How can cystic fibrosis be treated by gene therapy?

How can cystic fibrosis be treated by gene therapy?

The treatment uses a type of virus called a lentivirus to deliver a healthy copy of a gene called CFTR, which causes cystic fibrosis when it carries a mutation. The gene therapy will be given by inhalation to better target the right cells.

How is gene therapy used to treat medical conditions?

Gene therapy replaces a faulty gene or adds a new gene in an attempt to cure disease or improve your body’s ability to fight disease. Gene therapy holds promise for treating a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS.

What makes cystic fibrosis a good candidate for gene therapy approaches?

Summary. Cystic fibrosis is a single gene disorder viewed as a good candidate for gene therapy because the affected gene is known, the target tissue, the lung, is accessible and less than 50% gene transfer may confer clinical benefit.

Is CF a good candidate for gene therapy?

Gene therapy is particularly attractive for diseases that currently do not have satisfactory treatment options and probably easier for monogenic disorders than for complex diseases. Cystic fibrosis (CF) fulfills these criteria and is therefore a good candidate for gene therapy-based treatment.

Is there gene therapy for cystic fibrosis UK?

The UK Cystic Fibrosis Gene Therapy Consortium (Imperial College London, University of Edinburgh and University of Oxford) is currently working on a large and ambitious program to establish the clinical benefits of CF gene therapy. Wave 1, which has reached the clinic, uses a non-viral vector.

How does RNA therapy work for cystic fibrosis?

RNA Therapy Genes are composed of a series of DNA “letters” that spell out the instructions to make a protein. However, the cell does not use the DNA directly. Instead, the cell makes a working copy of the gene using a similar alphabet called ribonucleic acid (RNA) and uses that working copy to build the protein.

How are gene mutations related to cystic fibrosis?

These proteins regulate how much of these substances enter and leave the cells to keep it functioning properly. The cystic fibrosis genetic mutations mean that the cells have an error in their instructions (in this case, the CFTR gene), so the protein is not made properly, or is not sent to the cell surface where it is needed.

How does non-integrating gene therapy work for CF?

Non-Integrating Gene Therapy In non-integrating gene therapy, a piece of DNA with a correct copy of CFTR is provided to an individual’s cells, but the DNA remains separate from the genome. This is like placing a new page between the covers of an existing book without permanently attaching it.

What is the genetic cause of cystic fibrosis?

The cause of cystic fibrosis (CF) is a defect in a gene called the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This gene makes a protein that controls the movement of salt and water in and out of the cells in your body.

What does it mean to be a cystic fibrosis carrier?

A cystic fibrosis carrier is a person who has the mutation that causes cystic fibrosis in his or her genes but does not have the disease. Each person has two cystic fibrosis transmembrane regulator genes. If there is one normal cystic fibrosis transmembrane regulator gene and the other is mutated, the person is a carrier.

What are the most common cystic fibrosis mutations?

CF is caused by a mutation in the gene cystic fibrosis transmembrane conductance regulator (CFTR). The most common mutation, ΔF508, is a deletion (Δ signifying deletion) of three nucleotides that results in a loss of the amino acid phenylalanine (F) at the 508th position on the protein.

What is mRNA therapy?

mRNA therapy is engineered to deliver mRNA encoding natural, functional proteins that replace defective or missing proteins, and has potential advantages, including that it: restores gene expression without entering the cell nucleus or changing the genome;