINE DEAMINASE DEFICIENCY Presented by SAMINA PARVEEN ROLL NO. 216 UID: 100484

GENE THERAPY It is the curing of a genetic disorder by introducing into the individual a normal gene in order to replace or overcome the effects of a mutant gene. There are two types of gene therapy: Somatic cell therapy Germ line cell therapy

TYPES OF GENE THERAPY Somatic cell therapy Germ line cell therapy Somatic cells are modified genetically to prevent a genetic defect in the offspring. Germ line cells are used to correct a genetic defect.

ADENOSINE DEAMINASE DEFICIENCY{ADA} ADA deficiency is one form of SCID (severe combined immunodeficiency), a type of disorder that affects the immune system. The disease is caused by a mutation in a gene on chromosome 20, which codes for the enzyme adenosine deaminase (ADA). Without this enzyme, the body is unable to break down a toxic substance called deoxyadenosine. The toxin builds up and destroys infection-fighting immune cells called T and B lymphocytes

GENE THERAPY FOR ADA TREATMENT First clinical gene therapy was given in 1990 to a 4 year old suffering from ADA. Steps for carrying it out- T cells were isolated from the girl and grown The normal ADA gene was introduced using a viral vector The “engineered cells” were then re-introduced into the patient

CHALLENGES IN GENE THERAPY Gene delivery and activation Immune Response Case of Jesse Gelsinger Disrupting important cells in target tissue

REFERANCES BOOK i-Genetics by Peter Russell Internet sites www.geneed.nlm.nih.gov www.his.nih.gov www.montegen.com www.learn.genetics.utah.edu