An experimental drug which aims to treat DMD by exon skipping. It introduces a 'molecular patch' over the mutated exon, enabling the gene to be read. A shorter, more functional type of dystrophin is synthesised.
An X-linked recessive condition that is characterised by muscle degeneration and weakness. It is caused by one or more mutations in the dystrophin gene that prevent the production of dystrophin.
A service that provides information and advice to people affected by or at risk of genetic diseases. This helps individuals and families to make informed decisions.
The modification of the genome of an organism by the insertion of a desired gene from another organism. This enables the formation of organisms with beneficial characteristics.
A type of gene therapy in which a faulty allele is replaced with a functional allele in germ cells or a very early embryo. The effects of this are permanent and can be inherited.
A genetic disorder characterised by the progressive degeneration of nerve cells in the brain, reducing the affected individual's ability to think, talk and move.
A disease caused by the protoctista Plasmodium that lives within two hosts, mosquitoes (Anopheles gambiae) and humans. It causes recurrent episodes of fever and can be fatal.
An additional gene inserted into a plasmid that is used to aid in the identification of host cells that have taken up the desired gene. Marker genes are easily recognisable e.g. provide antibiotic resistance.
The method of DNA sequencing used in the Human Genome Project that involves the formation of DNA fragments of varying lengths. This process takes a long time.