Customized and scalable therapy of gene aploins

Section: 

   KEYWORDS

  •   RNA
  •   Enzymes
  •   Haploinsufficiency

 

 

 

 

 

 

 

Description

Development of the NHMV platform, for RNA-dependent stimulation of gene transcription: a new family of RNA programmable artificial enzymes, designed and developed in the Laboratorio di Sviluppo della Corteccia Cerebrale of SISSA (http://lccd.sissa.it/). They are 7 times smaller than the most famous CRISPR factors and differ from the latter due to the more moderate gene expression gain, limited to the physiological fluctuation range, as well as to the restriction of their activity to the already active genes. Thanks to these characteristics, they constitute an elective tool for the specific and scalable therapy of haploinsufficiency. After the publication of the initial version of the platform, the principles of the current year, experimental investigations are underway for its strengthening and refinement.

Applications

Haploinsufficiency is a genetic alteration underlying a large number of diseases, including many affecting the central nervous system. They are cumulatively fairly frequent and together constitute a market with great potential. However, their individual prevalence is low and the molecular consequences are extremely varied. This has made the development of targeted therapies so far difficult, due to the complexity of the problem and the particularly high investment / benefits ratio. NMHV enzymes have the right properties to cope with this impasse, making the problem attackable in a serial and scalable way.

Scientific Reference

Prof. Antonello Mallamaci