Article
Gene Therapy in Retinal Dystrophies.
International journal of molecular sciences - 14 Nov 2019
Ziccardi Lucia, Cordeddu Viviana, Gaddini Lucia, Matteucci Andrea, Parravano Mariacristina, Malchiodi-Albedi Fiorella, Varano Monica
Abstract excerpt
Inherited retinal dystrophies (IRDs) are a group of clinically and genetically heterogeneous degenerative disorders. To date, mutations have been associated with IRDs in over 270 disease genes, but molecular diagnosis still remains elusive in about a third of cases. The methodologic developments in genome sequencing techniques that we have witnessed in this last decade have represented a turning point not only in...
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