Since its first identification in Scotland, over 1,000 cases of unexplained paediatric hepatitis
in children have been reported worldwide, including 278 cases in the UK 1 . Here we report …

Duchenne Muscular Dystrophy (DMD) is caused by a lack of dystrophin expression in patient
muscle fibres. Current DMD gene therapy strategies rely on the expression of internally …

Skeletal muscle is a complex tissue composed of multinucleated myofibers responsible for
force generation that are supported by multiple cell types. Many severe and lethal disorders …

Most inherited neurodegenerative disorders are incurable, and often only palliative treatment
is available. Precision medicine has great potential to address this unmet clinical need. We …

Lentiviral vector genomic RNA requires sequences that partially overlap wild-type HIV-1
gag and env genes for packaging into vector particles. These HIV-1 packaging sequences …

Autologous stem cells that have been genetically modified to express dystrophin are a
possible means of treating Duchenne Muscular Dystrophy (DMD). To maximize the therapeutic …

The manufacture of large quantities of high-quality DNA is a major bottleneck in the
production of viral vectors for gene therapy. Touchlight Genetics has developed a proprietary …

Explosion of gene therapy approaches for treating rare monogenic and common liver
disorders created an urgent need for disease models able to replicate human liver cellular …

The human adenovirus phylogenetic tree is split across seven species (A–G). Species D
adenoviruses offer potential advantages for gene therapy applications, with low rates of pre-…

Background Cancer cells remodel their local physical environment through processes of
matrix reorganisation, deposition, stiffening and degradation. Urokinase-type plasminogen …