Gene Therapy
Genes code for proteins + they give rise to characteristics (working enzymes, petal
colours
Alleles are different versions of the same gene (proteins) = (variety of petal colours)
Recessive alleles code for non-functioning protein
Homozygous dominant + heterozygous have at least 1 allele that codes for a
functioning protein
Homozygous recessive – no alleles code for functioning protein, so non-function
protein made.
Genetic disorders can be caused by both dominant + recessive alleles (mostly
recessive alleles cause genetic diseases)
What is Gene Therapy + How it works?
Medical treatment for genetic disorders that alters/replaces allele that cause the
disorder.
If disorder is caused by….
Recessive allele – dominant allele is added = cystic fibrosis
Dominant allele – extra DNA inserted into allele to inactivate it = Huntington’s
Somatic vs germ line:
Somatic (body) gene therapy = alleles altered in specific somatic cells (normal
body cells)
Vector = delivery mechanism for desired allele to get into cells of interest. Cells of interest
change depending on the genetic disease present.
Vector injected into muscles = muscular dystrophy.
Vector inhaled into respiratory system = cystic fibrosis
Vectors introduced into blood stem cells/bone marrow transplant = scid
Germ line gene therapy = alleles altered in gametes
Genes code for proteins + they give rise to characteristics (working enzymes, petal
colours
Alleles are different versions of the same gene (proteins) = (variety of petal colours)
Recessive alleles code for non-functioning protein
Homozygous dominant + heterozygous have at least 1 allele that codes for a
functioning protein
Homozygous recessive – no alleles code for functioning protein, so non-function
protein made.
Genetic disorders can be caused by both dominant + recessive alleles (mostly
recessive alleles cause genetic diseases)
What is Gene Therapy + How it works?
Medical treatment for genetic disorders that alters/replaces allele that cause the
disorder.
If disorder is caused by….
Recessive allele – dominant allele is added = cystic fibrosis
Dominant allele – extra DNA inserted into allele to inactivate it = Huntington’s
Somatic vs germ line:
Somatic (body) gene therapy = alleles altered in specific somatic cells (normal
body cells)
Vector = delivery mechanism for desired allele to get into cells of interest. Cells of interest
change depending on the genetic disease present.
Vector injected into muscles = muscular dystrophy.
Vector inhaled into respiratory system = cystic fibrosis
Vectors introduced into blood stem cells/bone marrow transplant = scid
Germ line gene therapy = alleles altered in gametes
