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

Slides:



Advertisements
Similar presentations
Gene Therapy.
Advertisements

In conventional treatments of gene therapy viral and non- viral vectors are commonly used for the delivery of the gene. These are used to deliver normal.
How might we cure diseases in the future?. Using what we know about genes Pharmacogenetics/ Pharmacogenomics Gene Therapy Regenerative medicine.
 Gene therapy is a technique used to correct defective genes responsible for disease development.  There are several techniques to do this:  Normal.
Newer cancer therapies immunotherapy angiotherapy gene therapy.
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.
Protein Therapeutics: a summary and pharmacological classification
- Genetic Testing - - Genetic Counseling - - Genetic Therapy - By: Austin Justin Amanda Brie.
Cancer Gene Therapy …Using Tumor Suppressor Genes.
Gene therapy progress and prospects cancer. Gene Therapy Primary challenge for gene therapy – Successfully delivery an efficacious dose of a therapeutic.
GENE THERAPY Presented to : Dr.Leslye Jhonson Presented by: Khazeema Yousaf & Maheen Alam Biot 412: Medical Biotechnology.
GENE THERAPY.
Genetic Engineering What is genetic engineering ? Genetic engineering, genetic modification (GM), and the now-deprecated gene splicing are terms for.
Gene therapy for eye disease UCL Institute of Ophthalmology Department of Genetics An experimental technique that uses the delivery of genetic material.
Molecular Diagnosis & Gene Therapy Asmarinah Department of Medical Biology Faculty of Medicine, University of Indonesia.
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.
Medical Genetics 19 基因治疗 Gene Therapy. Medical Genetics 1. What is gene therapy? Genes, which are carried on chromosomes, are the basic physical and functional.
Viruses are used for gene therapy
© 2013 The McGraw-Hill Companies, Inc. All rights reserved. Ch 13 Health Care Trends and Forecasts.
Can Humans Live Forever? Human beings do not live forever because the human body cannot maintain itself indefinitely. We either grow old or we get diseased.
Human genetics. Sex-influenced traits are autosomal traits that are influenced by sex. If a male has one recessive allele, he will show that trait, but.
Gene therapy- Methods, Status and Limitations. Methods of gene delivery (therapeutic constructs) It Includes two methods: Nonviral gene-delivery systems.
Gene Therapy. What is Gene Therapy? Defective genes make non-functional proteins, creating genetic disorders Gene therapy corrects defective genes by.
Biotechnology Packet #12 Chapter #9. Introduction Since the 1970’s, humans have been attempted to manipulate and modify genes in a way that was somewhat.
Anti-mRNA Strategies What is the antisense oligonucleotides? - Synthetic genetic material. - Interacts with natural genetic material (DNA or RNA) prevent.
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.
Gene Therapy. Gene Therapy is a technique for correcting defective genes responsible for disease development Gene Therapy is a technique for correcting.
Restriction Enzymes, Recombinant DNA, Gene Therapy & Genetically Modified Organisms Biology 2011.
GENE THERAPY.
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
©1999 Timothy G. Standish Pharmaceutical Biotechnology PHG 424 Mounir M. Salem, Ph.D. King Saud University College of Pharmacy Departments.
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.
What is gene therapy? Do now: In your own words,
Gene editing in embryos and germ line
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.
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.
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.
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.
Viral Genetics. The objectives What genetic changes are detected in viruses? The effects of viral genetic changes on viral virulence. The application.
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 Mostafa A. Askar NCRRT By M.Sc. In Molecular Biology
Gene Therapy Biology 11: Section 6.5. Learning Goals Students will describe gene therapy Students will describe the process of gene insertion.
Gene therapy.
How might we cure diseases in the future?
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.
Dr. Peter John M.Phil, PhD Atta-ur-Rahman School of Applied Biosciences (ASAB) National University of Sciences & Technology (NUST)
Gene Therapy.
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
KEY CONCEPT Genetics provides a basis for new medical treatments.
KEY CONCEPT Genetics provides a basis for new medical treatments.
Gene Therapy.
KEY CONCEPT Genetics provides a basis for new medical treatments.
KEY CONCEPT Genetics provides a basis for new medical treatments.
KEY CONCEPT Genetics provides a basis for new medical treatments.
Presentation transcript:

Gene Therapy

antisense oligonucleotides(ODNs) antisense oligonucleotides(ODNs) Ribozymes Ribozymes DNAzymes DNAzymes RNA interference(RNAi). RNA interference(RNAi). Antisense Technologies

Gene Therapy idea is to replace defective genes ----idea is to replace defective genes Gene therapy is a technique for correcting defective genes responsible for disease development.

Several approaches for correcting faulty genes 1. A normal gene may be inserted into a nonspecific location within the genome to replace a nonfunctional gene. 2. An abnormal gene could be swapped for a normal gene through homologous recombination. 3. The abnormal gene could be repaired through selective reverse mutation, which returns the gene to its normal function. 4. The regulation (the degree to which a gene is turned on or off) of a particular gene could be altered.

Deliver the therapeutic gene Some of the different types of viruses used as gene therapy vectors: Some of the different types of viruses used as gene therapy vectors: Retroviruses Retroviruses Adenoviruses Adenoviruses Adeno-associated viruses Adeno-associated viruses Herpes simplex viruses Herpes simplex viruses

Deliver the therapeutic gene Adenoviruses

Efficient delivery Efficient delivery Enhanced stability Enhanced stability Minimization of off-target effects Minimization of off-target effects Identification of sensitive sites in the target RNAs Identification of sensitive sites in the target RNAs Challenge

The Food and Drug Administration (FDA) has not yet approved any human gene therapy product for sale. The Food and Drug Administration (FDA) has not yet approved any human gene therapy product for sale. Status of gene therapy

Gene Therapy in the clinic Trials Wordldwide 66% Phase I 21% Phase I-II 11% Phase II 0.8% Phase II-III 0.7% Phase III in spite of 13 year- research only less than 1% of the trials has reached phase III

Genetic Modification Clinical Research Information System (GeMCRIS) Genetic Modification Clinical Research Information System (GeMCRIS) For more details

Thanks~!! ~*_*~

Feedback: Privacy Policy Feedback
Do Not Sell My Personal Information
About project: SlidePlayer Terms of Service

© 2024 SlidePlayer.com Inc. All rights reserved.