Gene Therapy

  1. A corrective therapy to a person born with hereditary disease

Process

  1. Transfer of Normal gene into a person's cell that carries one or more mutant alleles
  2. Delivery - Uring vector

Tetreat disease

  1. Cystic florosis
  2. Haemophilia
  3. ADA - Adenosine deaminase deficiency
  4. SCID - Severe combined Immuno Deficiency

Strategies

    1. Gene Augmentation
      1. To replace missing gene
      2. Insertion of dna into genome
    2. Gene Inhibition
      1. Inhibit expression of derminant gene
      2. Insenstion of anti sense gene

2 Approaches

  1. Somatic Cell Gone therapy
    1. insertion of fully functional & expressible gene into target somatic cells
    2. Correct Genetic disease permanently
    3. Introduced into Bone marrowcells, Blood cells, Skin cells etc
    4. Not Inherited to Next generation
  2. Germ Line Gene Therapy
    1. Introduction of dna into germ cells
    2. Introduced into Eggs & Sperm
    3. Passed onto Successive generation

issues

  1. Gene has to be harmless to patient
  2. Bodys Immune response should not react to foreign proteins produced by New genes