Biblio
Drug screening identifies tazarotene and bexarotene as therapeutic agents in multiple sulfatase deficiency. EMBO Mol Med. 2023:e14837.
Gain and loss of function variants in EZH1 disrupt neurogenesis and cause dominant and recessive neurodevelopmental disorders. Nat Commun. 2023;14(1):4109.
Generation of a human Tropomyosin 1 knockout iPSC line. Stem Cell Res. 2023;71:103161.
. Mapping PTBP2 binding in human brain identifies SYNGAP1 as a target for therapeutic splice switching. Nat Commun. 2023;14(1):2628.