Vectors in gene therapy
The state of the art of adeno associated virus based vectors in gene therapy
... adenoviral vectors, and turn it into a very attractive tool for gene ther- ...AAV vectors only retain about 300 nucleotides of viral sequence in the form of nontranscribed ITRs, which greatly ...
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Retroviral vectors and transposons for stable gene therapy: advances, current challenges and perspectives
... MLV vectors have the biological disadvantage that they are unable to effi- ciently transduce non-dividing or slowly dividing ...MLV vectors were gradually replaced by len- tiviral vectors based on ...
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Hemophilia A gene therapy via intraosseous delivery of factor VIII-lentiviral vectors
... replacement therapy, and FVIII-mimetic bispecific antibody is promising as an alternative treatment especially for patients with in- hibitory antibodies, these therapies require frequent, at least weekly dosing of ...
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Long-term gene therapy with Del1 fragment using nonviral vectors in mice with explanted tumors
... Cancer gene therapy using nonviral vectors is useful for long periods of treatment because such vectors are both safe and inexpensive, and thus can be used ...that gene therapy ...
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E- vectors: development of novel self-inactivating and self-activating retroviral vectors for safer gene therapy.
... retroviral vectors based on the previously observed high-frequency deletion of direct ...viral vectors that contained large direct repeats flanking the viral encapsidation sequence ...self-inactivating ...
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Gene Therapy Vectors Based on Adeno-Associated Virus Type 1
... and vectors were created with AAV-2 inverted terminal repeats and AAV-1 Rep and Cap ...2-based vectors transduced murine liver and muscle in vivo; AAV-1 was more efficient for muscle, while AAV-2 transduced ...
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Multicomponent nanoparticles as nonviral vectors for the treatment of Fabry disease by gene therapy
... the vectors in Hep G2 cells, we performed transfection studies with nano- particles bearing the pCMS-EGFP plasmid that encodes the green fluorescent ...new vectors for gene therapy, reporter ...
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Biology of adenovirus vectors with E1 and E4 deletions for liver-directed gene therapy.
... the therapy lead to toxicity, loss of transgene expression, and difficulties with vector ...adenovirus vectors that have both E1 and E4 ...liver-directed gene therapy indicated that the E1- ...
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The use of high-frequency ultrasound imaging and biofluorescence for in vivoevaluation of gene therapy vectors
... a gene therapy vector on tumour ...GFP gene or other com- ponents of the vector ...Indeed, gene therapy vectors are also used in non-cancer applications such as diabetes or organ ...
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Progresses towards safe and efficient gene therapy vectors
... with vectors pseudotyped with glycoproteins derived from Measles virus [143] or Tupaia paramyxovirus ...chimeric vectors might have a relevant clinical importance for lymphocyte-gene therapy ...
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Alpharetroviral Vectors: From a Cancer-Causing Agent to a Useful Tool for Human Gene Therapy
... and gene transcript truncation are the three most prevalent mechanisms of retroviral vector genotoxicity described to ...one gene and induce transcriptional deregulation by more than one mechanism ...
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The use of high-frequency ultrasound imaging and biofluorescence for in vivo evaluation of gene therapy vectors
... a gene therapy vector on tumour ...GFP gene or other com- ponents of the vector ...Indeed, gene therapy vectors are also used in non-cancer applications such as diabetes or organ ...
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HIV-Derived Lentiviral Vectors: Current Progress toward Gene Therapy and DNA Vaccination
... Lentiviral vectors are promising gene delivery tools capable of transducing a variety of dividing and non-dividing cells, including pluripotent stem cells which are refractory for transduction by murine ...
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Using a barcoded AAV capsid library to select for clinically relevant gene therapy vectors
... vivo gene therapy, either by supplying or repressing certain transcription factors using different viral or nonviral delivery systems (for example adeno-associated virus (AAV), adenovirus, lentivirus, ...
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Incidence and Prevalence of Neutralizing Antibodies to the Common Adenoviruses in Children With Cystic Fibrosis: Implication for Gene Therapy With Adenovirus Vectors
... infection. Limited published data on serum neutral- izing antibody in healthy children are available, but to the best of our knowledge no study in CF children has been performed. In our study, CF children seemed to make ...
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Gene therapy strategies for treating brain tumors: Retroviruses are still good candidates for therapeutic vectors
... Many gene therapy strategies for GBM have been developed and a few have progressed to clinical ...Retroviral vectors have superior features for gene therapy in brain cancers, including ...
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Adenovirus Vectors with the 100K Gene Deleted and Their Potential for Multiple Gene Therapy Applications
... 100K gene would allow for the production of a series of new classes of Ad vector, including one that is replication competent but blocked in the ability to carry out many late-phase Ad ...several gene ...
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Self-deleting retrovirus vectors for gene therapy.
... Gene therapy, one of the fastest-growing areas in medical research, bears the promise that in the near future it may be possible to correct aberrant gene function by the expression of normal genes in ...
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Evaluation of the concentration and bioactivity of adenovirus vectors for gene therapy.
... adenovirus vectors as potential therapeutic agents for multiple applications of in vivo human gene therapy has resulted in numerous preclinical and clinical ...of gene transfer (transgene ...
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THE USE OF VIRAL VECTORS IN GENE TRANSFER THERAPY
... Gene therapy is strategy based on using genes as pharmaceuticals. Gene therapy is a treatment that involves altering the genes inside body's cells to stop ...disease. Gene ...
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