What is gene therapy for cystic fibrosis?
Gene therapy: a promising candidate for cystic fibrosis treatment. Cystic fibrosis is caused by mutations in the CFTR gene. This gene contains the production code for a protein that functions as a chloride/bicarbonate channel and regulates fluid secretion across the epithelium in different organs (airways, pancreas, intestine,...
What are the treatment options for cystic fibrosis?
Since cystic fibrosis is hereditary, it cannot be treated like a normal infection caused by external bacteria or viruses. The condition is normally treated with antibiotics to address secondary chest infections and drugs that thin the mucus in the lungs.
Are animal models of cystic fibrosis disease relevant for gene therapy?
Animal models of CF are valuable tools that may be utilized to further understand disease pathogenesis and test new therapeutics. There are two fundamental issues that remain to be resolved before gene therapy can become viable for patients, and animal models provide a relevant platform through which these obstacles may be safely addressed.
What is the genetic mutation responsible for cystic fibrosis?
The genetic mutation responsible for cystic fibrosis is a mutation in both copies of the cystic fibrosis membrane conductance regulator (CFTR) gene. Genetic engineering techniques like CRISPR can locate the 20 DNA markers that are responsible for the CFTR mutation.
What is a promising treatment for cystic fibrosis?
In 2019, the FDA approved Vertex's Trikafta, a triple-drug combination of ivacaftor, elexacaftor and tezacaftor that increased lung function in people with cystic fibrosis by an average of 14% in clinical trials1.
What genetic technology is used for cystic fibrosis?
A type of integrating gene therapy, known as CAR-T therapies, has already been approved to treat patients with certain kinds of leukemia and lymphoma. An integrating gene therapy to treat CF is being tested in animals, and a clinical trial to test the safety.
How might cystic fibrosis be treated with genetic engineering?
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 has technology help cystic fibrosis?
The smartphone-based solution allows people with cystic fibrosis to monitor their health at home with devices that measure key indicators such as lung function, blood oxygen levels and activity.
Is CRISPR used for cystic fibrosis?
CRISPR/Cas9 is an experimental approach for treating cystic fibrosis (CF). The therapy features a novel protein-RNA complex that is designed to address the genetic mutations that cause the disease by editing a patient's genetics, correcting the mutations themselves.
What is the best vector for gene therapy for cystic fibrosis Why?
Highlights Box. Since the cystic fibrosis gene was discovered in 1989, researchers have worked to develop a gene therapy as an ultimate cure. One of the most promising and enduring vectors is the adeno-associated virus (AAV), which has been shown to be safe in several clinical trials.
Why is ex vivo gene therapy better?
Selecting an ex vivo treatment may be better suited for treating conditions that affect the blood, such as hemophilia, where a person's own self-renewing stem cells can be engineered with a functional working gene and then delivered into their body.
Gene therapy: a promising candidate for cystic fibrosis treatment
An improved gene therapy treatment can cure mice with cystic fibrosis (CF). Cell cultures from CF patients, too, respond well to the treatment. Those are the encouraging results of a study presented by the Laboratory for Molecular Virology and Gene Therapy at KU Leuven, Belgium.
Publication
rAAV-CFTRΔR Rescues the Cystic Fibrosis Phenotype in Human Intestinal Organoids and Cystic Fibrosis Mice. Vidović D, Carlon MS, da Cunha MF, Dekkers JF, Hollenhorst MI, Bijvelds MJ, Ramalho AS, Van den Haute C, Ferrante M, Baekelandt V, Janssens HM, De Boeck K, Sermet-Gaudelus I, de Jonge HR, Gijsbers R, Beekman JM, Edelman A, Debyser Z. Am J Respir Crit Care Med.
What is gene therapy?
Gene therapy involves the transfer of correct copies of cystic fibrosis transmembrane conductance regulator (CFTR) DNA to the epithelial cells in the airways.
Is CF a lifelong disease?
As CF is a lifelong disease, turnover of the airway epithelium necessitates repeat administration. To date, this has been difficult to achieve with viral gene transfer agents due to host recognition leading to loss of expression.
Is gene therapy a treatment for cystic fibrosis?
Gene therapy is being developed as a novel treatment for cystic fibrosis (CF), a condition that has hitherto been widely-researched yet for which no treatment exists that halts the progression of lung disease.
What is the most common disease causing variant with a frequency of about 70% in CF population?
Among these is F508del, a three nucleotide deletion that leads to the omission of a phenylalanine residue at codon 508. The F508del is the most common disease causing variant with a frequency of about 70% in CF population.
What is the difference between genetics and genomics?
Genetics is the branch of biology concerned with study of individual genes and how they work whereas genomics is involved with the analysis of all genes and their interactions. Both of these approaches have been applied extensively to CF. Identification of the CFTRgene initiated the dissection of CF genetics at the molecular level.
What organ systems are affected by CFTR?
In general, variants that cause complete or near complete loss of CFTR function results in severe features in the organ systems primarily affected in CF (i.e., lungs, pancreas, intestine and sweat gland). However, there can be considerable intra- and inter- individual differences in the degree of organ dysfunction.