FRRS1L gene replacement ameliorates disease phenotypes in the mouse model of developmental and epileptic encephalopathy 37
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Gene therapy offers unique therapeutic potential for treating rare genetic disorders such as DEE37, a severe Developmental and Epileptic Encephalopathy caused by biallelic loss-of-function mutations in the Ferric Chelate Reductase 1 Like (FRRS1L) gene, present... ...
