Gene Therapy
- Created by: beth-marie2511
- Created on: 21-06-16 11:21
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- Gene Therapy
- Inserting a normal gene/DNA sequence to replace the faulty gene
- Ways to Correct Faulty Genes
- Normal gene is inserted into a non specific location within the genome to replace a non-functioning gene.
- Abnormal gene is swapped for a normal gene by homologous recombination
- Abnormal gene could be repaired through selective reverse mutation, returning the gene to its normal function
- Regulation of a particular gene could be altered
- 2 Ways of Replacing Faulty Genes
- Gene therapy involving somatic cell therapy targets cells in the affected tissues
- This method may be therapeutic but the genes are not inherited
- Germ-line Therapy involves the introduction of corrective genes into germ-line cells (oocyte or sperm)
- The gene is replaced in the egg and will enable genetic corrections to be inherited
- Gene therapy involving somatic cell therapy targets cells in the affected tissues
- How Does It Work?
- A carrier molecule called a 'vector' must be used to deliver the therapeutic gene to the patient's target cells
- Most common vector is a VIRUS that has been genetically altered to carry normal human DNA
- Target cells are infected with the viral vector and the vector unloads the genetic material carrying the therapeutic gene into the target cells
- Target cells are returned to a 'normal' state.
- Liposomes can also be used as vectors as they are capable of passing DNA through the target cell's membrane
- Factors preventing it from becoming an effective treatment for genetic disease
- Short-lived nature
- Therapeutic DNA introduced to target cells must remain functional and the cells must be long-lived and stable
- The rapidly dividing nature of many cells prevents therapeutic gene therapy from achieving long term benefits
- Therapeutic DNA introduced to target cells must remain functional and the cells must be long-lived and stable
- Immune Response
- Immune systems are designed to attack any foreign invader introduced into human tissues
- Reduces the effectiveness of gene therapy
- Immune systems are designed to attack any foreign invader introduced into human tissues
- Problems with Viral Vectors
- Potential problems with patients include toxicity, immune and inflammatory responses
- Gene control
- Has the potential to recover its ability to cause disease once inside the patient
- Potential problems with patients include toxicity, immune and inflammatory responses
- Multigene Disorders
- Most common diseases are caused by a combination of effects in multiple genes
- Alzeimers
- Heart Disease
- Diabetes
- Difficult to treat effectively
- Most common diseases are caused by a combination of effects in multiple genes
- Short-lived nature
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