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Open AccessHIV- 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...
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Article
Open AccessProduction 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...
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Article
Open AccessGene 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...
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Article
Open AccessExpanding 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...
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Article
Erratum to: Increased glucocerebrosidase (GBA) 2 activity in GBA1 deficient mice brains and in Gaucher leucocytes