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

B A C E D

 Specific sequence of bases that encode instructions on how to make proteins.  Genes are passed on from parent to child.  When genes are altered, and they don’t produce the right proteins or correctly, a child may have genetic disorder.  What genetic disorders have you heard of?

 An experimental technique used to treat / or prevent diseases. - by correcting faulty genes.  This is so that, in the future a gene can be inserted into a patient’s cell instead of using surgery or drugs.

 Replacing a mutated gene that causes disease with a healthy copy of the gene. (most common)

 A ‘normal’ gene is inserted into the genome to replace and ‘abnormal’ disease-causing gene.  A vector (carrier molecule) is used to deliver the ‘normal’ gene to the patient’s target cell. - normally a virus is the most common vector which has been genetically altered so that it can carry normal human DNA.  Virus vectorVirus vector

 Vectors are used to deliver ‘normal genes’ to target cells  The target cells like the patient’s liver or lung cells are infected with the viral vector.  The vector then unloads its genetic material containing the gene into target cell.  Functional proteins are produced from the ‘normal’ gene and restores the target cell to normal state.

 The technique is risky and is still under study to make sure it is safe and effective.  Only being tested for treatment of diseases that have no cure

 This approach is limited in its application as its used only with certain tissues and requires large amounts of DNA.  Patients have to undergo multiple rounds of gene therapy.  May trigger immune response  Problems with viral response

 Are disabilities diseases? Should they be cured or prevented?  Do searching for the cure demean the lives of those who are presently affected by disabilities.  It is very expensive – so who’ll have access to these? And who’ll pay?