Gene Therapy

  • 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.

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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
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