Gene Therapy & Cell Replacement Therapy

What is Gene Therapy?

Gene Therapy 

• Aims to treat or cure disease by identifying faulty genes and inserting healthy ones 

• Still in research phase 

• Concept is that a vector (e.g. a virus) can be used to deliver the desired DNA into the cell, to be incorporated by the nucleus and produce desired protein. 

• Areas of possibility: 

  • Replacing mutated gene with healthy copy 

    • e.g. huntingtons disease, cystic fibrosis, type 1 diabetes 

  • Fixing or inactivating mutant genes 

    • e.g. huntingtons disease 

    • Inserting a new gene that will fight the disease 

  • Making the immune system recognise disease cells 

    • e.g. therapy for some cancers 

Gene Therapy: Cystic Fibrosis 

• Relatively common 

• Recessive condition, due to faulty gene: 

• Causes inability of body to process certain secretions 

  • Buildup of mucus in lungs, and digestive tract 

  • Can significantly shorten life 

• Fire experimental gene therapy given in 1993 

  • Used the common cold virus as vector to carry the normal gene into lungs 

  • Mainly assessed safety of procedure 

  • Not yet used as therapy, still in research phase 

What is Cell Replacement Therapy?

Cell Replacement Therapy 

• Uses stem cells 

  • Undifferentiated 

  • Often sourced from embryo's 

• Stem cell replacement theory 

  • Especially for neurological diseases e.g. 

    • Parkinson's 

    • Alzheimer's 

 

• Can be used in future for neurodegenerative disorders such as Alzheimer's and Parkinson's 

 

• Replacement of dying neurons with healthy ones derived from stem cells 

  • Can grow and differentiate to replace damaged tissue 

What does Gene Therapy aim to do?

• Aims to treat or cure disease by identifying faulty genes and inserting healthy ones