- Gene therapy is a method of treating a medical condition by altering the organism at genetic level.
- Most of the diseases are as a result of reduced protein production, defective protein, mis-folded proteins etc. Since genes are the blueprint for protein production reprogramming cells or making changes at the genetic makeup level DNA is said to revolutionise medicine.
- Gene and cell therapies have immense applications in both regenerative medicine where organs are grown and drug development specific to a patient (personalised medicine).
Gene therapy involves
- Correcting a defective gene (permanent)
- Altering gene expression (temporary)
- Translation
- Transcription
Types of Gene Therapy
- Gene replacement
- Gene silencing
- Gene editing
- Gene addition/augmentation (CAR-T cell therapy)
Gene Delivery
- Vectors could be
- Viral
- Retrovirus
- Adenovirus
- AAV
- Non-viral
- Naked DNA
Further delivery could be in-vivo or ex-vivo.
Gene Therapy: Applications
- Haematological diseases (Haemophilia, Thalassaemia)
- Eye Diseases (Corneal diseases)
- Degenerative Neurological Diseases
- Immunological Diseases (SCID, HIV)
- Rare Diseases
- Oncology
- Dermatology
- Metabolic diseases
- Nucleic acid vaccines Eg: m-RNA Vaccines
