5 Result(s)

Article

HIV- 1 lentivirus tethering to the genome is associated with transcription factor binding sites found in genes that favour virus survival

Lentiviral vectors (LV) are attractive for permanent and effective gene therapy. However, integration into the host genome can cause insertional mutagenesis highlighting the importance of understanding of LV i...

Saqlain Suleman, Annette Payne, Johnathan Bowden, Sharmin Al Haque… in Gene Therapy (2022)

Article

Production of lentiviral vectors using novel, enzymatically produced, linear DNA

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 abiological technology that ad...

Rajvinder Karda, John R. Counsell, Kinga Karbowniczek, Lisa J. Caproni… in Gene Therapy (2019)

Article

Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects

Over the last decade, pioneering liver-directed gene therapy trials for haemophilia B have achieved sustained clinical improvement after a single systemic injection of adeno-associated virus (AAV) derived vect...

Julien Baruteau, Simon N. Waddington… in Journal of Inherited Metabolic Disease (2017)

Article

Expanding the phenotype in argininosuccinic aciduria: need for new therapies

This UK-wide study defines the natural history of argininosuccinic aciduria and compares long-term neurological outcomes in patients presenting clinically or treated prospectively from birth with ammonia-lower...

Julien Baruteau, Elisabeth Jameson… in Journal of Inherited Metabolic Disease (2017)

Article

Erratum to: Increased glucocerebrosidase (GBA) 2 activity in GBA1 deficient mice brains and in Gaucher leucocytes

Derek G. Burke, Ahad A. Rahim… in Journal of Inherited Metabolic Disease (2013)