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2nd RNA'14 Therapeutics

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This meeting uses to explore new and in-depth developments currently being addressed in RNA therapies.

G. Pavlovic from PHENOMIN-ICS will present "The efficiency and simplicity of CRISPR/Cas9 technology to make precise changes to the genome of living cells has led to a new revolution in genetics"

  • CRISPR/Cas9 genome editing opens new possibilities for modelling human disease in rodents like easy generation of structural variant models and large humanisation of large locus in mouse
  • Scientists are concerned by CRISPR/Cas9 mediated off-targets but other CRISPR drawbacks are poorly evaluated
  • Especially, careful characterisation of mouse and rat generated by CRISPR/Cas9 show unexpected mutations like concatemers, ssODN random integration or chromosomal rearrangements
  • Finally, we will discuss how rodents can be used to evaluate safety of CRISPR/Cas9 therapeutic approaches especially for unexpected genome modifications

 

- Meeting

Barcelona, Spain

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