Gene Therapy

Gene Therapy

Inserting a functional allele of a particular gene into cells that contain only mutated and non-functioning alleles of that gene. This allows them to produce a functional protein.

Two types of gene therapy:

1. Somatic cell gene therapy - functioning alleles are inserted into body cells.Changes are short lived and treatment needs to be repeated. Alterations are not passed to the patient’s offspring.

Liposomes (in cystic fibrosis)

• CFTR alleles packaged within liposomes placed into aerosol inhaler and sprayed into noses of patients

• Liposome passes through plasma membrane of cells in respiratory tract

• They pass through the nuclear envelope and insert into host genome

• The host cell will express the CFTR protein (a transmembrane chloride ion channel)

    Alleles are inserted into a liposome which then fuses with the target cell membrane.

Problems: Liposomes are nonspecific and it is difficult to target cells. Functioning allele not passed onto…