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Progress and problems with the use of viral vectors for gene therapy
With the development of a leukaemia-like syndrome in two patients cured of a disease by gene therapy, it is timely to contemplate how far this technology has come, and how far it still has to go.
Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways
The risk of oversaturating endogenous small RNA pathways can be minimized by optimizing shRNA dose and sequence, as exemplified here by the report of persistent and therapeutic RNAi against human hepatitis B virus in vivo.
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
We have previously shown that a single portal vein infusion of a recombinant adeno-associated viral vector (rAAV) expressing canine Factor IX (F.IX) resulted in long-term expression of therapeutic…
Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics
Further vector refinement and/or development is required before gene therapy will become standard care for any individual disorder, and some clinical successes are over the horizon.
Robust expansion of human hepatocytes in Fah−/−/Rag2−/−/Il2rg−/− mice
This system provides a robust platform to produce high-quality human hepatocytes for tissue culture and may be useful for testing the toxicity of drug metabolites and for evaluating pathogens dependent on human liver cells for replication.
Human tRNA-derived small RNAs in the global regulation of RNA silencing.
Findings support that tsRNAs are involved in the global control of small RNA silencing through differential Argonaute association, suggesting that small RNA-mediated gene regulation may be even more finely regulated than previously realized.
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.
Peripheral-vein infusion of scAAV2/8-LP1-hFIXco resulted in FIX transgene expression at levels sufficient to improve the bleeding phenotype, with few side effects.
A nonviral minicircle vector for deriving human iPS cells
Owing to the risk of insertional mutagenesis, viral transduction has been increasingly replaced by nonviral methods to generate induced pluripotent stem cells (iPSCs). We report the use of…
Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8.
In Vitro and In Vivo Gene Therapy Vector Evolution via Multispecies Interbreeding and Retargeting of Adeno-Associated Viruses
This study validates DNA family shuffling and viral peptide display as two powerful and compatible approaches to the molecular evolution of novel AAV vectors for human gene therapy applications.