Matches in SemOpenAlex for { <https://semopenalex.org/work/W2301544183> ?p ?o ?g. }
Showing items 1 to 77 of
77
with 100 items per page.
- W2301544183 endingPage "25" @default.
- W2301544183 startingPage "1" @default.
- W2301544183 abstract "The ideal aim of gene therapy approach for the treatment of inherited disorders should involve a lasting and tissue specific expression of the functional gene. Besides, in gene therapy any technology used should fulfil several requirements, including safety, simplicity of use, cost effectiveness and amenability to industrial scale. To this end an in situ permanent correction of the defective endogenous gene (gene targeting approach) is preferable to a transient addition of exogenous non-integrating vectors expressing the wild type version of the gene or its cDNA (gene augmentation approach). In fact, the site-specific modification leads to a long term and genetically inheritable expression of the corrected gene, regardless its size.Moreover, in terms of time and space an in situ correction approach allows physiological expression of the targeted gene, since it remains under cell-specific regulatory regions. Gene targeting therapeutic approaches can be broadly divided into viral and non-viral gene transfer technology. Viral vectors take the advantage on the easy integration of the gene of interest into the host and high probability of its long–term expression but they are plagued by safety concerns. although less efficient at introducing and maintaining foreign gene expression Non-viral vectors (naked DNA fragments and plasmid DNA), have the profound advantage of being non-pathogenic and non-immunogenic.With reference to the naked DNA approach, the recent availability of better delivery methods (e.g. electroporation and microinjection) has made the non-viral gene transfer an increasingly more important and viable method for gene therapy.Since the viral based approaches are being investigated as well, the current chapter focuses on the use of naked DNA for the production of targeted alterations in the genome of mammalian cells. In particular it will review the most representative experimental strategies recently exploited, trying to consider advantages and down sides of techniques such as the non viral Sleeping Beauty transposon system (SB), the Short Fragment Homologous Recombination (SFHR), the chimeric RNA/DNA Oligonucleotides (RDO), the small interfering RNA (siRNA) and the homologous recombination dependent Gene Targeting (hrdGT)." @default.
- W2301544183 created "2016-06-24" @default.
- W2301544183 creator A5026998978 @default.
- W2301544183 creator A5033522371 @default.
- W2301544183 creator A5053120451 @default.
- W2301544183 creator A5085182142 @default.
- W2301544183 creator A5089434487 @default.
- W2301544183 creator A5091196696 @default.
- W2301544183 date "2005-01-01" @default.
- W2301544183 modified "2023-09-26" @default.
- W2301544183 title "Progress in Gene Therapy Research (Horizons in Cancer Research; Vol. 20)" @default.
- W2301544183 hasPublicationYear "2005" @default.
- W2301544183 type Work @default.
- W2301544183 sameAs 2301544183 @default.
- W2301544183 citedByCount "0" @default.
- W2301544183 crossrefType "journal-article" @default.
- W2301544183 hasAuthorship W2301544183A5026998978 @default.
- W2301544183 hasAuthorship W2301544183A5033522371 @default.
- W2301544183 hasAuthorship W2301544183A5053120451 @default.
- W2301544183 hasAuthorship W2301544183A5085182142 @default.
- W2301544183 hasAuthorship W2301544183A5089434487 @default.
- W2301544183 hasAuthorship W2301544183A5091196696 @default.
- W2301544183 hasConcept C104317684 @default.
- W2301544183 hasConcept C111599444 @default.
- W2301544183 hasConcept C111829913 @default.
- W2301544183 hasConcept C135983454 @default.
- W2301544183 hasConcept C150194340 @default.
- W2301544183 hasConcept C186852380 @default.
- W2301544183 hasConcept C195856716 @default.
- W2301544183 hasConcept C32470452 @default.
- W2301544183 hasConcept C40767141 @default.
- W2301544183 hasConcept C43953973 @default.
- W2301544183 hasConcept C54355233 @default.
- W2301544183 hasConcept C70721500 @default.
- W2301544183 hasConcept C86803240 @default.
- W2301544183 hasConceptScore W2301544183C104317684 @default.
- W2301544183 hasConceptScore W2301544183C111599444 @default.
- W2301544183 hasConceptScore W2301544183C111829913 @default.
- W2301544183 hasConceptScore W2301544183C135983454 @default.
- W2301544183 hasConceptScore W2301544183C150194340 @default.
- W2301544183 hasConceptScore W2301544183C186852380 @default.
- W2301544183 hasConceptScore W2301544183C195856716 @default.
- W2301544183 hasConceptScore W2301544183C32470452 @default.
- W2301544183 hasConceptScore W2301544183C40767141 @default.
- W2301544183 hasConceptScore W2301544183C43953973 @default.
- W2301544183 hasConceptScore W2301544183C54355233 @default.
- W2301544183 hasConceptScore W2301544183C70721500 @default.
- W2301544183 hasConceptScore W2301544183C86803240 @default.
- W2301544183 hasLocation W23015441831 @default.
- W2301544183 hasOpenAccess W2301544183 @default.
- W2301544183 hasPrimaryLocation W23015441831 @default.
- W2301544183 hasRelatedWork W1521389467 @default.
- W2301544183 hasRelatedWork W1577348060 @default.
- W2301544183 hasRelatedWork W1596792303 @default.
- W2301544183 hasRelatedWork W2094481373 @default.
- W2301544183 hasRelatedWork W2101875696 @default.
- W2301544183 hasRelatedWork W2110680416 @default.
- W2301544183 hasRelatedWork W2124451920 @default.
- W2301544183 hasRelatedWork W2145845738 @default.
- W2301544183 hasRelatedWork W2176653738 @default.
- W2301544183 hasRelatedWork W2235549814 @default.
- W2301544183 hasRelatedWork W2348289656 @default.
- W2301544183 hasRelatedWork W2386570364 @default.
- W2301544183 hasRelatedWork W2413608033 @default.
- W2301544183 hasRelatedWork W2606255398 @default.
- W2301544183 hasRelatedWork W2790991798 @default.
- W2301544183 hasRelatedWork W3108970728 @default.
- W2301544183 hasRelatedWork W3144607323 @default.
- W2301544183 hasRelatedWork W348520554 @default.
- W2301544183 hasRelatedWork W52529036 @default.
- W2301544183 hasRelatedWork W1480771986 @default.
- W2301544183 isParatext "false" @default.
- W2301544183 isRetracted "false" @default.
- W2301544183 magId "2301544183" @default.
- W2301544183 workType "article" @default.