Gene Therapy

Understanding the nature of the gene involved in cystic fibrosis can lead
to gene therapy
In gene therapy- the genotype and phenotype of target cells is altered
The normal alleles if the genes are inserted into target cells either using
genetically modified virus to infect the target cell or use liposomes
The normal form of a gene is transcribed and translated.
The protein that works is produced in target cells

Gene therapy altering the alleles inside cells to cure genetic disorders.
Alleles are inserted into cells using vectors.

Two types of gene therapy:

Somatic therapy changing alleles in body cells-doesnt affect individual sex cells through so
offspring can still inherit the disease
Cells are most affected by the disorder

Germ line therapy Changing the allele in sex cells

Every cell of offspring wont suffer from the disease
Healthy gene must be copied

Reverse- transcriptase
Enzyme used to copy the healthy gene into mRNA and from this mRNA
produce the DNA containing the sequence of bases further correct code
Endonuclease
Enzyme used to cut the healthy gene out of a section of DNA
Ligase
Enzyme used to paste healthy gene into DNA
Vector (means of transport) is used
Viruses are used as a vector-they invade cells
Viruses
Used as a vector to carry new healthy gene into nucleus of the cell for
incorporation into the DNA
Somatic
Any cell in the body that isnt a sex cell

 Germ cell
Every new cell will be non-defect
Changing original cells
Done on an embryo mainly
Villis sampling
Placental tissue is removed early on
Amniocentesis
Inserting a needle into the amniotic fluid to collect cells that have fallen off
the placenta and foetus-but this is done late
Male X chromosome is inactive for a while
Villis sampling wont be able to test it out
Phenylketonuria
Lack of the enzyme phenylalanine hydroxylase
Its a disease